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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03155620




Registration number
NCT03155620
Ethics application status
Date submitted
15/05/2017
Date registered
16/05/2017
Date last updated
16/12/2024

Titles & IDs
Public title
Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)
Scientific title
NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol
Secondary ID [1] 0 0
NCI-2017-01251
Secondary ID [2] 0 0
NCI-2017-01251
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Advanced Malignant Solid Neoplasm 0 0
Ann Arbor Stage III Non-Hodgkin Lymphoma 0 0
Ann Arbor Stage IV Non-Hodgkin Lymphoma 0 0
Histiocytic Sarcoma 0 0
Juvenile Xanthogranuloma 0 0
Langerhans Cell Histiocytosis 0 0
Malignant Glioma 0 0
Recurrent Childhood Rhabdomyosarcoma 0 0
Recurrent Ependymoma 0 0
Recurrent Ewing Sarcoma 0 0
Recurrent Glioma 0 0
Recurrent Hepatoblastoma 0 0
Recurrent Langerhans Cell Histiocytosis 0 0
Recurrent Malignant Germ Cell Tumor 0 0
Recurrent Malignant Solid Neoplasm 0 0
Recurrent Medulloblastoma 0 0
Recurrent Neuroblastoma 0 0
Recurrent Non-Hodgkin Lymphoma 0 0
Recurrent Osteosarcoma 0 0
Recurrent Peripheral Primitive Neuroectodermal Tumor 0 0
Recurrent Primary Central Nervous System Neoplasm 0 0
Recurrent Rhabdoid Tumor 0 0
Recurrent Soft Tissue Sarcoma 0 0
Refractory Ewing Sarcoma 0 0
Refractory Glioma 0 0
Refractory Hepatoblastoma 0 0
Refractory Langerhans Cell Histiocytosis 0 0
Refractory Malignant Germ Cell Tumor 0 0
Refractory Malignant Solid Neoplasm 0 0
Refractory Medulloblastoma 0 0
Refractory Neuroblastoma 0 0
Refractory Non-Hodgkin Lymphoma 0 0
Refractory Osteosarcoma 0 0
Refractory Peripheral Primitive Neuroectodermal Tumor 0 0
Refractory Primary Central Nervous System Neoplasm 0 0
Refractory Rhabdoid Tumor 0 0
Refractory Rhabdomyosarcoma 0 0
Rhabdoid Tumor 0 0
Stage III Osteosarcoma AJCC v7 0 0
Stage III Soft Tissue Sarcoma AJCC v7 0 0
Stage IV Osteosarcoma AJCC v7 0 0
Stage IV Soft Tissue Sarcoma AJCC v7 0 0
Stage IVA Osteosarcoma AJCC v7 0 0
Stage IVB Osteosarcoma AJCC v7 0 0
Wilms Tumor 0 0
Condition category
Condition code
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma
Cancer 0 0 0 0
Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma
Cancer 0 0 0 0
Sarcoma (also see 'Bone') - soft tissue
Cancer 0 0 0 0
Bone
Cancer 0 0 0 0
Brain
Cancer 0 0 0 0
Neuroendocrine tumour (NET)
Cancer 0 0 0 0
Children's - Other
Cancer 0 0 0 0
Ovarian and primary peritoneal
Cancer 0 0 0 0
Testicular
Cancer 0 0 0 0
Other cancer types
Cancer 0 0 0 0
Children's - Brain
Cancer 0 0 0 0
Kidney
Cancer 0 0 0 0
Liver
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Skin 0 0 0 0
Other skin conditions
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Surgery - Biopsy
Treatment: Surgery - Biospecimen Collection
Treatment: Surgery - Bone Marrow Aspiration and Biopsy
Treatment: Surgery - Bone Scan
Treatment: Surgery - Computed Tomography
Treatment: Drugs - Ensartinib
Treatment: Drugs - Erdafitinib
Other interventions - Laboratory Biomarker Analysis
Treatment: Drugs - Larotrectinib Sulfate
Treatment: Surgery - Magnetic Resonance Imaging
Treatment: Surgery - Mutation Carrier Screening
Treatment: Drugs - Olaparib
Treatment: Drugs - Palbociclib
Other interventions - Pharmacological Study
Treatment: Surgery - Positron Emission Tomography
Treatment: Surgery - Radionuclide Imaging
Treatment: Drugs - Samotolisib
Treatment: Drugs - Selpercatinib
Treatment: Drugs - Selumetinib Sulfate
Treatment: Drugs - Tazemetostat
Treatment: Drugs - Tipifarnib
Treatment: Drugs - Ulixertinib
Treatment: Drugs - Vemurafenib
Treatment: Surgery - X-Ray Imaging

Experimental: Subprotcol M (HRAS gene alterations) - Patients receive tipifarnib PO or via nasogastric or gastric tube BID on days 1-7 and 15-21. Treatment repeats every 28 days for up to 26 cycles (2 years) in the absence of disease progression or unacceptable toxicity.

Experimental: Subprotocol A (NTRK1, NTRK2, or NTRK3 gene fusion) - Patients with a NTRK1, NTRK2, or NTRK3 gene fusion receive larotrectinib sulfate PO or via nasogastric- or gastric-tube BID on days 1-28. Cycles repeat every 28 days for 2 years in the absence of disease progression or unacceptable toxicity.

Experimental: Subprotocol B (FGFR1, FGFR2, FGFR3, or FGFR4 gene mutation) - Patients with a FGFR1, FGFR2, FGFR3, or FGFR4 gene mutation receive erdafitinib PO QD on days 1-28 of each cycle. Treatment repeats every 28 days for up to 26 cycles (2 years) in the absence of disease progression or unacceptable toxicity. Patients undergo an x-ray, CT scan, MRI, radionuclide imaging, and/or bone scan, as well as a bone marrow aspiration and/or biopsy during screening and on study. Patients also undergo blood sample collection on study.

Experimental: Subprotocol C (EZH2, SMARCB1, or SMARCA4 gene mutation) - Patients with an EZH2, SMARCB1, or SMARCA4 gene mutation receive tazemetostat PO BID on days 1-28. Cycles repeat every 28 days for 2 years in the absence of disease progression or unacceptable toxicity.

Experimental: Subprotocol D (TSC1, TSC2, or PI3K/mTOR gene mutation) - Patients with a TSC1, TSC2, or PI3K/mTOR gene mutations receive PI3K/mTOR inhibitor LY3023414 PO BID on days 1-28. Cycles repeat every 28 days for 2 years in the absence of disease progression or unacceptable toxicity.

Experimental: Subprotocol E (activating MAPK pathway gene mutation) - Patients with an activating MAPK pathway gene mutation receive selumetinib sulfate PO BID on days 1-28. Cycles repeat every 28 days for 2 years in the absence of disease progression or unacceptable toxicity.

Experimental: Subprotocol F (ALK or ROS1 gene alteration) - Patients with an ALK or ROS1 gene alteration receive ensartinib PO BID on days 1-28. Cycles repeat every 28 days for 2 years (up to 26 cycles) in the absence of disease progression or unacceptable toxicity. Patients undergo an x-ray, CT scan, MRI, PET scan, radionuclide imaging, and/or bone scan, as well as a bone marrow aspiration and/or biopsy during screening and on study. Patients also undergo blood sample collection on study.

Experimental: Subprotocol G (BRAF V600 gene mutation) - Patients with a BRAF V600 gene mutation receive vemurafenib PO BID on days 1-28. Cycles repeat every 28 days for 2 years in the absence of disease progression or unacceptable toxicity.

Experimental: Subprotocol H (ATM, BRCA1, BRCA2, RAD51C, RAD51D mutations) - Patients deleterious ATM, BRCA1, BRCA2, RAD51C, or RAD51D gene mutations receive olaparib PO BID on days 1-28. Cycles repeat every 28 days for 2 years in the absence of disease progression or unacceptable toxicity.

Experimental: Subprotocol I (Rb positive, alterations in cell cycle genes) - Patients with Rb positive advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with activating alterations in cell cycle genes receive palbociclib PO QD on days 1-21. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.

Experimental: Subprotocol J (MAPK pathway mutations) - Patients with MAPK pathway mutations receive ulixertinib PO BID. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.

Experimental: Subprotocol N (activating RET mutations) - Patients with activating RET gene alterations receive selpercatinib PO BID on days 1-28. Treatment repeats every 28 days for up to 26 cycles (2 years) in the absence of disease progression or unacceptable toxicity. Patients may also undergo PET, CT, MRI, PET/CT, PET/MRI, and/or CT/MRI, scintigraphy, and x-ray imaging throughout the trial.


Treatment: Surgery: Biopsy
Undergo biopsy

Treatment: Surgery: Biospecimen Collection
Undergo blood sample collection

Treatment: Surgery: Bone Marrow Aspiration and Biopsy
Undergo a bone marrow and/or biopsy

Treatment: Surgery: Bone Scan
Undergo a bone scan

Treatment: Surgery: Computed Tomography
Undergo CT, PET/Ct, and/or CT/MRI

Treatment: Drugs: Ensartinib
Given PO

Treatment: Drugs: Erdafitinib
Given PO

Other interventions: Laboratory Biomarker Analysis
Undergo molecular analysis

Treatment: Drugs: Larotrectinib Sulfate
Given PO or via nasogastric- or gastric-tube

Treatment: Surgery: Magnetic Resonance Imaging
Undergo MRI, PET/MRI, and/or CT/MRI

Treatment: Surgery: Mutation Carrier Screening
Undergo tumor tissue mutation screening

Treatment: Drugs: Olaparib
Given PO

Treatment: Drugs: Palbociclib
Given PO

Other interventions: Pharmacological Study
Correlative studies

Treatment: Surgery: Positron Emission Tomography
Undergo PET, PET/CT, and/or PET/MRI

Treatment: Surgery: Radionuclide Imaging
Undergo radionuclide imaging

Treatment: Drugs: Samotolisib
Given PO

Treatment: Drugs: Selpercatinib
Given PO

Treatment: Drugs: Selumetinib Sulfate
Given PO

Treatment: Drugs: Tazemetostat
Given PO

Treatment: Drugs: Tipifarnib
Given PO or via nasogastric or gastric tube

Treatment: Drugs: Ulixertinib
Receive PO

Treatment: Drugs: Vemurafenib
Given PO

Treatment: Surgery: X-Ray Imaging
Undergo an x-ray
Intervention code [1] 0 0
Treatment: Surgery
Intervention code [2] 0 0
Treatment: Drugs
Intervention code [3] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Objective response rate (complete response/partial response)
Timepoint [1] 0 0
From enrollment to the end of treatment, up to 2 years on subprotocol
Primary outcome [2] 0 0
Proportion of pediatric patients whose advanced tumors have pathway alterations that can be targeted by select anti-cancer drugs
Timepoint [2] 0 0
Up to 4 years
Secondary outcome [1] 0 0
Percentage of patients with grade 3 or 4 adverse events
Timepoint [1] 0 0
From enrollment to the end of treatment, up to 2 years on subprotocol
Secondary outcome [2] 0 0
Incidence of research biopsy related target toxicity
Timepoint [2] 0 0
Up to 14 days
Secondary outcome [3] 0 0
Progression free survival (PFS)
Timepoint [3] 0 0
From the initiation of protocol treatment to the occurrence of any of the following events: disease progression or disease recurrence or death from any cause, assessed up to 4 years
Secondary outcome [4] 0 0
Pharmacokinetic (PK) parameters
Timepoint [4] 0 0
Up to 4 years

Eligibility
Key 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
Minimum age
12 Months
Maximum age
21 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Other
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/07/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/09/2027
Actual
Sample size
Target
2316
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Perth Children's Hospital - Perth
Recruitment postcode(s) [1] 0 0
6009 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Alaska
Country [3] 0 0
United States of America
State/province [3] 0 0
Arizona
Country [4] 0 0
United States of America
State/province [4] 0 0
Arkansas
Country [5] 0 0
United States of America
State/province [5] 0 0
California
Country [6] 0 0
United States of America
State/province [6] 0 0
Colorado
Country [7] 0 0
United States of America
State/province [7] 0 0
Connecticut
Country [8] 0 0
United States of America
State/province [8] 0 0
Delaware
Country [9] 0 0
United States of America
State/province [9] 0 0
District of Columbia
Country [10] 0 0
United States of America
State/province [10] 0 0
Florida
Country [11] 0 0
United States of America
State/province [11] 0 0
Georgia
Country [12] 0 0
United States of America
State/province [12] 0 0
Hawaii
Country [13] 0 0
United States of America
State/province [13] 0 0
Idaho
Country [14] 0 0
United States of America
State/province [14] 0 0
Illinois
Country [15] 0 0
United States of America
State/province [15] 0 0
Indiana
Country [16] 0 0
United States of America
State/province [16] 0 0
Iowa
Country [17] 0 0
United States of America
State/province [17] 0 0
Kentucky
Country [18] 0 0
United States of America
State/province [18] 0 0
Louisiana
Country [19] 0 0
United States of America
State/province [19] 0 0
Maine
Country [20] 0 0
United States of America
State/province [20] 0 0
Maryland
Country [21] 0 0
United States of America
State/province [21] 0 0
Massachusetts
Country [22] 0 0
United States of America
State/province [22] 0 0
Michigan
Country [23] 0 0
United States of America
State/province [23] 0 0
Minnesota
Country [24] 0 0
United States of America
State/province [24] 0 0
Mississippi
Country [25] 0 0
United States of America
State/province [25] 0 0
Missouri
Country [26] 0 0
United States of America
State/province [26] 0 0
Nebraska
Country [27] 0 0
United States of America
State/province [27] 0 0
Nevada
Country [28] 0 0
United States of America
State/province [28] 0 0
New Hampshire
Country [29] 0 0
United States of America
State/province [29] 0 0
New Jersey
Country [30] 0 0
United States of America
State/province [30] 0 0
New York
Country [31] 0 0
United States of America
State/province [31] 0 0
North Carolina
Country [32] 0 0
United States of America
State/province [32] 0 0
Ohio
Country [33] 0 0
United States of America
State/province [33] 0 0
Oklahoma
Country [34] 0 0
United States of America
State/province [34] 0 0
Oregon
Country [35] 0 0
United States of America
State/province [35] 0 0
Pennsylvania
Country [36] 0 0
United States of America
State/province [36] 0 0
Rhode Island
Country [37] 0 0
United States of America
State/province [37] 0 0
South Carolina
Country [38] 0 0
United States of America
State/province [38] 0 0
South Dakota
Country [39] 0 0
United States of America
State/province [39] 0 0
Tennessee
Country [40] 0 0
United States of America
State/province [40] 0 0
Texas
Country [41] 0 0
United States of America
State/province [41] 0 0
Utah
Country [42] 0 0
United States of America
State/province [42] 0 0
Vermont
Country [43] 0 0
United States of America
State/province [43] 0 0
Virginia
Country [44] 0 0
United States of America
State/province [44] 0 0
Washington
Country [45] 0 0
United States of America
State/province [45] 0 0
West Virginia
Country [46] 0 0
United States of America
State/province [46] 0 0
Wisconsin
Country [47] 0 0
Canada
State/province [47] 0 0
Quebec
Country [48] 0 0
Puerto Rico
State/province [48] 0 0
San Juan

Funding & Sponsors
Primary sponsor type
Government body
Name
National Cancer Institute (NCI)
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Donald W Parsons
Address 0 0
Children's Oncology Group
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT03155620