Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.
Evaluating Nasal Nitric Oxide to Identify Evidence for PCD
The goal of the study is to measure nasal NO levels in patients suspected of having primary ciliary dyskinesia and to evaluate the usefulness of that measurement in the diagnosis of the condition.
Lessard, Margaret "Meg" -margaret.lessard@vcuhealth.org
Schechter, Michael, S
No gender available
Not specified
N/A
15162
HM20010023
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Epidemiology and Evolution of SARS-CoV-2 / COVID-19 and the Human Microbiome
The specific aim for this study is to document the evolution of the virus in the human nasopharynx and gut and determine the effect of the local microbiota on SARS-CoV-2 viral load, cytokine response, and clinical outcome.
National COVID Cohort Collaborative (N3C): A National Resource for Shared Analytics
The National COVID Cohort Collaborative (N3C) proposes to establish a central registry ofpatients who have been tested for COVID or have a clinical diagnosis of COVID.
ctrrecruit@vcu.edu
Gal, Tamas
No gender available
Not specified
N/A
16821
HM20019575
COVID-19
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Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis a
I. To determine whether the efficacy of treatment with selumetinib sulfate (selumetinib) as measured by event-free survival (EFS) is non-inferior to treatment with carboplatin/vincristine sulfate (vincristine) (CV) in previously untreated neurofibromatosis type 1 (NF1)-associated low-grade glioma (LGG).
II. To determine whether visual acuity (VA) using Teller acuity cards (TAC), in patients with NF1-associated LGG within the optic pathway, is better in those treated with selumetinib compared to CV.
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Massey, Gita, V.
All
2 year(s) to 21 year(s) old
III
NCT03871257
HM20018740
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Inclusion Criteria:
• Patients must be >= 2 years and =< 21 years at the time of enrollment
• Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment
• Patients must have neurofibromatosis type 1 (NF1) based on clinical criteria and/or
germline genetic testing
• Patients must be newly diagnosed or have previously diagnosed NF-1 associated LGG that
has not been treated with any modality other than surgery
• For patients with optic pathway gliomas (OPGs):
• Newly-diagnosed patients with OPG are eligible if there are neurologic symptoms
(including visual dysfunction, as defined below) or other exam findings
associated with the tumor
• Previously-diagnosed patients with OPG are eligible if they have new or worsening
neurologic symptoms (including visual dysfunction, as defined below) or have
tumor growth
• For both newly-diagnosed and previously-diagnosed OPG, the patient may be
eligible, irrespective of whether there has been tumor growth or other
neurological symptoms or worsening, if they meet at least one of the following
visual criteria:
• Visual worsening, defined as worsening of visual acuity (VA) or visual
fields (VF) documented within the past year (by examination or history); OR
• Significant visual dysfunction (defined as VA worse than normal for age by
0.6 logMAR [20/80, 6/24, or 2.5/10] or more in one or both eyes)
• For patients with LGG in other locations (i.e., not OPGs):
• Newly-diagnosed patients with LGG are eligible if there are neurologic symptoms
or other exam findings associated with the tumor
• NOTE: Newly-diagnosed patients with LGG without associated neurologic
symptoms or exam findings are not eligible
• Previously-diagnosed patients with LGG are eligible if they have new or worsening
neurologic symptoms or have tumor growth
• Although not required, if a biopsy/tumor resection is performed, eligible histologies
will include all tumors considered LGG or low-grade astrocytoma (World Health
Organization [WHO] grade I and II) by 5th edition WHO classification of central
nervous system (CNS) tumors with the exception of subependymal giant cell astrocytoma
• Patients must have two-dimensional measurable tumor >= 1 cm^2
• Patients with metastatic disease or multiple independent primary LGGs are allowed on
study
• Creatinine clearance or radioisotope glomerular filtration Rate (GFR) >= 70
mL/min/1.73 m^2 OR a serum creatinine based on age/gender (within 7 days prior to
enrollment) as follows:
• Age; maximum serum creatinine (mg/dL)
• 2 to < 6 years; 0.8 (male) and 0.8 (female)
• 6 to < 10 years; 1 (male) and 1 (female)
• 10 to < 13 years; 1.2 (male) and 1.2 (female)
• 13 to < 16 years; 1.5 (male) and 1.4 (female)
• >= 16 years; 1.7 (male) and 1.4 (female)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to
enrollment) (children with a diagnosis of Gilbert's syndrome will be allowed on study
regardless of their total and indirect [unconjugated] bilirubin levels as long as
their direct [conjugated] bilirubin is < 3.1 mg/dL)
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 3 x
upper limit of normal (ULN) = 135 U/L (within 7 days prior to enrollment). For the
purpose of this study, the ULN for SGPT is 45 U/L
• Albumin >= 2 g/dL (within 7 days prior to enrollment)
• Left ventricular ejection fraction (LVEF) >= 53% (or institutional normal; if the LVEF
result is given as a range of values, then the upper value of the range will be used)
by echocardiogram (within 4 weeks prior to enrollment)
• Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) (within 4 weeks
prior to enrollment)
• Absolute neutrophil count >= 1,000/uL (unsupported) (within 7 days prior to
enrollment)
• Platelets >= 100,000/uL (unsupported) (within 7 days prior to enrollment)
• Hemoglobin >= 8 g/dL (may be supported) (within 7 days prior to enrollment)
• Patients with a known seizure disorder should be stable and should have not
experienced a significant increase in seizure frequency within 2 weeks prior to
enrollment
• Patients 2-17 years of age must have a blood pressure that is =< 95th percentile for
age, height, and gender at the time of enrollment. Patients >= 18 years of age must
have a blood pressure =< 130/80 mmHg at the time of enrollment (with or without the
use of antihypertensive medications).
• Note: Adequate blood pressure can be achieved using medication for the treatment
of hypertension
• All patients must have ophthalmology toxicity assessments performed within 4 weeks
prior to enrollment
• For all patients, an MRI of the brain (with orbital cuts for optic pathway tumors)
and/or spine (depending on the site(s) of primary disease) with and without contrast
must be performed within 4 weeks prior to enrollment
• For patients who undergo a surgery on the target tumor (not required), a pre- and
post-operative* MRI of the brain (with orbital cuts for optic pathway tumors) or spine
(depending on the site(s) of primary disease) with and without contrast must also be
performed within 4 weeks prior to enrollment
• The post-operative MRIs should be performed ideally within 48 hours after surgery
if possible
• Patients must have a performance status corresponding to Eastern Cooperative Oncology
Group (ECOG) scores of 0, 1, or 2. Use Karnofsky for patients > 16 years of age and
Lansky for patients =< 16 years of age
• Patients must have the ability to swallow whole capsules
• Patients must have receptive and expressive language skills in English or Spanish to
complete the quality of life (QOL) and neurocognitive assessments
• All patients and/or their parents or legal guardians must sign a written informed
consent.
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met.
Exclusion Criteria:
• Patients must not have received any prior tumor-directed therapy including
chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior
surgical intervention is permitted
• Patients with a concurrent malignancy or history of treatment (other than surgery) for
another tumor within the last year are ineligible
• Patients may not be receiving any other investigational agents
• Patients with any serious medical or psychiatric illness/ condition, including
substance use disorders likely in the judgement of the investigator to interfere or
limit compliance with study requirements/treatment are not eligible
• Patients who, in the opinion of the investigator, are not able to comply with the
study procedures are not eligible
• Female patients who are pregnant are not eligible since fetal toxicities and
teratogenic effects have been noted for several of the study drugs. A pregnancy test
is required for female patients of childbearing potential
• Lactating females who plan to breastfeed their infants are not eligible
• Sexually active patients of reproductive potential who have not agreed to use an
effective contraceptive method for the duration of their study participation and for
12 weeks after stopping study therapy are not eligible
• Note: Women of child-bearing potential and males with sexual partners who are
pregnant or who could become pregnant (i.e., women of child-bearing potential)
should use effective methods of contraception for the duration of the study and
for 12 weeks after stopping study therapy to avoid pregnancy and/or potential
adverse effects on the developing embryo
• Cardiac conditions:
• Known genetic disorder that increases risk for coronary artery disease. Note: The
presence of dyslipidemia in a family with a history of myocardial infarction is
not in itself an exclusion unless there is a known genetic disorder documented
• Symptomatic heart failure
• New York Heart Association (NYHA) class II-IV prior or current cardiomyopathy
• Severe valvular heart disease
• History of atrial fibrillation
• Ophthalmologic conditions:
• Current or past history of central serous retinopathy
• Current or past history of retinal vein occlusion or retinal detachment
• Patients with uncontrolled glaucoma
• If checking pressure is clinically indicated, patients with intraocular
pressure (IOP) > 22 mmHg or ULN adjusted by age are not eligible
• Ophthalmological findings secondary to long-standing optic pathway glioma (such
as visual loss, optic nerve pallor, or strabismus) or longstanding
orbito-temporal plexiform neurofibroma (PN), such as visual loss, strabismus)
will NOT be considered a significant abnormality for the purposes of the study
• Treatments and/or medications patient is receiving that would make her/him ineligible,
such as:
• Supplementation with vitamin E greater than 100% of the daily recommended dose.
Any multivitamin containing vitamin E must be stopped prior to study enrollment
even if less than 100% of the daily recommended dosing for vitamin E
• Surgery within 2 weeks prior to enrollment, with the exception of surgical
placement for vascular access or cerebrospinal fluid (CSF) diverting procedures
such as endoscopic third ventriculostomy (ETV) and ventriculo-peritoneal (VP)
shunt.
• Note: Patients must have healed from any prior surgery prior to enrollment
• Patients who have an uncontrolled infection are not eligible
To identify key adverse events developing in patients (cases) with a primary cancer diagnosed at age 21 or younger
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Gowda, Madhu, S
All
up to 99 year(s) old
N/A
NCT00082745
HM6019
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Inclusion Criteria:
• ELIGIBILITY CRITERIA •CASES
• Diagnosis of primary cancer at age 21 or younger, irrespective of current age
• No prior history of allogeneic (non-autologous) hematopoietic cell transplant
• Development of one of the following key adverse events at any time following
initiation of cancer therapy:
• Cardiac dysfunction; please note: case enrollment has been closed due to
achievement of target accrual
• Ischemic stroke (IS)
• Subsequent malignant neoplasm (SMN)
• Avascular necrosis (AVN); please note: case enrollment has been closed due to
achievement of target accrual
• Submission of a blood specimen (or in certain cases a saliva specimen) to the
Coordinating Center at the University of Alabama at Birmingham as per the
requirements; please note: if a patient is currently receiving active cancer
treatment, it is preferable to obtain the blood sample at a time when the patient's
white blood cell (WBC) is > 2,000
• Written informed consent from the patient and/or the patient's legally authorized
guardian
• In active follow up by a COG institution; active follow up will be defined as date of
last visit or contact by a COG institution within the past 24 months; any type of
contact, including contact specifically for participation in ALTE03N1, qualifies as
active follow-up; please note: treatment on a COG (or legacy group) therapeutic
protocol for the primary cancer is NOT required
• ELIGIBILITY CRITERIA •CONTROLS
• CONTROL: Diagnosis of primary cancer at age 21 or younger, irrespective of current age
• CONTROLS: No prior history of allogeneic (non-autologous) hematopoietic cell
transplant
• CONTROLS: No clinical evidence of any of the following key adverse events:
• Cardiac dysfunction (CD); please note: if a patient is currently receiving active
cancer treatment, it is preferable to obtain the blood sample at a time when the
patient's WBC is > 2,000
• Ischemic stroke (IS)
• Avascular necrosis (AVN)
• Subsequent malignant neoplasm (SMN)
• CONTROLS: Submission of a blood specimen (or in certain cases a saliva specimen) to
the Coordinating Center Laboratory at the University of Alabama at Birmingham as per
the requirements
• CONTROLS: Written informed consent from the patient and/or the patient's legally
authorized guardian
• CONTROLS: In active follow up by a COG institution; active follow up will be defined
as date of last visit or contact by a COG institution within the past 24 months; any
type of contact, including contact specifically for participation in ALTE03N1,
qualifies as active follow-up; please note: treatment on a COG (or legacy group)
therapeutic protocol for the primary cancer is NOT required
Imatinib Mesylate + Chemo in Patients With Newly Diagnosed Philadelphia Chromosome+ ALL (BYPASS)
Primary Objective: To compare disease free survival (DFS) of Standard Risk (SR) pediatric Ph+ ALL treated with continuous imatinib combined with either a high-risk COG ALL chemotherapy backbone or the more intensive EsPhALL chemotherapy backbone.
Secondary Objective:
To determine the feasibility of administration of imatinib after allogeneic HSCT in High Risk (HR) Ph+ ALL patients.
To determine event-free survival (EFS) of High Risk pediatric Ph+ ALL patients treated with EsPhALL chemotherapy, HSCT in first complete remission and post-HSCT imatinib.
To compare rates of Grade 3 or higher infections in SR Ph+ ALL patients between the two randomized arms.
To evaluate EFS and overall survival (OS) of all enrolled participants.
To evaluate OS in SR patients.
To evaluate OS in HR patients.
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Griffin, Jordyn
All
1 year(s) to 21 year(s) old
III
NCT03007147
HM20015962
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Inclusion Criteria:
• For patients enrolled on APEC14B1 prior to enrollment on AALL1631, the required
diagnostic bone marrow sample has been fulfilled
• For patients who have not previously enrolled on APEC14B1 prior to enrollment on
AALL1631, a baseline diagnostic sample (or peripheral blood sample with blasts if
marrow sample unavailable) must be available to develop an MRD probe
• In addition, laboratory reports detailing evidence of BCR-ABL1 fusion or
ABL-class fusion must be submitted for rapid central review within 72 hours of
study enrollment
• >= 1 year (365 days) and =< 21 years at ALL diagnosis
• Ph+ (BCR-ABL1 fusion): newly diagnosed de novo ALL (B-ALL or T-ALL) or mixed
phenotypic acute leukemia (MPAL meeting 2016 World Health Organization [WHO]
definition) with definitive evidence of BCR-ABL1 fusion by karyotype, fluorescence in
situ hybridization (FISH) and/or molecular methodologies
• ABL-class fusion: newly diagnosed B-ALL with definitive evidence of ABL-class fusions.
ABL-class fusions are defined as those involving the following genes: ABL1, ABL2,
CSF1R, PDGFRB, PDGFRA. Methods of detection include fluorescence in-situ hybridization
(FISH, e.g. using break-apart or colocalization signals probes), multiplex or
singleplex reverse-transcription polymerase chain reaction (RT-PCR), whole
transcriptome or panel-based ribonucleic acid (RNA)-sequencing (e.g. TruSight RNA
Pan-Cancer Panel; Illumina, San Diego, CA, USA or similar)
• Ph+ patients must have previously started Induction therapy, which includes
vincristine, a corticosteroid, pegaspargase, with or without anthracycline, and/or
other standard cytotoxic chemotherapy
• Ph+ patients have not received more than 14 days of multiagent Induction therapy
beginning with the first dose of vinCRIStine
• Ph+ patients may have started imatinib prior to study entry but have not received more
than 14 days of imatinib
• ABL-class fusion patients must have previously completed the 4 or 5 weeks of
multiagent Induction chemotherapy (Induction IA phase)
• ABL-class fusion patients may have started imatinib during Induction IA, at the same
time of or after the first vinCRIStine dose
• Patients must have a performance status corresponding to Eastern Cooperative Oncology
Group (ECOG) scores of 0, 1, or 2
• Direct bilirubin =< 2.0 mg/dL
• Shortening fraction of >= 27% by echocardiogram
• Ejection fraction of >= 50% by radionuclide angiogram or echocardiogram
• Corrected QT interval, QTc < 480 msec
• Note: Repeat echocardiogram and electrocardiogram are not required if they were
performed at or after initial ALL diagnosis, before study enrollment
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 or serum creatinine within normal limits based on age/gender, as
follows:
• 1 to < 2 years: maximum serum creatinine 0.6 mg/dL (both male and female)
• 2 to < 6 years: maximum serum creatinine 0.8 mg/dL (both male and female)
• 6 to < 10 years: maximum serum creatinine 1 mg/dL (both male and female)
• 10 to < 13 years: maximum serum creatinine 1.2 mg/dL (both male and female)
• 13 to < 16 years: maximum serum creatinine 1.5 mg/dL (male), 1.4 mg/dL (female)
• >= 16 years: maximum serum creatinine 1.7 mg/dL (male), 1.4 mg/dL (female)
Exclusion Criteria:
• Known history of chronic myelogenous leukemia (CML)
• ALL developing after a previous cancer treated with cytotoxic chemotherapy
• Active, uncontrolled infection, or active systemic illness that requires ongoing
vasopressor support or mechanical ventilation
• Down syndrome
• Pregnancy and breast feeding
• Female patients who are pregnant since fetal toxicities and teratogenic effects
have been noted for several of the study drugs; a pregnancy test is required for
female patients of childbearing potential
• Lactating females who plan to breastfeed their infants
• Sexually active patients of reproductive potential who have not agreed to use an
effective contraceptive method for the duration of treatment according to
protocol
• Patients with congenital long QT syndrome, history of ventricular arrhythmias or heart
block
• Prior treatment with dasatinib, or any TKI other than imatinib
STOP PIV - Phase III DAS181 Lower Tract PIV Infection in Immunocompromised Subjects (STOP PIV)
The primary objective of the study is to demonstrate the effect of DAS181 on clinical outcome as measured by percentage of subjects returning to room air by Day 28 starting from the first day of treatment in only the PE subpopulation of the immunocompromised lower respiratory tract PIV-infected subject population.
Toft, Robin -robin.toft@vcuhealth.org
Miller, Kristin, B.
All
Not specified
III
NCT03808922
HM20016591
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Inclusion Criteria:
1. At the time of randomization, requires supplemental oxygen ?2 LPM due to hypoxemia.
2. Immunocompromised, as defined by one or more of the following:
• Received an autologous or allogeneic hematopoietic stem cell transplantation
(HSCT) at any time in the past
• Received a solid organ transplant at any time in the past
• Has been or is currently being treated with chemotherapy for hematologic
malignancies (e.g., leukemia, myeloma, lymphoma) and/or solid tumor malignancies
(e.g., lung, breast, brain cancer) at any time in the past
• Has an immunodeficiency due to congenital abnormality (only applicable to
subjects age < 18 years old) or pre-term birth (only applicable to subjects age ?
2 years old)
3. Has, within 3 days prior to randomization, a confirmed LRTI with a sialic acid
dependent respiratory virus
4. If female, subject must meet one of the following conditions:
• Not be of childbearing potential or
• Be of childbearing potential and have a negative urine/serum pregnancy test and
agrees to practice an acceptable method of contraception
5. Non-vasectomized males are required to practice effective birth control methods
6. Capable of understanding and complying with procedures as outlined in the protocol
7. Provides signed informed consent prior to the initiation of any screening or
study-specific procedures
For COVID-19 sub study:
1. Be ?18 years of age
2. Provide adequate medical history to permit accurate stratification (but health status
may be healthy, high-risk conditions, or immunocompromised).
3. Prior to SARS CoV 2 infection, has the ability to carry out self-care activities of
daily living (basic ADL)
4. Have lower respiratory tract infection (LRTI) confirmed by CT imaging, with or without
contrast, to involve at least 2 lobes of the lung.
5. Has laboratory-confirmation of the presence of SARS CoV 2 in the respiratory tract by
at least one of the following samples
6. Satisfy inclusion criteria #1, 4, 5, 6, 7 of the main study
Exclusion Criteria:
1. Subjects may not be on hospice care or, in the opinion of the investigator, have a low
chance of survival during the first 10 days of treatment
2. Subjects with Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), or
Alkaline Phosphatase (ALP) ?3x ULN and Total Bilirubin (TBILI) ?2x ULN Note: Subjects
with ALT/AST/ALP ? 3x ULN AND TB ?2x ULN that have been chronically stable (for >1
year on more than one assessments) due to known liver pathology including malignancy
(primary or metastasis), chronic medications, transplantation, or chronic infection
will not be excluded
3. Female subjects breastfeeding or planning to breastfeed at any time through 30 days
after the last dose of study drug
4. Subjects taking any other investigational drug used to treat pulmonary infection.
5. Psychiatric or cognitive illness or recreational drug/alcohol use that, in the opinion
of the principal investigator, would affect subject safety and/or compliance
6. Subjects with known hypersensitivity to DAS181 and/or any of its components
7. Subjects with severe sepsis due to either their baseline SAD-RV infection or a
concurrent viral, bacterial, or fungal infection and meet at least one of the
following criteria:
• Has evidence of vital organ failure outside of the lung (e.g., liver, kidney)
• Requires vasopressors to maintain blood pressure
For COVID-19 sub study:
1. Subjects requiring invasive mechanical, Bi-PAP or CPAP ventilation at randomization.
2. Subjects receiving any other investigational or empiric treatment for SARS-2-CoV
(either as part of a clinical trial or under emergency approval (approved agents for
the management of symptoms, e.g., fever, are permitted).
3. Subjects who are known HIV-positive (and not undetectable at most recent HIV RNA
assessment)
4. Subjects who are currently taking immunomodulating biologics (e.g, interferons,
interleukin)
5. Subjects with severe sepsis due to either their SARS-CoV-2 infection or a concurrent
viral, bacterial, or fungal infection and meeting at least one of the following
criteria:
• Have evidence of vital organ failure outside of the lung (e.g., liver, kidney)
• Require vasopressors to maintain blood pressure
6. Subjects meeting exclusion criteria #2, 3, 5 and 6 of the main study
The primary objective of this study is to determine whether the presence of APOL1 RRVs in a kidney donor shortens death-censored renal allograft survival.
The secondary objectives of this study are to:
1. define whether the presence of APOL1 RRVs in a kidney donor is associated with worse renal function or greater proteinuria after transplantation of the kidney;
2. define whether the presence of APOL1 RRVs is associated with worse renal outcomes in living kidney donors after nephrectomy, and;
3. (if donor APOL1 RRVs are associated with worse kidney function in recipients of renal allografts) identify modifying factors that increase susceptibility for shortened allograft survival, reduced renal allograft function or greater proteinuria in recipients of kidneys from donors with APOL1 high-risk genotypes.
Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
The intent of this protocol is to provide continued access to vamorolone for subjects in the United States who have completed the VBP15-LTE or VBP15-004 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time that VBP15-004 is ongoing, and while a new drug application for vamorolone is under preparation and review.
Qureshi, Mehreen -mehreen.qureshi@vcuhealth.org
Harper, Amy
No gender available
Not specified
II
NCT03863119
HM20016492
drug: Vamorolone
Diseases of Nervous System and Sense Organs (320-389)
ENhancing Recovery in CHildren Undergoing Surgery for IBD (ENRICH-US) (ENRICH-US)
Aim 1: Examine effectiveness of ENRICH-P for elective gastrointestinal (GI) surgery in pediatric patients with IBD, using a multicenter, cluster-randomized, pragmatic clinical trial by:
a) Aim 1a: Measuring the impact of ENRICH-P on clinical outcomes including LOS, opioid use, post-surgical complications, and healthcare utilization (clinic/ED visits, telephone calls, re- hospitalizations); and
b) Aim 1b: Assessing patient- and parent-reported health-related quality of life (HRQoL) outcomes; and
Aim 2: Assess implementation fidelity, sustainability, and site-specific adaptations of ENRICH-P for GI Surgery by:
a) Aim 2a: Gathering quantitative and qualitative measures of adoption, fidelity, and sustainability; and
b) Aim 2b: Identifying organizational, leadership, and competency-based drivers of effective implementation and sustainability, using qualitative methodologies
MAP to provide alpelisib (BYL719) for patients with PROS
The purpose of this Cohort Treatment Plan is to allow access to alpelisib for patients diagnosed with PIK3CA-Related Overgrowth Spectrum (PROS) who fulfill certain eligibility criteria.
Lessard, Margaret "Meg" -margaret.lessard@vcuhealth.org
Lastrapes, Kelly, K
All
2 year(s) to 99 year(s) old
N/A
NCT04085653
HM20023385
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Patients eligible for inclusion in this Treatment Plan have to meet all of the following
criteria:
1. Adult or pediatric patients ? 2 years of age, with a diagnosis of PROS preferably with
evidence of a mutation in the PIK3CA gene
2. The treating physician has determined that the patient's condition is severe or life
threatening, treatment is necessary and there are no other feasible alternatives for
the patient.
3. Confirmed adequate bone marrow function Written patient informed consent must be
obtained prior to start of treatment
Exclusion criteria
Patients eligible for this Treatment Plan must not meet any of the following criteria:
1. Patient has history of hypersensitivity to any drugs or metabolites of PI3K inhibitor
or any of the excipients of alpelisib.
2. Patient with uncontrolled diabetes mellitus type I or not controlled type II (based on
FPG and HbA1c, see inclusion criterion 2)
3. Patient who has other concurrent severe and/or uncontrolled medical conditions that
would, in the Treating Physician's judgment, contraindicate administration of
alpelisib (eg. active or uncontrolled severe infection, chronic active hepatitis,
immuno-compromised, acute or chronic pancreatitis, uncontrolled high blood pressure,
interstitial lung disease, etc.)
4. Patient has a known history of Severe Cutaneous Adverse Reactions (SCAR) like Steven
Johnson's syndrome (SJS), Erythema Multiforme (EM), Toxic Epidermal Necrolysis (TEN),
or Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS).
5. History of pancreatitis within 1 year of screening or past medical history of chronic
pancreatitis
6. Subject with Child Pugh score B or C
7. Subjects with unresolved osteonecrosis of the jaw
8. Subject is currently receiving any of the following medications and cannot be
discontinued 7 days prior to the start of the treatment:
• Strong inducers of CYP3A4
• Inhibitors of BCRP
9. Patient has a known history of Human Immunodeficiency Virus (HIV) infection (testing
not mandatory unless required by local regulations or requirements).
10. Patient who is concurrently being treated with drugs known to be strong inhibitors or
inducers of the isoenzyme CYP3A; switching to different medications prior to start of
program treatment is allowed within the last 5 days prior to starting program
treatment
11. Patient is currently receiving or has received systemic corticosteroids ? 2 weeks
prior to start of program treatment, or who have not fully recovered from side effects
of such treatment.
Note: The following uses of corticosteroids are permitted: single doses, topical
applications (e.g., for rash), inhaled sprays (e.g., for obstructive airways
diseases), eye drops or local injections (e.g., intra-articular).
12. Male patient who does not apply highly effective contraception during the treatment
with alpelisib and through the duration as defined below after the final dose of
alpelisib. Sexually active males should use a condom during intercourse while taking
drug and for at least 4 weeks after stopping alpelisib and should not father a child
in this period. A condom is required to be used also by vasectomized men in order to
prevent delivery of the drug via seminal fluid
13. Subject is not able to understand and to comply with treatment instructions and
requirements
14. Subject is a nursing (lactating) or pregnant woman as confirmed by a positive serum
(hCG) test prior to initiating study treatment
15. Subject is a woman of child-bearing potential defined as all women physiologically
capable of becoming pregnant, unless they are using highly effective methods of
contraception during study treatment and at least for 1 week after the last dose of
any study treatment.
Highly effective contraception methods include:
• Total abstinence (when this is in line with the preferred and usual lifestyle of
the subject ). Periodic abstinence (e.g., calendar, ovulation, symptom-thermal,
post-ovulation methods) and withdrawal are not acceptable methods of
contraception. Female sterilization (have had surgical bilateral oophorectomy
with or without hysterectomy), total hysterectomy or bilateral tubal ligation at
least 6 weeks before taking study treatment. In case of oophorectomy alone, only
when the reproductive status of the woman has been confirmed by follow up hormone
level assessment
• Male sterilization (at least 6 months prior to screening). For female subjects on
the study the vasectomized male partner should be the sole partner for that
subject
• Use of oral (estrogen and progesterone), injected or implanted combined hormonal
method of contraception or placement of an intrauterine device (IUD) or
intrauterine system (IUS), or forms of hormonal contraception that have
comparable efficacy (failure rate <1%), for example hormonal vaginal ring or
transdermal hormone contraception. In case of use or oral contraception, women
should have been stable on the same pill for a minimum of 3 months before taking
study treatment.
Note: Women are considered postmenopausal and not of child bearing potential if they
have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical
profile (i.e., age appropriate, history of vasomotor symptoms) or have had surgical
bilateral oophorectomy (with or without hysterectomy), total hysterectomy, or
bilateral tubal ligation at least 6 weeks before taking study treatment. In the case
of oophorectomy alone, only when the reproductive status of the woman has been
confirmed by follow up hormone level assessment is she considered not of child bearing
potential.
16. Subject is a sexually active male unwilling to use a condom during intercourse while
taking study treatment, and for 1 week after stopping alpelisib. A condom is required
for all sexually active male participants to prevent them from fathering a child AND
to prevent delivery of study treatment via seminal fluid to their partner. In
addition, male participants must not donate sperm during study and up to the time
period specified above.
Drug: alpelisib, Modality: No vcuhs billing,
drug: Byl719
Blinatumomab + Chemo in Treating Patients +/- Down Syndrome and Newly Diagnosed B-ALL
Primary Aims
1.1.1 To determine in a randomized manner if the addition of 2 cycles of blinatumomab to standard therapy improves disease-free survival (DFS) in patients with SR B-ALL and higher risk features (SR-High), and patients with standard-risk average (SR-Avg) B-ALL who are negative for minimal residual disease (MRD) by flow cytometry but have detectable or indeterminate MRD as measured by high-throughput sequencing (HTS) at end of Induction (EOI).
1.1.2 To confirm that boys in the standard-risk favorable (SR-Fav) subset of B-ALL, with or without DS, will maintain a 5-year DFS of greater than 93% when treated with a standard chemotherapy regimen with a treatment duration of 2 years from the start of Interim Maintenance I (IM1).
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Sisler, India, Y
All
1 year(s) to 31 year(s) old
III
NCT03914625
HM20017413
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Inclusion Criteria:
• All B-ALL patients must be enrolled on APEC14B1 and consented to Eligibility Screening
(Part A) prior to treatment and enrollment on AALL1731. APEC 14B1 is not a requirement
for B-LLy patients. B-LLy patients may directly enroll on AALL1731.
• Age at diagnosis:
• Patients must be >= 365 days and < 10 years of age (B-ALL patients without DS).
• Patients must be >= 365 days and =< 31 years of age (B-ALL patients with DS).
• Patients must be >= 365 days and =< 31 years of age (B-LLy patients with or
without DS).
• B-ALL patients without DS must have an initial white blood cell count < 50,000/uL
(performed within 7 days prior to enrollment).
• B-ALL patients with DS are eligible regardless of the presenting white blood cell
count (WBC) (performed within 7 days prior to enrollment).
• Patient has newly diagnosed B-cell ALL, with or without Down syndrome: > 25% blasts on
a bone marrow (BM) aspirate;
• OR if a BM aspirate is not obtained or is not diagnostic of B-ALL, the diagnosis
can be established by a pathologic diagnosis of B-ALL on a BM biopsy;
• OR a complete blood count (CBC) documenting the presence of at least 1,000/uL
circulating leukemic cells;
• OR patient has newly diagnosed B-cell LLy Murphy stages I or II, with or without
Down syndrome.
• Note: For B-LLy patients with tissue available for flow cytometry, the criterion
for diagnosis should be analogous to B-ALL. For tissue processed by other means
(i.e., paraffin blocks), the methodology and criteria for immunophenotypic
analysis to establish the diagnosis of B-LLy defined by the submitting
institution will be accepted (diagnostic biopsy for B-LLy must be performed
within 14 days prior to enrollment).
• All patients and/or their parents or legal guardians must sign a written informed
consent.
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met.
Exclusion Criteria:
• Patient must not have secondary ALL that developed after treatment of a prior
malignancy with cytotoxic chemotherapy. Note: patients with Down syndrome with a prior
history of transient myeloproliferative disease (TMD) are not considered to have had a
prior malignancy. They would therefore be eligible whether or not the TMD was treated
with cytarabine.
• With the exception of steroid pretreatment or the administration of intrathecal
cytarabine, patients must not have received any prior cytotoxic chemotherapy for
either the current diagnosis of B ALL or B LLy or for any cancer diagnosed prior to
initiation of protocol therapy on AALL1731.
• For patients receiving steroid pretreatment, the following additional exclusion
criteria apply:
• Non-DS B-ALL patients must not have received steroids for more than 24 hours in
the 2 weeks prior to diagnosis without a CBC obtained within 3 days prior to
initiation of the steroids.
• DS and non-DS B-LLy patients must not have received > 48 hours of oral or IV
steroids within 4 weeks of diagnosis.
• Patients who have received > 72 hours of hydroxyurea within 1 week (7 days) prior to
the start of systemic protocol therapy.
• B-ALL patients who do not have sufficient diagnostic bone marrow submitted for
APEC14B1 diagnostic testing and who do not have a peripheral blood sample submitted
containing > 1,000/uL circulating leukemia cells.
• Patient must not have acute undifferentiated leukemia (AUL).
• Non-DS B-ALL patients with central nervous system [CNS]3 leukemia (CNS status must be
known prior to enrollment).
• Note: DS patients with CNS3 disease are eligible but will be assigned to the
DS-High B-ALL arm. CNS status must be determined based on a sample obtained prior
to administration of any systemic or intrathecal chemotherapy, except for steroid
pretreatment.
• Non-DS B-ALL patients with testicular leukemia. (Note: DS patients with testicular
disease are eligible but will be assigned to the DS-High B-ALL arm).
• For LLy patients, the following additional exclusion criteria apply:
• T-Lymphoblastic Lymphoma.
• Morphologically unclassifiable lymphoma.
• Absence of both B-cell and T-cell phenotype markers in a case submitted as
lymphoblastic lymphoma.
• CNS positive disease or testicular involvement.
• M2 (5% •25% blasts) or M3 (> 25% blasts) marrow.
• Patients with known Charcot-Marie-Tooth disease.
• Patients with known MYC translocation associated with mature (Burkitt) B-cell ALL,
regardless of blast immunophenotype.
• Patients requiring radiation at diagnosis.
• Female patients who are pregnant since fetal toxicities and teratogenic effects have
been noted for several of the study drugs. A pregnancy test is required for female
patients of childbearing potential.
• Lactating females who plan to breastfeed their infants.
• Sexually active patients of reproductive potential who have not agreed to use an
effective contraceptive method for the duration of their study participation.
TKI to Assess Treatment-Free Remission in Pediatric CML - Chronic Phase (CML-CP)
Primary Aims
To determine the 2-year treatment free remission (TFR) rate of children, adolescents, and young adults with CML-CP following discontinuation TKI.
To estimate the re-induction rate and maintenance of major molecular remission (MMR/MR3) at 1 year after restarting TKI for children, adolescents, and young adults.
Secondary Aim
To describe clinical factors and laboratory correlates affecting the persistence of MMR and re-initiation of treatment after stopping TKI (e.g. patient demographics, duration and level of prior molecular remission, duration and type of TKI, clinical presentation at diagnosis and immune studies).
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Massey, Gita, V.
All
up to 25 year(s) old
II
NCT03817398
HM20019724
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Inclusion Criteria:
• Patient must have been diagnosed with CML-CP at < 18 years of age.
• Patient must have histologic verification of CML-CP at original diagnosis
• Patient must be in molecular remission (MR) with a BCR-ABL1 level of =< 0.01% BCR-ABL1
as measured using the International Scale (IS) by RQ-PCR for >= 2 consecutive years at
the time of enrollment
• Please note: The lab evaluating disease status and molecular response for this
study must be College of American Pathology (CAP) and/or Clinical Laboratory
Improvement Amendments (CLIA) certified (United States [US] only), sites in other
countries must be certified by their accredited authorities. All labs must use
the International Scale guidelines with a sensitivity of detection assay =< 0.01%
BCR-ABL1 and be able to report results in =< 2 weeks
• Patient must have received any TKI for a minimum of 3 consecutive years at time of
enrollment
• Patient agrees to discontinue TKI therapy
• REGULATORY REQUIREMENTS
• All patients and/or their parents or legal guardians must sign a written informed
consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met
• ELIGIBILITY FOR PATIENT-REPORTED OUTCOMES (PROs):
• Age >= 8 years at the time of enrollment
• Ability to understand English or Spanish
• Cognitive ability to complete instruments according to the primary team
• ELIGIBILITY FOR AAML18P1 NEUROCOGNITIVE STUDY:
• Patient must be 5 years or older at the time of enrollment
• English-, French- or Spanish-speaking
• No known history of neurodevelopmental disorder prior to diagnosis of CML (e.g., Down
syndrome, Fragile X, William syndrome, mental retardation)
• No significant visual or motor impairment that would prevent computer use or
recognition of visual test stimuli
Exclusion Criteria:
• Known T3151 mutation
• Additional clonal chromosomal abnormalities in Philadelphia chromosome (Ph) positive
(+) cells at any time prior to enrollment that include "major route" abnormalities
(second Ph, trisomy 8, isochromosome 17q, trisomy 19), complex karyotype or
abnormalities of 3q26.2
• History of accelerated phase or blast crisis CML
• Female patients who are pregnant
• Lactating females are not eligible unless they have agreed not to breastfeed their
infants
• Female patients of childbearing potential are not eligible unless a negative pregnancy
test result has been obtained
Overall:
Our approach is to enroll large cohorts from each of the four most prevalent, amenable to therapy, LGMDs (n=20 each): CAPN3, ANO5, DYSF, and DNAJB6. We will perform a 12-month longitudinal study using 8 sites in our LGMD Research Consortium. We hypothesize that COAs representing the major shoulder and pelvic girdle functional burden can be shared across all LGMDs, and genetic variability can be addressed by adding select mutation-relevant COAs to create a LGMD COA toolbox for future trials.
Specific:
1. To define a common set of pelvic and shoulder girdle COAs, and COAs useful for capturing phenotypic diversity. Participants will have visits for a minimum of 12 months. Visit frequency will be annually. We will use standard measures of motor strength and evaluator-administered functional motor tasks which target proximal, mid-limb, and distal upper and lower extremity function. We will also assess pulmonary capacity and patient-reported measures of functional disease burden. We will collect biological samples and store them for future biomarker discovery. Cross sectional analysis will assess variance between cohorts and within cohorts on select COAs to determine convergent and divergent phenotypes.
2. To determine the sensitivity to longitudinal disease progression. We will determine the responsiveness of our COAs at 12 months both across the cohorts and within each cohort. We will use statistical and anchoring methods to determine what will be a minimally important clinical difference. We will use factor analysis to identify COAs that capture variation in disease severity across the cohort.
3. To refine clinical trial strategies based on baseline and longitudinal phenotypic characteristics. We will focus on COAs that are applicable across cohorts. We will determine which baseline characteristics, mutation, age, gender, and baseline functional status are most likely to predict progression over 12 months, using regression trees to assess subgroups likely to progress.
Bulti, Abel -abel.bulti@vcuhealth.org Jennifer Raymond -Jennifer.Raymond@vcuhealth.org
Johnson, Nicholas, E
All
4 year(s) to 65 year(s) old
N/A
NCT03981289
HM20018721
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Inclusion Criteria •Arm 1:
• Age between 4-65 at enrollment
• Clinically affected (defined as weakness on bedside evaluation in either a limb-girdle
pattern, or in a distal extremity)
• A genetically or functionally confirmed mutation in ANO5, CAPN3, DYSF, DNAJB6 or
SGCA-G.
• Willing and able to give informed consent and follow all study procedures and
requirements
Inclusion Criteria •Arm 2:
• Age between 4-65 at enrollment
• Clinically affected (defined as weakness on bedside evaluation in either a limb-girdle
pattern, or in a distal extremity)
• a genetically confirmed mutation in SGCA-G
• Willing and able to give informed consent and follow all study procedures and
requirements
Exclusion Criteria •Arm 1:
• Any other illness that would interfere with the ability to undergo safe testing or
would interfere with interpretation of the results in the opinion of the site
investigator.
• History of a bleeding disorder, platelet count <50,000, current use of an
anticoagulant.
• Positive pregnancy test at time any timepoint during the trial.
Exclusion Criteria •Arm 2:
• Any other illness that would interfere with the ability to undergo safe testing or
would interfere with interpretation of the results in the opinion of the site
investigator.
• History of a bleeding disorder, platelet count <50,000, current use of an
anticoagulant
• Positive pregnancy test at time any timepoint during the trial.
Limb Girdle Muscular Dystrophy, Muscular Dystrophies, Diseases of Nervous System and Sense Organs (320-389)
Inotuzumab Ozogamicin and Post-Induction Chemo in Treating Patients With High-Risk B-ALL, Mixed Phen
To determine in a randomized manner if the addition of 2 blocks of inotuzumab ozogamicin to modified Berlin-Frankfurt-Münster (mBFM) chemotherapy will improve 5-year disease-free survival (DFS) in children and young adults with High-Risk (HR) B-cell acute
lymphoblastic leukemia (B-ALL).
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Griffin, Jordyn
All
1 year(s) to 25 year(s) old
III
NCT03959085
HM20018568
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Inclusion Criteria:
• B-ALL and MPAL patients must be enrolled on APEC14B1 and consented to eligibility
studies (Part A) prior to treatment and enrollment on AALL1732. Note that central
confirmation of MPAL diagnosis must occur within 22 business days after enrollment for
MPAL patients. If not performed within this time frame, patients will be taken off
protocol.
• APEC14B1 is not a requirement for B-LLy patients but for institutional compliance
every patient should be offered participation in APEC14B1. B-LLy patients may directly
enroll on AALL1732.
• Patients must be > 365 days and < 25 years of age
• White blood cell count (WBC) criteria for patients with B-ALL (within 7 days prior to
the start of protocol-directed systemic therapy):
• Age 1-9.99 years: WBC >= 50,000/uL
• Age 10-24.99 years: Any WBC
• Age 1-9.99 years: WBC < 50,000/uL with:
• Testicular leukemia
• CNS leukemia (CNS3)
• Steroid pretreatment.
• White blood cell count (WBC) criteria for patients with MPAL (within 7 days prior to
the start of protocol-directed systemic therapy):
• Age 1-24.99 years: any WBC.
• Patient has newly diagnosed B-ALL or MPAL (by World Health Organization [WHO] 2016
criteria) with >= 25% blasts on a bone marrow (BM) aspirate;
• OR If a BM aspirate is not obtained or is not diagnostic of acute leukemia, the
diagnosis can be established by a pathologic diagnosis of acute leukemia on a BM
biopsy;
• OR A complete blood count (CBC) documenting the presence of at least 1,000/uL
circulating leukemic cells if a bone marrow aspirate or biopsy cannot be
performed.
• Patient has newly diagnosed B-LLy Murphy stages III or IV.
• Patient has newly diagnosed B-LLy Murphy stages I or II with steroid pretreatment.
• Note: For B-LLy patients with tissue available for flow cytometry, the criterion for
diagnosis should be analogous to B-ALL. For tissue processed by other means (i.e.,
paraffin blocks), the methodology and criteria for immunophenotypic analysis to
establish the diagnosis of B-LLy defined by the submitting institution will be
accepted.
• All patients and/or their parents or legal guardians must sign a written informed
consent.
• All institutional, Food and Drug Administration (FDA), and NCI requirements for human
studies must be met.
Exclusion Criteria:
• Patients with Down syndrome are not eligible (patients with Down syndrome and B-ALL
are eligible for AALL1731, regardless of NCI risk group).
• With the exception of steroid pretreatment or the administration of intrathecal
cytarabine, patients must not have received any prior cytotoxic chemotherapy for the
current diagnosis of B-ALL, MPAL, or B-LLy or for any cancer diagnosed prior to
initiation of protocol therapy on AALL1732.
• Patients who have received > 72 hours of hydroxyurea within one week prior to start of
systemic protocol therapy.
• Patients with B-ALL or MPAL who do not have sufficient diagnostic bone marrow
submitted for APEC14B1 testing and who do not have a peripheral blood sample submitted
containing > 1,000/uL circulating leukemia cells.
• Patients with acute undifferentiated leukemia (AUL) are not eligible.
• For Murphy stage III/IV B-LLy patients, or stage I/II patients with steroid
pretreatment, the following additional exclusion criteria apply:
• T-lymphoblastic lymphoma.
• Morphologically unclassifiable lymphoma.
• Absence of both B-cell and T-cell phenotype markers in a case submitted as
lymphoblastic lymphoma.
• Patients with known Charcot-Marie-Tooth disease.
• Patients with known MYC translocation associated with mature (Burkitt) B-cell ALL,
regardless of blast immunophenotype.
• Patients requiring radiation at diagnosis.
• Female patients who are pregnant, since fetal toxicities and teratogenic effects have
been noted for several of the study drugs. A pregnancy test is required for female
patients of childbearing potential.
• Lactating women who plan to breastfeed their infants while on study and for 2 months
after the last dose of inotuzumab ozogamicin.
• Sexually active patients of reproductive potential who have not agreed to use an
effective contraceptive method for the duration of study participation. For those
patients randomized to inotuzumab ozogamicin, there is a minimum of 8 months after the
last dose of inotuzumab ozogamicin for females and 5 months after the last dose of
inotuzumab ozogamicin for males.
B Acute Lymphoblastic Leukemia, B Lymphoblastic Lymphoma, Central Nervous System Leukemia, Mixed Phenotype Acute Leukemia, Testicular Leukemia, Lymphoid Leukemia, Non-Hodgkin's Lymphoma, Myeloid and Monocytic Leukemia, Leukemia, other
HeadStart4: Newly Diagnosed Children <10 y/o w/ Medulloblastoma and Other CNS Embryonal Tumors
Dose-intensive tandem Consolidation will be compared with single cycle consolidation via randomization. The randomized consolidations will provide an event-free survival (EFS) analysis after completing "Head Start 4" Induction.
Dose-intensive tandem Consolidation will be compared with single cycle consolidation via randomization. The randomized consolidations will provide an overall survival (OS) analysis after completing "Head Start 4" Induction.
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Wang, Zhihong, Joanne
All
up to 10 year(s) old
IV
NCT02875314
HM20015352
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Inclusion Criteria:
• Patients 10 years of age at the time of definitive confirmatory eligible histologic or
cytologic diagnosis of eligible CNS tumor (brain or spinal cord)
• Patients may not have received irradiation or chemotherapy (except corticosteroids)
• Have histologically proven diagnosis of medulloblastoma or CNS embryonal tumors of the
brain or spinal cord
• Medulloblastoma
• Posterior fossa classic, desmoplastic or extensive nodular or anaplastic/large
cell medulloblastoma with appropriate and sufficient tumor material (FFPE or snap
frozen) for proposed assays: all stages, age less than 6 years at diagnosis
• Posterior fossa classic or anaplastic/large cell medulloblastoma with sufficient
tumor material (FFPE or snap frozen) for proposed assays: clinically high-stage
(neuraxis or extra-neural dissemination, M1-4), age greater than 6 years to less
than 10 years at diagnosis
• Posterior fossa medulloblastoma, those 6 years of age and above at diagnosis,
will only be eligible if they have evidence of neuraxis or extraneural
dissemination. Patients 6 years of age and above with low-stage (standard-risk,
M0) medulloblastoma will NOT be eligible for this study, irrespective of
molecular subgroup and extend of local resection
• CNS Embryonal Tumors:
•Pineoblastoma, CNS neuroblastoma, CNS ganglioneuroblastoma, embryonal tumor with
multi-layered rosettes (ETMR, including embryonal tumor with abundant neuropil and
true rosettes (ETANTR), ependymoblastoma and ETMR not otherwise specified),
medulloepithelioma, CNS embryonal tumor with rhabdoid features (INI1 intact) and CNS
embryonal tumor, not otherwise specified.
• Must commence Induction chemotherapy within 28 days of the most recent definitive
surgical procedure and within 21 days of the most recent neuro-imaging studies (MRI of
brain, performed with and without gadolinium contrast, and MRI of total spine,
performed with gadolinium contrast) and lumbar CSF cytological examination
• Patients must have adequate organ functions at the time of registration:
• Liver: bilirubin less than 1.5 mg/dL (except for patients with Gilbert's Syndrome
of indirect hyperbilirubinemia) and transaminases [SGPT or ALT, and SGOT or AST]
less than 2.5 (two and a half) times the upper limits of institutional normal.
• Renal: Creatinine clearance and/or glomerular filtration rate (GFR) greater than
or equal to 60 mL/min/1.73m² within 21 days of protocol therapy.
• Bone Marrow Function:
1. Peripheral absolute phagocyte count (APC) > 1000/ µL. APC = numbers of
banded neutrophils + segmented neutrophils + metamyelocytes + monocytes +
eosinophils Please note, if institution reports differential as a
percentage, then APC = [percentage of banded neutrophils + segmented
neutrophils+ metamyelocytes+monocytes+eosinophils] x total white cell count.
2. Platelet Count > 100,000/µL (transfusion independent)
3. Hemoglobin > 8 gm/dL (may have received RBC transfusions).
Exclusion Criteria:
• Patients older than 10 years of age at time of diagnosis
• Following diagnoses are not eligible for study enrollment: CNS atypical
teratoid/rhabdoid tumor (AT/RT); all ependymomas including anaplastic ependymomas of
the brain or spinal cord; all choroid plexus carcinomas; all high-grade glial and
glio-neuronal tumors; all primary CNS germ cell tumors; all primary CNS sarcomas; all
primary or metastatic CNS lymphomas and solid leukemic lesions (i.e., chloromas,
granulocytic sarcomas).
• Patients with unbiopsied diffuse intrinsic pontine tumors will NOT be eligible for
this study.
Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
The primary aim of this study is to examine the incidence of neutrophil recovery of 500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not FDA licensed.
Lantis, Kristin -kllantis@vcu.edu
McCarty, John, M.
All
Not specified
N/A
NCT01351545
HM13913
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Inclusion Criteria:
• Disorders affecting the hematopoietic system that are inherited, acquired, or result
from myeloablative treatment
• Signed informed consent (and signed assent, if applicable) obtained prior to study
enrollment
• Pediatric and adult patients of any age
Exclusion Criteria:
• Patients who are receiving only licensed CBUs
• Cord blood transplant recipients at international transplant centers
• Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)
• Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs), Modality: Therapy (nos)
Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-Hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Autoimmune Diseases, Severe Aplastic Anemia, Other Hematopoietic, Non-Hodgkin's Lymphoma, Myeloid and Monocytic Leukemia, Multiple Myeloma, Lymphoid Leukemia, Hodgkin's Lymphoma
Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units (CBUs)
The primary aim of this study is to examine the safety of administration of the unlicensed investigational NCBP HPC-CORD BLOOD products in a multi-institution setting. The study will evaluate prospectively the incidence of serious adverse reactions as well as the incidence of all infusion related reactions after administration of unlicensed, investigational NCBP CBUs.
Lantis, Kristin -kllantis@vcu.edu
McCarty, John, M.
All
Not specified
II
NCT01656603
HM14815
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Inclusion Criteria:
1. Diagnosis: Patients with disorders affecting the hematopoietic system that are
inherited, acquired, or result from myeloablative treatment.
2. Patients: Patients of any age and either gender
3. Cord blood product manufactured by the NCBP (at least one, if the graft contains more
than one units)
Exclusion Criteria:
1. Patients who are receiving licensed cord blood products (only)
2. Patients who are receiving unlicensed cord blood products from other banks (only)
3. Patients who are transplanted at non-US transplant centers
4. Patients who are receiving cord blood products that will be "manipulated" post-thaw
(e.g., ex vivo expansion, incubation in vitro, etc.)
Active Surveillance, Bleomycin, Carbo, Etoposide, or Cisplatin in Pediatric & Adult Germ Cell Tumors
1 To evaluate whether a strategy of complete surgical resection followed by surveillance can maintain an overall survival rate of at least 95.7% at two years for pediatric, adolescent and adult patients (ages 0- 50 years) with Stage I (low risk) malignant germ cell tumors, and at least 98% for patients with ovarian pure immature teratoma.
2 To compare the event-free survival of a carboplatin vs. cisplatin-based regimen in the treatment of pediatric, adolescent and young adult patients with standard risk germ cell tumors.
2.1 To compare the EFS of a carboplatin-based regimen (CEb) vs. a cisplatin-based regimen (PEb) in children (less than 11 years in age) with standard risk GCT.
2.2 To compare the EFS of a carboplatin-based regimen (BEC) vs. a cisplatin-based regimen (BEP) in adolescents and young adults (ages 11 - 25 years) with standard risk GCT.
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Austin, Frances
All
Not specified
III
NCT03067181
HM20015718
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Inclusion Criteria:
• There is no age limit for the low risk stratum (stage I ovarian immature teratoma and
stage I non-seminoma or seminoma malignant GCT [all sites])
• Standard risk 1: Patient must be < 11 years of age at enrollment
• Standard risk 2: Patients must be >= 11 and < 25 years of age at enrollment
• Patients enrolling on one of the low risk arms must be newly diagnosed with a stage I
germ cell tumor; for the standard risk arms, patients must be newly diagnosed with
metastatic germ cell tumor (stage II or higher); histologic confirmation of a primary
extracranial germ cell tumor in any of the categories outlined below is required of
all patients at enrollment except for those who were initially diagnosed with stage I
non-seminoma malignant GCT and later recur during observation post surgery off study;
for these patients, if elevated tumor markers rise to > 5 x upper limit of normal
(ULN) on at least 2 measurements taken at least 1 week apart, a diagnostic biopsy is
not required for enrollment
• Low risk stage I immature teratoma (IT); site: ovarian; stage: Children's Oncology
Group (COG) stage I, Federation of Gynecology and Obstetrics (FIGO) stage IA and IB;
grade: 2 or 3; histology: pure immature teratoma (may contain microscopic foci of yolk
sac tumor), mixed immature and mature teratoma, (no pathological evidence of MGCT);
tumor markers: alpha-FP =< 1,000 ng/mL, beta-HCG institutional normal; all ages
• Low risk stage I non-seminoma MGCT; site: ovarian, testicular, or extragonadal; stage:
COG stage I, FIGO stage IA and IB, American Joint Committee on Cancer (AJCC)
testicular stage IA, IB and IS; histology: must contain at least one of the following:
yolk sac tumor, embryonal carcinoma, or choriocarcinoma (pure or mixed); all ages
• Low risk stage I seminoma-MGCT; site: testicular; stage: COG stage I; AJCC testicular
stage IA IB, and IS; histology: must contain at least one of the following: may
contain immature/mature teratoma; may NOT contain yolk sac tumor, embryonal carcinoma,
or choriocarcinoma; all ages
• Standard risk 1 (SR1); site: ovarian, testicular, or extragonadal; stage: COG stage
II-IV, FIGO stage IC, FIGO stage II-IV (International Germ Cell Consensus
Classification [IGCCC] criteria DO NOT apply); histology: must contain at least one of
the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) <
11
• Standard risk 2 (SR2)
• Site: ovarian; stage: COG stage II and III, FIGO stage IC, II and III; histology:
must contain at least one of the following: yolk sac tumor, embryonal carcinoma,
or choriocarcinoma; age (years) >= 11 and < 25
• Site: testicular; stage: COG stage II-IV, AJCC stage II, III, IGCCC good risk;
histology: must contain at least one of the following: yolk sac tumor, embryonal
carcinoma, or choriocarcinoma; tumor markers: must be IGCCC good risk; post op:
alpha-FP < 1,000 ng/mL, beta-HCG < 5,000 IU/mL and lactate dehydrogenase (LDH) <
3.0 x normal; age (years) >= 11 and < 25
• Site: extragonadal; stage: COG stage II; histology: must contain at least one of
the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age
(years) >= 11 and < 25
• Notes:
• IGCCC criteria only apply to SR2 patients with a testicular primary tumor
• Use post-op tumor marker levels to determine IGCCC risk group
• Stage 1 seminoma patients are not eligible for the standard risk arms of the
study
• For the low risk stage I non-seminoma MGCT and the standard risk arms, components
of yolk sac tumor, embryonal carcinoma, or choriocarcinoma can be mixed with
other forms of GCT, such as seminoma or mature or immature teratoma; if yolk sac
tumor is the only malignant component present, then it must be deemed by the
pathologist to be greater than a "microscopic component" of yolk sac tumor
• Patients must have a performance status corresponding to Eastern Cooperative Oncology
Group (ECOG) scores of 0, 1, 2 or 3; use Karnofsky for patients > 16 years of age and
Lansky for patients =< 16 years of age
• Organ function requirements apply ONLY to patients who will receive chemotherapy (SR1
and SR2 patients)
• Adequate renal function defined as:
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 (within 7 days prior to enrollment) OR
• A serum creatinine based on age/gender as follows (within 7 days prior to enrollment):
(mg/dL)
• 1 month to < 6 months male: 0.4 female: 0.4
• 6 months to < 1 year male: 0.5 female: 0.5
• 1 to < 2 years male: 0.6 female: 0.6
• 2 to < 6 years male: 0.8 female: 0.8
• 6 to < 10 years male: 1 female: 1
• 10 to < 13 years male: 1.2 female: 1.2
• 13 to < 16 years: male: 1.5 female: 1.4
• >= 16 years male: 1.7 female: 1.4
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to
enrollment)
• Serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or
serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x
upper limit of normal (ULN) for age (for the purpose of this study, the ULN for SGPT
is 45 U/L) (within 7 days prior to enrollment)
• Peripheral absolute neutrophil count (ANC) >= 1,000/mm^3 (within 7 days prior to
enrollment) AND
• Platelet count >= 100,000/mm^3 (within 7 days prior to enrollment)
• Patients enrolling on the standard risk arms must be medically fit to receive protocol
treatment and with no contraindications to protocol treatment
• Eligibility criteria to participate in the pilot study of the AYA-Hears instrument
(patient reported outcomes [PROs] of ototoxicity) Note: participants in group 1 will
not receive AGCT1531 protocol-directed therapy; all other AYA-HEARS patients must be
enrolled on the AGCT1531 SR2 arm in order to participate
• >= 11 and < 25 years old at enrollment
• Able to fluently speak and read English
• Has received prior cisplatin- or carboplatin-based chemotherapy regimen for malignancy
including diagnoses other than germ cell tumor
• Followed for cancer or survivorship care at one of the following institutions:
• Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
• Dana Farber/Harvard Cancer Center
• Hospital for Sick Children
• Children's Hospital of Eastern Ontario
• Oregon Health and Science University
• Seattle Children's Hospital
• Yale University
Exclusion Criteria:
• Patients with any diagnoses not listed including:
• Stage I testicular cancer patients who have undergone primary RPLND
(retroperitoneal lymph node dissection)
• Pure dysgerminoma
• Pure mature teratoma
• Pure immature teratoma COG stage I, grade I
• Pure immature teratoma COG stage I, grade 2,3 with alpha-fetoprotein (AFP) >=
1000 ng/mL
• Pure immature teratoma COG stage II •IV or FIGO stage IC to IV
• "Poor risk" GCT (age >= 11 years old and COG stage IV ovarian, COG stage III or
IV EG, or IGCCC intermediate or poor risk testicular), or
• Primary central nervous system (CNS) germ cell tumor
• Germ cell tumor with somatic malignant transformation
• Spermatocytic seminoma
• Patients must have had no prior systemic therapy for the current cancer diagnosis
• Patients must have had no prior radiation therapy with the exception of CNS
irradiation of brain metastases; (this exception only applies to SR1 patients; any
patients over age 11 with distant metastases to brain [stage IV disease] would be
considered poor risk and therefore not eligible for this trial)
• Patients with significant, pre-existing co-morbid respiratory disease that
contraindicate the use of bleomycin are ineligible for the standard risk arms of the
trial
• Female patients who are pregnant since fetal toxicities and teratogenic effects have
been noted for several of the study drugs; a pregnancy test is required for female
patients of childbearing potential; (this criteria applies ONLY to patients who will
receive chemotherapy [SR1 and SR2 patients])
• Lactating females who plan to breastfeed their infants; (this criteria applies ONLY to
patients who will receive chemotherapy [SR1 and SR2 patients])
• Sexually active patients of reproductive potential who have not agreed to use an
effective contraceptive method for the duration of their study participation; (this
criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2
patients])
Childhood Extracranial Germ Cell Tumor, Extragonadal Embryonal Carcinoma, Germ Cell Tumor, Malignant Germ Cell Tumor, Malignant Ovarian Teratoma, Stage I Ovarian Choriocarcinoma, Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage I Ovarian Teratoma AJCC v6 and v7, Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage I Testicular Choriocarcinoma AJCC v6 and v7, Stage I Testicular Embryonal Carcinoma AJCC v6 and v7, Stage I Testicular Seminoma AJCC v6 and v7, Stage I Testicular Yolk Sac Tumor AJCC v6 and v7, Stage II Ovarian Choriocarcinoma, Stage II Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage II Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage II Testicular Choriocarcinoma AJCC v6 and v7, Stage II Testicular Embryonal Carcinoma AJCC v6 and v7, Stage II Testicular Yolk Sac Tumor AJCC v6 and v7, Stage III Ovarian Choriocarcinoma, Stage III Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage III Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage III Testicular Choriocarcinoma AJCC v6 and v7, Stage III Testicular Embryonal Carcinoma AJCC v6 and v7, Stage III Testicular Yolk Sac Tumor AJCC v6 and v7, Stage IV Ovarian Choriocarcinoma, Stage IV Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage IV Ovarian Yolk Sac Tumor AJCC v6 and v7, Testicular Mixed Choriocarcinoma and Embryonal Carcinoma, Testicular Mixed Choriocarcinoma and Teratoma, Testicular Mixed Choriocarcinoma and Yolk Sac Tumor, Other Female Genital, Other Male Genital, Ovary
Targeted Therapy in Pediatric Patients with Advanced Solid Tumors (APEC1621SC) (MATCH)
To utilize clinical and biological data to screen for eligibility to phase 2 pathway-targeting specific subprotocols of pathway-targeting agents in pediatric patients with advanced solid tumors, non-Hodgkin lymphomas, and histiocytic disorders.
To determine the proportion of pediatric patients whose advanced tumors have pathway alterations that can be targeted by select anti-cancer drugs.
To determine the objective response rates (ORR; complete response + partial response) in pediatric patients with advanced solid tumors, non-Hodgkin lymphomas, and histiocytic disorders harboring a priori specified genomic alterations treated with pathway-targeting agents.
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Massey, Gita, V.
All
1 year(s) to 21 year(s) old
II
NCT03155620
HM20012289
Show full eligibility criteria
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Inclusion Criteria:
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients must be >= 12 months and
=< 21 years of age at the time of study enrollment
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients with recurrent or
refractory solid tumors, including non-Hodgkin lymphomas, histiocytoses (e.g.
langerhans cell histiocytosis [LCH], juvenile xanthogranuloma [JXG], histiocytic
sarcoma), and central nervous system (CNS) tumors are eligible; patients must have had
histologic verification of malignancy at original diagnosis or relapse except in
patients with intrinsic brain stem tumors, optic pathway gliomas, or patients with
pineal tumors and elevations of cerebrospinal fluid (CSF) or serum tumor markers
including alpha-fetoprotein or beta-human chorionic gonadotropin (HCG); in cases where
patient enrolls prior to histologic confirmation of recurrent disease, patient is
ineligible and should be withdrawn from study if histology fails to confirm
recurrence; please note: Patients with Hodgkin lymphoma and plexiform neurofibroma are
not eligible
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Tumor Testing Requirement: Tumor
sample availability requirement for stage 1 of Pediatric MATCH (patients enrolled from
start of study in July 2017 through 12/31/21); Patients must have an formalin-fixed
paraffin-embedded (FFPE) tumor sample available for MATCH study testing from a biopsy
or surgery that was performed at any point after initial tumor recurrence/progression,
or be planned to have a procedure to obtain such a sample that is considered to be of
potential benefit by the treating clinicians; a tumor sample from a clinically
performed diagnostic (pre-treatment) biopsy will be acceptable for enrollment onto
Pediatric MATCH only for children with high-grade gliomas of the brainstem (diffuse
intrinsic pontine gliomas) or thalamus
• Please note: Samples that have been decalcified using standardly utilized
acid-based decalcification methods are not generally suitable for MATCH study
testing; the nucleic acids will have been degraded in the decalcification process
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Tumor molecular profiling report
availability requirement for Stage 2 of Pediatric MATCH (patients enrolled starting
2022): In stage 2 of the study, no tumor samples will be submitted for centralized
clinical tumor profiling; instead, a tumor molecular profiling report from a College
of American Pathologists (CAP)/ Clinical Laboratory Improvements Amendments
(CLIA)-approved testing laboratory must be submitted for review by the Molecular
Review Committee (MRC)
• This molecular profiling must have been performed on a tumor sample that was
obtained at any point after initial tumor recurrence/progression and must be
accompanied by a pathology report for the same tumor specimen; a molecular
profiling report for a diagnostic (pre-treatment) tumor sample will be acceptable
for enrollment onto Pediatric MATCH only for children with high-grade gliomas of
the brainstem (diffuse intrinsic pontine gliomas) or thalamus. In the event that
molecular profiling reports are available from multiple timepoints, the most
recent report should be prioritized for study submission
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Karnofsky >= 50% for patients >
16 years of age and Lansky >= 50 for patients =< 16 years of age); note: neurologic
deficits in patients with central nervous system (CNS) tumors must have been stable
for at least 7 days prior to study enrollment; patients who are unable to walk because
of paralysis, but who are up in a wheelchair, will be considered ambulatory for the
purpose of assessing the performance score
• ELIGIBILITY CRITERIA FOR ENROLLMENT ONTO APEC1621SC: Patients must have
radiographically measurable disease; measurable disease based on imaging obtained less
than or equal to 56 days prior to enrollment; patients with neuroblastoma who do not
have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable
disease are eligible; measurable disease in patients with CNS involvement is defined
as any lesion that is at minimum 10 mm in one dimension on standard magnetic resonance
imaging (MRI) or computed tomography (CT)
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or
positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or CSF
• Previously radiated lesions that have not demonstrated clear progression
post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for
Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: NOTE: patient does not need to meet all
subprotocol criteria at time of enrollment onto the APEC1621SC screening protocol, but
will need to meet all criteria prior to enrollment on any assigned treatment
subprotocol. Patients must be enrolled onto a subprotocol within 2 weeks (14 days) of
treatment assignment
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Karnofsky >= 50% for patients > 16 years
of age and Lansky >= 50 for patients =< 16 years of age); Note: neurologic deficits in
patients with CNS tumors must have been stable for at least 7 days prior to study
enrollment; patients who are unable to walk because of paralysis, but who are up in a
wheelchair, will be considered ambulatory for the purpose of assessing the performance
score
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: At the time of treatment with subprotocol
specified therapy, the patients must have radiographically measurable disease;
patients with neuroblastoma who do not have measurable disease but have MIBG+
evaluable are eligible; measurable disease in patients with CNS involvement is defined
as any lesion that is at minimum 10 mm in one dimension on standard MRI or CT
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or
positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or CSF
• Previously radiated lesions that have not demonstrated clear progression
post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for
RECIST 1.1
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: At the time of enrollment onto a
subprotocol, the following general criteria for initiation of therapy will be
required:
• Patients must have fully recovered from the acute toxic effects of all prior
anticancer therapy and must meet the following minimum duration from prior
anticancer directed therapy prior to enrollment to the subprotocol; if after the
required timeframe, the numerical eligibility criteria are met, e.g. blood count
criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anticancer agents known to be
myelosuppressive: for agents not listed, the duration of this interval must
be discussed with the study chair and the study-assigned research
coordinator prior to enrollment >= 21 days after the last dose of cytotoxic
or myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anticancer agents not known to be myelosuppressive (e.g. not associated with
reduced platelet or absolute neutrophil counts [ANC]): >= 7 days after the
last dose of agent; for agents not listed, the duration of this interval
must be discussed with the study chair and the study-assigned research
coordinator prior to enrollment
• Antibodies: >= 21 days must have elapsed from infusion of last dose of
antibody, and toxicity related to prior antibody therapy must be recovered
to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior
therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a
long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth
factor; for agents that have known adverse events occurring beyond 7 days
after administration, this period must be extended beyond the time during
which adverse events are known to occur; the duration of this interval must
be discussed with the study chair and the study-assigned research
coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth
factors): >= 21 days after the completion of interleukins, interferon or
cytokines (other than hematopoietic growth factors)
• Stem cell infusions (with or without total-body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any
stem cell infusion including donor lymphocyte infusion (DLI) or boost
infusion: >= 84 days after infusion and no evidence of graft versus
host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular
therapy (e.g. modified T cells, natural killer (NK) cells, dendritic cells,
etc.)
• X-ray therapy (XRT)/External Beam Irradiation including Protons: >= 14 days
after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to
>= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM)
radiation; note: radiation may not be delivered to "measurable disease"
tumor site(s) being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42
days after systemically administered radiopharmaceutical therapy
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: For patients with solid tumors without
known bone marrow involvement:
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving
platelet transfusions for at least 7 days prior to enrollment)
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Patients with known bone marrow
metastatic disease will be eligible for study provided they meet the blood counts (may
receive transfusions provided they are not known to be refractory to red cell or
platelet transfusions); these patients will not be evaluable for hematologic toxicity
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Creatinine clearance or radioisotope
glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2 or a serum creatinine based on
age/gender as follows:
• Age: 1 to < 2 years; maximum serum creatinine (mg/dL): male 0.6; female 0.6
• Age: 2 to < 6 years; maximum serum creatinine (mg/dL): male 0.8; female 0.8
• Age: 6 to < 10 years; maximum serum creatinine (mg/dL): male 1; female 1
• Age: 10 to < 13 years; maximum serum creatinine (mg/dL): male 1.2; female 1.2
• Age: 13 to < 16 years; maximum serum creatinine (mg/dL): male 1.5; female 1.4
• Age: >= 16 years; maximum serum creatinine (mg/dL): male 1.7; female 1.4
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Bilirubin (sum of conjugated +
unconjugated) =< 1.5 x upper limit of normal (ULN) for age
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Serum glutamate pyruvate transaminase
(SGPT) (alanine transferase [ALT]) =< 135 U/L (for the purpose of this study, the ULN
for SGPT is 45 U/L)
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Patients must be able to swallow intact
capsules/tablets, unless otherwise specified in the subprotocol to which they are
assigned
• GENERAL INCLUSION CRITERIA FOR SUBPROTOCOLS: Agent specific limitations on prior
therapy will be included with specific treatment subprotocols
Exclusion Criteria:
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Pregnant or breast-feeding women will not
be entered on this study due to risks of fetal and teratogenic adverse events as seen
in animal/human studies, or because there is currently no available information
regarding human fetal or teratogenic toxicities; pregnancy tests must be obtained in
females who are post-menarchal; males or females of reproductive potential may not
participate unless they have agreed to use an effective contraceptive method
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Concomitant medications
• Corticosteroids: at the time of consent and enrollment to regimen specific
subprotocols, patients receiving corticosteroids who have not been on a stable or
decreasing dose of corticosteroid for at least 7 days prior to enrollment to the
subprotocol will not be eligible; if used to modify immune adverse events related
to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Investigational drugs: patients must meet criteria for prior therapy at the time
of consent and enrollment to a subprotocol; other investigational agents may not
be administered to patients while they are receiving study drug as part of a
subprotocol
• Anticancer agents: patients must meet criteria for prior therapy at the time of
consent and enrollment to a subprotocol; other investigational agents may not be
administered to patients while they are receiving study drug as part of a
subprotocol
• Anti-GVHD agents post-transplant: patients who are receiving cyclosporine,
tacrolimus or other agents to prevent graft-versus-host disease post bone marrow
transplant are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Patients who have an uncontrolled
infection are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Patients who have had a prior solid organ
transplant are not eligible
• GENERAL EXCLUSION CRITERIA FOR SUBPROTOCOLS: Additional agent specific criteria will
be included with specific treatment subprotocols
A Longitudinal Observational Study of Patients with NASH
Primary Aims
Establish an understanding of the current natural history of NASH at academic and
community medical centers
- Evaluate NASH treatment regimens being used in clinical practice
- Examine populations underrepresented in phase II-III clinical trials
- Evaluate optimal duration and combination of NASH therapies to achieve clinical response and clinical remission
- Examine liver histology
- Estimate adverse event frequency and severity and describe management practices
- Evaluate the impact of NASH therapies on medical co-morbidities
Secondary Aims
Describe response rates and safety in special populations
- Evaluate drug-drug interactions
- Evaluate health outcomes and durability of clinical response/clinical remission and time to relapse or treatment failure
- Evaluate changes in components of the metabolic syndrome in the setting of NASH response
- Evaluate mortality, cardiovascular events, and malignancies
- Evaluate patient reported outcomes measures
- Evaluate surgeries and hospitalizations
Boyett, Sherry -sherry.boyett@vcuhealth.org
Luketic, Velimir, A
All
2 year(s) and over
N/A
NCT02815891
HM20010841
Show full eligibility criteria
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Inclusion Criteria:
1. Adults and children (age 2 or older) being managed or treated for nonalcoholic fatty
liver disease. Diagnosis is based on the clinical judgement of the care provider.
Exclusion Criteria:
1. Inability to provide informed assent/consent.
Nonalcoholic Fatty Liver, Nonalcoholic Steatohepatitis, Diseases of Digestive System (520-579)
Phase 2 subprotocol of Ensartinib in patients with tumors harboring ALK or ROS1 genomic alterations (MATCH F)
To determine the objective response rate (ORR, complete response + partial response) in pediatric patients treated with ensartinib with advanced solid tumors (including CNS tumors), non-Hodgkin lymphomas or histiocytic disorders that harbor ALK or ROSI fusions or that harbor ALK missense Mutations
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Massey, Gita, V.
All
1 year(s) to 21 year(s) old
II
NCT03213652
HM20012289F
Show full eligibility criteria
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Inclusion Criteria:
• Patient must have enrolled onto APEC1621SC and must have been given a treatment
assignment to Molecular Analysis for Therapy Choice (MATCH) to APEC1621F based on the
presence of an actionable mutation
• Patients must be >= than 12 months and =< 21 years of age at the time of study
enrollment.
• Patients must have a body surface area >= 0.5 m^2 at enrollment
• Patients must have radiographically measurable disease at the time of study
enrollment. Patients with neuroblastoma who do not have measurable disease but have
iobenguane (MIBG) positive (+) evaluable disease are eligible; measurable disease in
patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one
dimension on a standard MRI or CT
• Note: The following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or
positron emission tomography [PET] scans) except as noted for neuroblastoma
• Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
• Previously radiated lesions that have not demonstrated clear progression
post radiation
• Leptomeningeal lesions that do not meet the measurement requirements for
Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
• Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16
years of age
• Note: Neurologic deficits in patients with CNS tumors must have been relatively
stable for at least 7 days prior to study enrollment; patients who are unable to
walk because of paralysis, but who are up in a wheelchair, will be considered
ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior
anti-cancer therapy and must meet the following minimum duration from prior
anti-cancer directed therapy prior to enrollment; if after the required timeframe, the
numerical eligibility criteria are met, e.g. blood count criteria, the patient is
considered to have recovered adequately
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive:
>= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42
days if prior nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (e.g. not associated with
reduced platelet or absolute neutrophil counts [ANC] counts): >= 7 days after the
last dose of agent
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody,
and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: if used to modify immune adverse events related to prior
therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting
growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor; for
growth factors that have known adverse events occurring beyond 7 days after
administration, this period must be extended beyond the time during which adverse
events are known to occur; the duration of this interval must be discussed with
the study chair and the study-assigned research coordinator
• Interleukins, interferons and cytokines (other than hematopoietic growth
factors): >= 21 days after the completion of interleukins, interferon or
cytokines (other than hematopoietic growth factors)
• Stem cell Infusions (with or without total body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem
cell infusion including donor lymphocyte infusion (DLI) or boost infusion:
>= 84 days after infusion and no evidence of graft versus host disease
(GVHD)
• Autologous stem cell infusion including boost infusion: >= 42 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy
(e.g. modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days
after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >=
50% of the pelvis; >= 42 days if other substantial none marrow (BM) radiation
• Note: Radiation may not be delivered to "measurable disease" tumor site(s)
being used to follow response to subprotocol treatment
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-MIBG): >= 42 days
after systemically administered radiopharmaceutical therapy
• Patients must not have received prior exposure to ensartinib; prior treatment
with other ALK inhibitors is permitted given that at least 5 half-lives or 21
days have elapsed since therapy discontinuation, whichever is greater
• For patients with solid tumors without known bone marrow involvement:
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3 (within 7 days prior to
enrollment)
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving
platelet transfusions for at least 7 days prior to enrollment) (within 7 days
prior to enrollment)
• Patients with known bone marrow metastatic disease will be eligible for study provided
they meet the blood counts (may receive transfusions provided they are not known to be
refractory to red cell or platelet transfusions); these patients will not be evaluable
for hematologic toxicity
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
ml/min/1.73 m^2 (within 7 days prior to enrollment) or a serum creatinine based on
age/gender as follows (within 7 days prior to enrollment):
• Age 1 to < 2 years: maximum serum creatinine 0.6 mg/dL for male and 0.6 mg/dL for
female
• Age 2 to < 6 years: maximum serum creatinine 0.8 mg/dL for male and 0.8 mg/dL for
female
• Age 6 to < 10 years: maximum serum creatinine 1 mg/dL for male and 1 mg/dL for
female
• Age 10 to < 13 years: maximum serum creatinine 1.2 mg/dL for male and 1.2 mg/dL
for female
• Age 13 to < 16 years: maximum serum creatinine 1.5 mg/dL for male and 1.4 mg/dL
for female
• Age >= 16 years: maximum serum creatinine 1.7 mg/dL for male and 1.4 mg/dL for
female
• Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for
age (within 7 days prior to enrollment)
• Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L
(within 7 days prior to enrollment) (for the purpose of this study, the ULN for SGPT
is 45 U/L)
• Serum albumin >= 2 g/dL (within 7 days prior to enrollment)
• Patients must be able to swallow intact capsules
• All patients and/or their parents or legally authorized representatives must sign a
written informed consent; assent, when appropriate, will be obtained according to
institutional guidelines
Exclusion Criteria:
• Pregnant or breast-feeding women will not be entered on this study because there is
currently no available information regarding human fetal or teratogenic toxicities;
pregnancy tests must be obtained in girls who are post-menarchal; males or females of
reproductive potential may not participate unless they have agreed to use an effective
contraceptive method for the duration of study treatment and for one week after the
last dose of ensartinib
• Concomitant medications
• Corticosteroids: patients receiving corticosteroids who have not been on a stable
or decreasing dose of corticosteroid for at least 7 days prior to enrollment are
not eligible; if used to modify immune adverse events related to prior therapy,
>= 14 days must have elapsed since last dose of corticosteroid
• Investigational drugs: patients who are currently receiving another
investigational drug are not eligible
• Anti-cancer agents: patients who are currently receiving other anti-cancer agents
are not eligible
• Anti-GVHD agents post-transplant: patients who are receiving cyclosporine,
tacrolimus or other agents to prevent graft-versus-host disease post bone marrow
transplant are not eligible for this trial
• CYP3A4 agents: patients who are currently receiving drugs that are strong
inducers or strong inhibitors of CYP3A4 are not eligible; strong inducers or
inhibitors of CYP3A4 should be avoided from 14 days prior to enrollment to the
end of the study
• Note: CYP3A4 inducing anti-epileptic drugs and dexamethasone for CNS tumors
or metastases, on a stable dose, are allowed
• Patients who have an uncontrolled infection are not eligible
• Patients who have received a prior solid organ transplantation are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the
safety monitoring requirements of the study are not eligible
Primary Objectives:
1. To elucidate, through the cooperative effort of a multidisciplinary and multicenter group of collaborators, the etiology, natural history, diagnosis, treatment, and prevention of NAFLD, and in particular its more severe form of NASH and its complications
2. To enroll at least 2250 patients (1500 adult patients and 750 pediatric patients) with a diagnosis of NAFLD, supported by a standard of care liver biopsy, with a broad range of severity. Core data collection will include clinical, demographic, laboratory, imaging, and histological features
3. To increase the population diversity of the NAFLD Database 2 to provide greater representation of Hispanic, Native American, African American, and Asian patients recruited into the NAFLD Database 3
4. To expand the current specimen bank comprised of liver tissue, serum, plasma, and DNA obtained from patients undergoing a liver biopsy with the specific goal of optimizing the collection of plasma or serum suitable for biomarker development studies by obtaining specimens in close temporal proximity to the performance of liver biopsy
Secondary Objectives:
1. To continue the analysis of the data obtained in the NAFLD Database 2 study.
2. To add to current NASH CRN resources for developing clinical and pathological criteria for standardizing diagnostic and staging criteria for NAFLD or NASH-related cirrhosis
3. To add to current NASH CRN resources for developing clinical and pathological criteria and measures and endpoints for therapeutic studies of NAFLD or NASH-related cirrhosis
4. To evaluate the utility of FibroScan® as a diagnostic modality for the non-invasive staging and grading of NAFLD
5. To add to current NASH CRN resources for ancillary studies of the pathogenesis, diagnosis or diagnostic biomarker development, genomic, proteomic and lipidomic characterization, natural history and treatment of NAFLD or NASH-related cirrhosis
Draper, Anna Kate -anna.draper@vcuhealth.org
Sanyal, Arun, J
All
2 year(s) and over
N/A
NCT04454463
HM20019928
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Inclusion Criteria:
• 2 years of age or older as of the initial screening interview and provision of consent
• Willingness to participate in the study for 1 or more years
• Histologic evidence of NAFLD or NASH based upon a standard of care liver biopsy
• Collection of serum and plasma up to 90 days before or 4- 90 days after standard of
care liver biopsy
• Absence of regular or excessive use of alcohol within 2 years prior to initial
screening
Exclusion Criteria:
• Clinical or histological evidence of alcoholic liver disease: Regular and excessive
use of alcohol within the 2 years prior to interview defined as alcohol intake greater
than 14 drinks per week in a man or greater than 7 drinks per week in a woman.
Approximately 10 g of alcohol equals one 'drink' unit. One unit equals 1 ounce of
distilled spirits, one 12-oz beer, or one 4-oz glass of wine
• Total parenteral nutrition for more than 1 month within a 6-month period before
baseline liver biopsy
• Short bowel syndrome
• History of gastric or jejunoileal bypass preceding the diagnosis of NAFLD. Bariatric
surgery performed following enrollment is not exclusionary. Liver biopsies obtained
during bariatric surgery cannot be used for enrollment because of the associated
surgical or anesthetic acute changes and the weight loss efforts that precede
bariatric surgery
• History of biliopancreatic diversion
• Evidence of advanced liver disease defined as a Child-Pugh-Turcotte score equal to or
greater than 10
• Evidence of chronic hepatitis B as marked by the presence of HBsAg in serum (patients
with isolated antibody to hepatitis B core antigen, anti-HBc total, are not excluded)
• Evidence of chronic hepatitis C as marked by the presence of anti-HCV or HCV RNA in
serum
• Low alpha-1-antitrypsin level and ZZ phenotype (both determined at the discretion of
the investigator)
• Wilson's disease
• Known glycogen storage disease
• Known dysbetalipoproteinemia
• Known phenotypic hemochromatosis (HII greater than 1.9 or removal of more than 4 g of
iron by phlebotomy)
• Prominent bile duct injury (florid duct lesions or periductal sclerosis) or bile duct
paucity
• Chronic cholestasis
• Vascular lesions (vasculitis, cardiac sclerosis, acute or chronic Budd-Chiari,
hepatoportal sclerosis, peliosis)
• Iron overload greater than 3+
• Zones of confluent necrosis, infarction, massive or sub-massive, pan-acinar necrosis
• Multiple epithelioid granulomas
• Congenital hepatic fibrosis
• Polycystic liver disease
• Other metabolic or congenital liver disease
• Evidence of systemic infectious disease
• Known HIV positive
• Disseminated or advanced malignancy
• Concomitant severe underlying systemic illness that in the opinion of the investigator
would interfere with completion of follow-up
• Active drug use or dependence that, in the opinion of the study investigator, would
interfere with adherence to study requirements
• Any other condition, which in the opinion of the investigator would impede compliance
or hinder completion of study
• Inability to complete the appropriate informed consent process
Liver Diseases, Diseases of Digestive System (520-579)
The overall objective of this study is to perform baseline and repeat assessments over time of the metabolic and immunologic status of individuals at risk for type 1 diabetes (T1D) to (a) characterize their risk for developing T1D and identify subjects eligible for prevention trials, (b) describe the pathogenic evolution of T1D and (c) increase the understanding of the pathogenic factors involved in the development of T1D.
Memoli, Erica, H -erica.memoli@vcuhealth.org
Nelson, Bryce
No gender available
Not specified
N/A
NCT00097292
HM20012389
Endocrine, Nutritional And Metabolic Diseases, And Immunity Disorders, Endocrine, Nutritional, Metabolic Diseases, Immunity Disorders (240-279)
• Pediatric patients undergoing GA for dental restorative work at Virginia Commonwealth
University Children's Hospital of Richmond Pavilion and Brook Road Centers
Exclusion Criteria:
• Children with any sensory or behavioral issues
• Patients requiring extractions or surgical procedures
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Primary: To collect long-term follow-up safety and efficacy data of in patients with spinal muscular atrophy (SMA) Type 1, Type 2, or Type 3 who were treated with AVXS-101 in any AVXS-101 clinical trial conducted at a United States (US) site, including (but not limited to) AVXS-101-CL-102 (Phase 1), AVXS-101-CL-303 (Phase 3), AVXS-101-CL-304 (Phase 3), or AVXS-101-CL-305 (Phase 3).
In addition; North American patients treated at ex-North American sites in AVXS-101-CL-302
(European Phase 3), AVXS-101-CL-304 (Phase 3), or at ex-North American sites in other
AVXS-101 studies who wish to engage in long-term follow-up in North America may be enrolled.
Qureshi, Mehreen -mehreen.qureshi@vcuhealth.org
Harper, Amy
All
Not specified
IV
NCT04042025
HM20017313
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Inclusion Criteria:
• Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement
therapy in a Novartis Gene Therapies-sponsored clinical study
• Participant/parent/legal guardian willing and able to complete the informed consent
process and comply with study procedures and visit schedule
Exclusion Criteria:
• Parent/legal guardian unable or unwilling to participate in the long-term follow-up
safety study
Biological: Onasemnogene Abeparvovec-xioi
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA, Diseases of Nervous System and Sense Organs (320-389)
The Safety and Efficacy of Alpha-1 Antitrypsin (AAT) for the Prevention of Graft-Versus-host Disease (GVHD) in Patients Receiving Hematopoietic Cell Transplant (MODULAATE)
ctrrecruit@vcu.edu
All
12 year(s) and over
Phase 2/Phase 3
NCT03805789
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Inclusion Criteria:
• Male or female subjects, ?12 years of age (? 18 years of age for subjects at German
sites only), undergoing HCT for hematological malignancies, including leukemia,
lymphoma, multiple myeloma, myelodysplastic syndrome and myeloproliferative neoplasms
• Planned myeloablative conditioning regimen
Exclusion Criteria:
• Prior autologous or allogeneic HCT
• T-cell depleted transplant or planned use of anti-T cell antibody therapy either ex
vivo or in vivo (ie, anti-thymocyte globulin [ATG], alemtuzumab) for GVHD prophylaxis
• Planned umbilical cord blood (UCB) transplant
Study of ERAS in Patients Undergoing Urologic Reconstructive Surgery (ERAS)
The pilot portion of this study will be used to determine the overall success in ERAS protocol processes and implementation at each center. If successful, study centers will transition to the larger, exploration phase of the study which will include comparison to matched historical con-trols.
Welch, Valre -valre.welch@vcuhealth.org
Herndon, Claude
All
4 year(s) to 25 year(s) old
Pilot
NCT03245242
HM20015891
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Inclusion criteria:
• Age greater than or equal to 4 or <26 years at time of surgery
• Undergoing urologic reconstructive surgery that includes primary bowel anastomosis
(i.e., creation of continent ileovesicostomy [Monti], sigmoid Monti channel,
enterocystoplasty)
• Undergoing urologic reconstructive surgery that includes possible bowel anastomosis
(planned creation of continent appendicovesicostomy [Mitrofanoff] with inability to
use appendix at time of surgery and creation of alternative channel requiring primary
bowel anastomosis)
Exclusion criteria:
• Clinically constipated (defined as Bristol 1 or 2 stools more than once per week, bowel
movement interval > every other day [e.g. only has bowel movement every 3 days, or palpable
stool in > 50% of colon on physical preoperative exam)
Other: Enhanced Recovery after Surgery, Other: Historical usual surgical care
Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
To demonstrate that the efficacy of treatment with selumetinib as measured by event-free survival (EFS) is non-inferior compared to treatment with carboplatin/vincristine (CV) in previously-untreated low-grade glioma (LGG) not associated with BRAFV600E mutations or systemic neurofibromatosis type 1 (NF1).
Gwaltney, Lindsey -lbgwaltney@vcu.edu
Massey, Gita, V.
All
2 year(s) to 21 year(s) old
III
NCT04166409
HM20020781
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Inclusion Criteria:
• Patients must be >= 2 years and =< 21 years at the time of enrollment
• Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment
• Patients must have non-neurofibromatosis type 1 (non-NF1) low-grade glioma (LGG)
without a BRAFV600E mutation as confirmed by Rapid Central Pathology and Molecular
Screening Reviews performed on APEC14B1 (NCT02402244) and that has not been treated
with any modality besides surgery. Note: Patients may be newly-diagnosed OR previously
diagnosed, and there is no required time frame between biopsy/surgery and treatment
initiation.
• Patients with residual tumor after resection or progressive tumor after initial
diagnosis (with or without surgery) who have not received treatment (chemotherapy
and/or radiation) are eligible
• Patients must have two-dimensional measurable tumor >= 1 cm^2 to be eligible
• Eligible histologies will include all tumors considered low-grade glioma or low-grade
astrocytoma (World Health Organization [WHO] grade I and II) by 5th edition WHO
classification of central nervous system (CNS) tumors with the exception of
subependymal giant cell astrocytoma
• Patients with metastatic disease or multiple independent primary LGG are eligible
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 OR a serum creatinine based on age/gender as follows (performed within
7 days prior to enrollment):
• Age: Maximum Serum Creatinine (mg/dL)
• 2 to < 6 years: 0.8 mg/dL (male); 0.8 mg/dL (female)
• 6 to < 10 years: 1 mg/dL (male); 1 mg/dL (female)
• 10 to < 13 years: 1.2 mg/dL (male); 1.2 mg/dL (female)
• 13 to < 16 years: 1.5 mg/dL (male); 1.4 mg/dL (female)
• >= 16 years: 1.7 mg/dL (male); 1.4 mg/dL (female)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (performed within 7 days
prior to enrollment) (children with a diagnosis of Gilbert's syndrome will be allowed
on study regardless of their total and indirect [unconjugated] bilirubin levels as
long as their direct [conjugated] bilirubin is < 3.1 mg/dL)
• Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L
(performed within 7 days prior to enrollment). For the purpose of this study, the ULN
for SGPT is 45 U/L
• Albumin >= 2 g/dL (performed within 7 days prior to enrollment)
• Left ventricular ejection fraction (LVEF) >= 53% (or institutional normal; if the LVEF
result is given as a range of values, then the upper value of the range will be used)
by echocardiogram (performed within 4 weeks prior to enrollment)
• Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) (performed within
4 weeks prior to enrollment)
• Absolute neutrophil count >= 1,000/uL (unsupported) (performed within 7 days prior to
enrollment)
• Platelets >= 100,000/uL (unsupported) (performed within 7 days prior to enrollment)
• Hemoglobin >= 8 g/dL (may be supported) (performed within 7 days prior to enrollment)
• Patients with a known seizure disorder should be stable and should not have
experienced a significant increase in seizure frequency within 2 weeks prior to
enrollment
• Patients 2-17 years of age must have a blood pressure that is =< 95th percentile for
age, height, and gender at the time of enrollment (with or without the use of
anti-hypertensive medications)
• Patients >= 18 years of age must have a blood pressure =< 130/80 mmHg at the time of
enrollment (with or without the use of anti-hypertensive medications)
• Note for patients of all ages: Adequate blood pressure can be achieved using
medication for the treatment of hypertension
• All patients must have ophthalmology toxicity assessments performed within 4 weeks
prior to enrollment
• For all patients, a magnetic resonance imaging (MRI) of the brain (with orbital cuts
for optic pathway tumors) and/or spine (depending on the site(s) of primary disease)
with and without contrast must be performed within 4 weeks prior to enrollment
• Patients must have a performance status corresponding to Eastern Cooperative Oncology
Group (ECOG) scores of 0, 1, or 2. Use Karnofsky for patients > 16 years of age and
Lansky for patients =< 16 years of age
• Patients must have the ability to swallow whole capsules
• All patients have signed an appropriate consent form and Health Insurance Portability
and Accountability Act (HIPAA) authorization form (if applicable)
• All patients and/or their parents or legal guardians must sign a written informed
consent
• All patients have been consented and enrolled on APEC14B1 (NCT02402244) followed by
enrollment on the ACNS1833 Pre-Enrollment Eligibility Screening (Step 0) on the same
day to complete the Rapid Central Review
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met
Exclusion Criteria:
• Patients must not have received any prior tumor-directed therapy including
chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior
surgical intervention is permitted
• Patients with a concurrent malignancy or history of treatment (other than surgery) for
another tumor within the last year are ineligible
• Patients with diffuse intrinsic pontine tumors as seen on MRI (> 2/3 of pons
involvement on imaging) are not eligible even if biopsy reveals grade I/II histology
• Patients may not be receiving any other investigational agents
• Patients with any serious medical or psychiatric illness/condition, including
substance use disorders or ophthalmological conditions, likely in the judgment of the
investigator to interfere or limit compliance with study requirements/treatment
• Patients who, in the opinion of the investigator, are not able to comply with the
study procedures are not eligible
• Female patients who are pregnant are not eligible since fetal toxicities and
teratogenic effects have been noted for several of the study drugs. A pregnancy test
is required for female patients of childbearing potential
• Lactating females who plan to breastfeed their infants are not eligible
• Sexually active patients of reproductive potential who have not agreed to use an
effective contraceptive method for the duration of their study participation and for
12 weeks after stopping study therapy are not eligible.
• Note: Women of child-bearing potential and males with sexual partners who are
pregnant or who could become pregnant (i.e., women of child-bearing potential)
should use effective methods of contraception for the duration of the study and
for 12 weeks after stopping study therapy to avoid pregnancy and/or potential
adverse effects on the developing embryo
• Known genetic disorder that increases risk for coronary artery disease. Note: The
presence of dyslipidemia in a family with a history of myocardial infarction is not in
itself an exclusion unless there is a known genetic disorder documented
• Symptomatic heart failure
• New York Health Association (NYHA) class II-IV prior or current cardiomyopathy
• Severe valvular heart disease
• History of atrial fibrillation
• Current or past history of central serous retinopathy
• Current or past history of retinal vein occlusion or retinal detachment
• Patients with uncontrolled glaucoma
• If checking pressure is clinically indicated, patients with intraocular pressure
(IOP) > 22 mmHg or ULN adjusted by age are not eligible
• Supplementation with vitamin E greater than 100% of the daily recommended dose. Any
multivitamin containing vitamin E must be stopped prior to study enrollment even if
less than 100% of the daily recommended dosing for vitamin E
• Surgery within 2 weeks prior to enrollment, with the exception of surgical biopsy,
placement of a vascular access device or cerebral spinal fluid (CSF) diverting
procedure such as endoscopic third ventriculostomy (ETV) and ventriculoperitoneal (VP)
shunt.
• Note: Patients must have healed from any prior surgery
• Patients who have an uncontrolled infection are not eligible
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
StudyObjectives:TheprimarygoalofMotorOutcomestoValidateEvaluationsinFSHD (MOVE FSHD) is to hasten therapeutic development for FSHD and improve care delivery. This goal will be achieved by creating a standard clinic-based protocol informed by current best practice to determine the predictive value of clinical characteristics and motor assessments on motor and health outcomes (i.e. need for orthotics, ambulation aides, respiratory support, etc.).
Howell, Jodie -jodie.howell@vcuhealth.org
Johnson, Nicholas, E
All
Not specified
N/A
NCT04635891
HM20021534
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Inclusion Criteria:
• Genetically confirmed FSHD (types 1 or 2) or clinical diagnosis of FSHD with
characteristic findings on exam and an affected parent or offspring.
Exclusion Criteria:
• Unwilling or unable to provide informed consent.
• Any other medical condition which in the opinion of the investigator would interfere
with study participation.
FSHD, Diseases of Nervous System and Sense Organs (320-389)