Clinical trial NCT00602667: Risk-Adapted Therapy for Young Children With Embryonal Brain Tumors, Choroid Plexus Carcinoma, High Grade Glioma or Ependymoma
Official title: Risk-Adapted Therapy for Young Children With Embryonal Brain Tumors, Choroid Plexus Carcinoma, High Grade Glioma or Ependymoma
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Brain and Central Nervous System Tumors
Keywords: untreated childhood medulloblastoma
untreated childhood supratentorial primitive neuroectodermal tumor
untreated childhood pineoblastoma
childhood atypical teratoid/rhabdoid tumor
childhood choroid plexus tumor
childhood high grade glioma
newly diagnosed childhood ependymoma
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
Collaborators: name: University of Florida
name: National Cancer Institute (NCI)
name: The Pew Charitable Trusts
First posted: 2008-01-28
Enrollment: 293
Sex: ALL
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: RATIONALE: In this study a combination of anti-cancer drugs (chemotherapy) is used to treat brain tumors in young children. Using chemotherapy gives the brain more time to develop before radiation is given. The chemotherapy in this study includes the drug methotrexate. This drug was an important part of the two clinical trials which resulted in the best survival results for children less than 3 years of age with medulloblastoma. Most patients treated on this trial will also receive radiation which is carefully targeted to the area of the tumor. This type of radiation (focal conformal or proton beam radiotherapy) may result in fewer problems with thinking and learning than radiation to the whole brain and spinal cord.

PURPOSE: This clinical trial is studying how well giving combination chemotherapy together with radiation therapy works in treating young patients with newly diagnosed central nervous system tumors.

Detailed description: All patients with medulloblastoma who were diagnosed prior to their 3rd birthday will contribute to both the biology and therapeutic primary objectives of this protocol. Furthermore patients who were ≥3 and \<5 years old at the time of diagnosis will also be included in the cohort for these primary objectives as long as they meet the eligibility criteria as outlined in Amendment 8.0 of this protocol. Patients in the 3-5 year old age cohort who enrolled on previous versions of this protocol and who do not meet the criteria as outlined in Amendment 8.0 of this protocol will be excluded from the outcome analyses of the biology and therapeutic primary objectives of the protocol.

OBJECTIVES:

Primary

* To identify patterns of methylation profiling that are associated with progression-free survival among young pediatric patients with medulloblastoma treated with risk-adapted therapy.
* To estimate the event-free survival distribution of young medulloblastoma patients treated with risk-adapted therapy.

Secondary

* To perform high-resolution genome-wide analyses of chromosomal abnormalities and gene expression patterns, and evaluate the relationship of these to other clinicopathological variables.
* To evaluate specific tumor types for molecular abnormalities with suspected prognostic or therapeutic significance.
* To evaluate the feasibility of collecting frozen and fixed tumor samples for analysis using high-resolution molecular biology tools.
* To estimate the event-free and overall survival of patients treated with the proposed risk-adapted therapy regimen, and to descriptively compare these survival rates to historical controls.
* To estimate the rates of local and distant disease progression in patients treated with focal radiotherapy (RT) to the post-operative tumor bed using a 5 mm clinical target volume margin.
* To estimate the objective response rate (sustained for 8 weeks) to induction chemotherapy including high-dose intravenous methotrexate for patients with residual or metastatic disease.
* To evaluate the feasibility and toxicity of administering low-dose intravenous vinblastine in conjunction with induction chemotherapy to patients with metastatic disease.
* To evaluate the feasibility and toxicity of administering consolidation therapy including cyclophosphamide and pharmacokinetically targeted topotecan to patients with metastatic disease, and to estimate the sustained (for 8 weeks) objective response rate (complete response and partial response) to such therapy in patients with measurable residual disease after induction.
* To evaluate the feasibility and toxicity of administering oral maintenance therapy in young children.
* To use quantitative magnetic resonance (MR) measures (volumetric, diffusion, and perfusion) of young brain tumor patients receiving chemotherapy including high-dose intravenous methotrexate to assess impact of treatment on developing brain.
* To investigate the feasibility of using PET as an in-vivo dosimetric and distal edge verification system for patients treated with proton beam therapy (for participants enrolled at St Jude only).

OUTLINE: This is a multicenter study. Patients are stratified according to disease risk (low-risk vs intermediate-risk vs high-risk). Therapy consists of risk adapted induction, consolidation and maintenance chemotherapy. Focal irradiation is given to intermediate risk patients who have reached at least 12 months of age upon completion of induction. Intermediate risk patients who have not will receive low risk chemotherapy to delay RT until the age of 12 months.

Patients may consent to provide tumor tissue and blood samples for biological studies. Tumor tissues are analyzed for the activation of the wnt signaling pathway (β-catenin), activation of the shh signaling pathway (Gli-1/SFRP1), and ERBB2; validation of novel patterns of gene expression via immunohistochemical (IHC) analysis; loss of chromosomes 6, 8p, 9q22, isochromosome 17q; amplification of MYCC, MYCN, and MYCL; validation of genetic abnormalities via interphase fluorescence in situ hybridization (iFISH); construction of gene expression profiles via microarray analysis; single nucleotide polymorphism (SNP) analysis for DNA purity and integrity using UV spectrophotometry and agarose gel electrophoresis; amplification of DNA via PCR and a combination of previously published and 'in-house' generated primers; potential oncogenes and tumor suppressor genes via DNA sequence analysis; expression of a number of cell signal proteins implicated in the biology of medulloblastoma via western blot; expression of additional proteins encoded by genes associated through SNP and gene expression array analysis with clinical disease behavior; and differential expression pattern of genes detected using microarray analysis via RT-PCR. DNA extraction and construction of tissue microarrays (TMAs) from tumor tissue will also be used for future IHC and FISH analysis. Blood samples are analyzed for constitutional DNA from patients whose tumors contain gene mutations via sequence analysis of constitutional DNA; cyclophosphamide and its metabolites via liquid chromatography mass spectroscopy method; topotecan lactone via isocratic high-performance liquid chromatography assay with fluorescence detection; and alpha-1-acid glycoprotein (AAGP) concentrations via immunoturbidimetric assay.

After completion of study treatment, patients are followed every 6 months for 5 years.

Eligibility criteria: Histologically confirmed newly diagnosed CNS tumors of any of the following :

* Medulloblastoma (all histologic subtypes, including medullomyoblastoma and melanotic medulloblastoma)
* Supratentorial primitive neuroectodermal tumor (PNET) (including CNS neuroblastoma or ganglioneuroblastoma, medulloepithelioma, and ependymoblastoma)
* Pineoblastoma
* Atypical teratoid rhabdoid tumor (ATRT)
* Choroid plexus carcinoma
* High grade glioma (including anaplastic astrocytoma, anaplastic oligodendroglioma, anaplastic ganglioglioma, pleomorphic xanthoastrocytoma with anaplastic features, high-grade astroblastoma , anaplastic pilocytic astrocytoma, malignant glioneuronal tumor, glioblastoma multiforme), or gliosarcoma,
* Ependymoma (including all ependymoma histological variants)
* Age \< 3 years at time of diagnosis for all histological diagnosis. Medulloblastoma patients ≥ 3 and \< 5years old at diagnosis who have non-metastatic disease with no more than 1cm2 of residual tumor are also eligible.

  * Meets criteria for 1 of the following risk groups:
* Low-risk group:

  * Histologically confirmed nodular desmoplastic medulloblastoma, including medulloblastoma with extensive nodularity

    * Focal areas of anaplasia or other atypical features suggesting more aggressive phenotype in a tumor otherwise considered nodular desmoplastic should be treated on the intermediate-risk group, with final risk stratification at the discretion of principal investigator and study pathologist
  * No evidence of CNS metastasis 7 to 28 days after surgery by MRI and cytologic examination of lumbar cerebrospinal fluid (CSF)

    * Ventricular CSF from a shunt or Ommaya reservoir may be used to rule out M1 disease when lumbar puncture is medically contraindicated
    * Intermediate-risk group assignment when there is no other evidence of metastasis and CSF sampling is not possible
  * Gross total resection, defined as residual tumor or imaging abnormality (not definitive for residual tumor) with a size of \< 1 cm2 confirmed on postoperative CT scan or MRI
  * Brain stem invasion by the tumor in the absence of imaging evidence of residual tumor (tumor size \< 1 cm2) and otherwise meets criteria for the low-risk group, the patient will be classified as low-risk
  * Desmoplastic medulloblastoma patients who are ≥3 -\<5 years of age will NOT be eligible for the low risk arm of the protocol.
* Intermediate-risk group:

  * Histologically confirmed nodular desmoplastic medulloblastoma with less than gross total resection and no evidence of metastasis
  * Any eligible histologic diagnosis other than desmoplastic medulloblastoma with no evidence of CNS metastasis
  * Medulloblastoma patients who are ≥3 and \< 5 yrs of age irrespective of histology and with no evidence of CNS metastasis
* High-risk group:

  * Any eligible histologic diagnosis with evidence of CNS metastasis
  * Patients with extraneural metastasis are eligible for treatment on the high-risk group

PATIENT CHARACTERISTICS:

* Lansky performance status ≥ 30 (except for posterior fossa syndrome)
* WBC \> 2,000/mm3
* Platelets \> 50,000/mm3 (without support)
* Hemoglobin \> 8 g/dL (with or without support)
* ANC \> 500/mm3
* Serum creatinine \< 3 times upper limit of normal (ULN)
* ALT \< 5 times ULN
* Total bilirubin \< 3 times ULN

PRIOR CONCURRENT THERAPY:

* See Disease Characteristics
* No more than 31 days since prior definitive surgery
* No prior radiotherapy or chemotherapy other than corticosteroid therapy

Primary outcomes:
measure: Percent Probability of Progression-free Survival (PFS) for Medulloblastoma Patients
timeFrame: From date on treatment until date of first progression or relapse or disease related death or date of last contact, estimated at 1 year after treatment
measure: Percent Probability of Progression-free Survival (PFS) for Medulloblastoma Patients by DNA Methylation Subgroup
timeFrame: From date on treatment to date of first progression or relapse or disease related death or date of last contact, estimated at 1 year after treatment
measure: Percent Probability of Event-free Survival (EFS) for Medulloblastoma Patients
timeFrame: From date on treatment to date of first progression, relapse, second malignancy or death from any cause or to date of last contact, estimated at 1 year after
Secondary outcomes:
measure: Number of Participants With Chromosomal Abnormalities
timeFrame: Based on samples obtained at the time of initial surgery or repeat surgery prior to treatment
measure: Numbers of Patients With Gene Alterations
timeFrame: Based on samples obtained at the time of initial surgery or repeat surgery prior to treatment
measure: Numbers of Patients With Molecular Abnormalities by Tumor Type
timeFrame: Based on samples obtained at the time of initial surgery or repeat surgery prior to treatment
measure: Number of Successful Collections for Frozen and Fixed Tumor Samples
timeFrame: Based on samples obtained at the time of initial surgery or repeat surgery prior to treatment
measure: Event-free Survival (EFS) Compared to Historical Controls
timeFrame: From date on treatment until date of first event (progression, second malignancy or death) or until date of last contact, assessed up to 10 years
measure: Overall Survival (OS) Compared to Historical Controls
timeFrame: 1 year after treatment initiation of last patient
measure: Percentage of Patients With Objective Responses Rate to Induction Chemotherapy
timeFrame: From on-study date to 2 months after completion of induction chemotherapy (up to 4 months after on-study date)
measure: Feasibility and Toxicity of Administering Vinblastine With Induction Chemotherapy for Patients With Metastatic Disease as Measured by the Percentage of Courses Delayed for More Than 7 Days Due to Toxicity
timeFrame: From on-study date up to 4 months after on-study date
measure: Feasibility and Toxicity of Administering Consolidation Therapy Including Cyclophosphamide and Pharmacokinetically Targeted Topotecan to Patients With Metastatic Disease Based on the Percentage of Courses Delayed for More Than 7 Days Due to Toxicity
timeFrame: At completion of consolidation therapy (up to 6 months after on-study date)
measure: Percent of Patients With Sustained Objective Responses Rate After Consolidation
timeFrame: 8 weeks after completion of consolidation therapy (up to 8 months after on-study date)
measure: Feasibility and Toxicity of Administering Oral Maintenance Therapy in Children <3 Years of Age as Measured by the Percentage of Total Scheduled Maintenance Doses Received
timeFrame: From start of oral maintenance therapy (approximately 6 months after on-study date) to completion of oral maintenance therapy (up to 1 year after on-study date)
measure: Percent of PET Scans With Loss of Signal Intensity
timeFrame: Up to 3 times during RT consolidation
measure: Concentration of Cerebrospinal Fluid Neurotransmitters
timeFrame: Baseline, at the completion of therapy, and every 12 months up to 36 months after off therapy date
measure: Number and Type of Genetic Polymorphisms
timeFrame: At study enrollment (Day 0)
measure: Pharmacogenetic Variation on Central Nervous System Transmitters
timeFrame: At study enrollment (Day 0)
measure: Number of Participants With Endocrinopathy
timeFrame: Baseline, end of therapy, and at 6- and 24-months after completion of therapy
measure: Longitudinal Change in Growth Hormone Secretion
timeFrame: Baseline, end of therapy, and at 6- and 24-months after completion of therapy
measure: Methotrexate Clearance in Induction Cycle 1
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of Methotrexate (MTX)
measure: Methotrexate Clearance in Induction Cycle 2
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate Clearance in Induction Cycle 3
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate Clearance in Induction Cycle 4
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate Volume of Central Compartment in Induction Cycle 1
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate Volume of Central Compartment in Induction Cycle 2
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate Volume of Central Compartment in Induction Cycle 3
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate AUC0-66h in Induction Cycle 1
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate AUC0-66h in Induction Cycle 2
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate Volume of Central Compartment in Induction Cycle 4
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate AUC0-66h in Induction Cycle 3
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate AUC0-66h in Induction Cycle 4
timeFrame: Pre-infusion and 6, 23, 42, 66 hours from start of MTX
measure: Methotrexate Concentration at 42 Hours Post-dose in Induction Cycle 1
timeFrame: 42 hours from start of MTX
measure: Methotrexate Concentration at 42 Hours Post-dose in Induction Cycle 2
timeFrame: 42 hours from start of MTX
measure: Methotrexate Concentration at 42 Hours Post-dose in Induction Cycle 3
timeFrame: 42 hours from start of MTX
measure: Methotrexate Concentration at 42 Hours Post-dose in Induction Cycle 4
timeFrame: 42 hours from start of MTX
measure: Cyclophosphamide Clearance in Induction Chemotherapy
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: Cyclophosphamide Clearance in Consolidation Chemotherapy Cycle 1
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: Cyclophosphamide Clearance in Consolidation Chemotherapy Cycle 2
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: Cyclophosphamide Apparent Oral Clearance in Maintenance Chemotherapy Cycle A1
timeFrame: Pre-dose, 0.5, 1.75, 3 and 6 hours post-dose
measure: Cyclophosphamide AUC0-24h in Induction Chemotherapy
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: Cyclophosphamide AUC0-24h in Consolidation Chemotherapy Cycle 1
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: 4-OH Cyclophosphamide AUC0-24h in Consolidation Chemotherapy Cycle 1
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: Cyclophosphamide AUC0-24h in Consolidation Chemotherapy Cycle 2
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: Cyclophosphamide AUC0-24h in Maintenance Chemotherapy Cycle A1
timeFrame: Pre-dose, 0.5, 1.75, 3, 6, and 24 hours post-dose
measure: 4-OH Cyclophosphamide AUC0-24h in Induction Chemotherapy
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: 4-OH Cyclophosphamide AUC0-24h in Consolidation Chemotherapy Cycle 2
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: 4-OH Cyclophosphamide AUC0-24h in Maintenance Chemotherapy Cycle A1
timeFrame: Pre-dose, 0.5, 1.75, 3, 6, and 24 hours post-dose
measure: CEPM AUC0-24h in Induction Chemotherapy
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: CEPM AUC0-24h in Consolidation Chemotherapy Cycle 1
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: CEPM AUC0-24h in Consolidation Chemotherapy Cycle 2
timeFrame: Pre-infusion, end of infusion, 3, 6, and 24 hours from end of cyclophosphamide infusion
measure: CEPM AUC0-24h in Maintenance Chemotherapy Cycle A1
timeFrame: Pre-dose, 0.5, 1.75, 3, 6, and 24 hours post-dose
measure: Participants With Empirical Dosage Achieving Target System Exposure of Intravenous Topotecan
timeFrame: Pre-infusion, 5 min., 1, and 3 hours from end of infusion
measure: Participants With PK-guided Dosage Adjustment Achieving Target System Exposure of Intravenous Topotecan
timeFrame: Pre-infusion, 5 min., 1, and 3 hours from end of infusion
measure: Topotecan Clearance in Consolidation Chemotherapy
timeFrame: Pre-infusion, 5 min., 1, and 3 hours from end of infusion
measure: Topotecan Apparent Oral Clearance in Maintenance Chemotherapy
timeFrame: Pre-dose, 0.25, 1.5 and 6 hours post-dose
measure: Topotecan AUC0-24h in Consolidation Chemotherapy
timeFrame: Pre-infusion, 5 min., 1, 3, and 24 hours from end of infusion
measure: Topotecan AUC0-24h in Maintenance Chemotherapy
timeFrame: Pre-dose, 0.25, 1.5, 6, and 24 hours post-dose
measure: Erlotinib Apparent Oral Clearance
timeFrame: Pre-dose, 1, 2, 4, 8, and 24 hours post-dose
measure: Erlotinib Apparent Volume of Central Compartment
timeFrame: Pre-dose, 1, 2, 4, 8, and 24 hours post-dose
measure: Erlotinib AUC0-24h
timeFrame: Pre-dose, 1, 2, 4, 8, and 24 hours post-dose
measure: OSI-420 AUC0-24h
timeFrame: Pre-dose, 1, 2, 4, 8, and 24 hours post-dose
measure: Rate of Local Disease Progression
timeFrame: 1 year after completion of radiation therapy for last patient
measure: Rate of Distant Disease Progression
timeFrame: 1 year after completion of radiation therapy for last patient
measure: Neurocognitive Performance Related to Global Cognitive Functioning as Measured by Cognitive Composite Scores and Estimated IQ Scores
timeFrame: Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy
measure: Neurocognitive Performance Related to Attention as Measured by Attention Problems T-scores
timeFrame: Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy
measure: Neurocognitive Performance Related to Processing Speed as Measured by Visual Matching Standard Scores
timeFrame: Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy
measure: Neurocognitive Performance Related to Executive Functioning as Measured by Global Executive Composite T-scores
timeFrame: Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy
measure: Neurocognitive Performance Related to Working Memory as Measured by Working Memory T-scores
timeFrame: Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy
measure: Neurocognitive Performance Related to Verbal Fluency as Measured by Retrieval Fluency Standard Scores
timeFrame: Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy
measure: Neurocognitive Performance Related to Visual-spatial Reasoning as Measured by Visual Motor Integration (VMI) T-scores
timeFrame: Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy
measure: Neurocognitive Performance Related to Visual-spatial Reasoning as Measured by Visual Perception T-scores
timeFrame: Baseline, prior to maintenance therapy, completion of therapy, and 12 months, 24 months, 36 months, 48 months, and 60 months off therapy
measure: Change in Neurostructure, Especially White Matter Volume and Integrity
timeFrame: From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)
measure: Change in Neurostructure, Especially White Matter Volume and Integrity
timeFrame: From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)
measure: Change in Neurostructure, Especially White Matter Volume and Integrity
timeFrame: From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)
measure: Change in Neurostructure, Especially White Matter Volume and Integrity
timeFrame: From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)
measure: Change in Neurostructure, Especially White Matter Volume and Integrity
timeFrame: From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)
measure: Change in Neurostructure, Especially White Matter Volume and Integrity
timeFrame: From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)
measure: Change in Neurostructure, Especially White Matter Volume and Integrity
timeFrame: From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)
measure: Change in Neurostructure, Especially White Matter Volume and Integrity
timeFrame: From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)
measure: Change in Quantitative Magnetic Resonance (MR) Measures in the Frontal Lobe
timeFrame: From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)
measure: Change in Quantitative MR Measures in the Right Frontal-parietal Regions
timeFrame: From baseline (month 0) to 60 months (therapy duration lasted approximately 48 weeks or 11 months)
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital at Stanford University Medical Center
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital
state: California
city: Saint Paul
country: United States
facility: Children's Hospitals and Clinics of Minnesota - St. Paul
state: Minnesota
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center at Dallas
state: Texas
city: Brisbane
country: Australia
facility: Lady Cilento Children's Hospital, Brisbane
state: Queensland
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Amar Gajjar, MD
role: STUDY_CHAIR
References:
citation: Ali JS, Ashford JM, Swain MA, Harder LL, Carlson-Green BL, Miller JM, Wallace J, Kaner RJ, Billups CA, Onar-Thomas A, Merchant TE, Gajjar A, Conklin HM. Predictors of Cognitive Performance Among Infants Treated for Brain Tumors: Findings From a Multisite, Prospective, Longitudinal Trial. J Clin Oncol. 2021 Jul 20;39(21):2350-2358. doi: 10.1200/JCO.20.01687. Epub 2021 May 4.
citation: Kumar R, Smith KS, Deng M, Terhune C, Robinson GW, Orr BA, Liu APY, Lin T, Billups CA, Chintagumpala M, Bowers DC, Hassall TE, Hansford JR, Khuong-Quang DA, Crawford JR, Bendel AE, Gururangan S, Schroeder K, Bouffet E, Bartels U, Fisher MJ, Cohn R, Partap S, Kellie SJ, McCowage G, Paulino AC, Rutkowski S, Fleischhack G, Dhall G, Klesse LJ, Leary S, Nazarian J, Kool M, Wesseling P, Ryzhova M, Zheludkova O, Golanov AV, McLendon RE, Packer RJ, Dunham C, Hukin J, Fouladi M, Faria CC, Pimentel J, Walter AW, Jabado N, Cho YJ, Perreault S, Croul SE, Zapotocky M, Hawkins C, Tabori U, Taylor MD, Pfister SM, Klimo P Jr, Boop FA, Ellison DW, Merchant TE, Onar-Thomas A, Korshunov A, Jones DTW, Gajjar A, Ramaswamy V, Northcott PA. Clinical Outcomes and Patient-Matched Molecular Composition of Relapsed Medulloblastoma. J Clin Oncol. 2021 Mar 1;39(7):807-821. doi: 10.1200/JCO.20.01359. Epub 2021 Jan 27.
citation: Robinson GW, Rudneva VA, Buchhalter I, Billups CA, Waszak SM, Smith KS, Bowers DC, Bendel A, Fisher PG, Partap S, Crawford JR, Hassall T, Indelicato DJ, Boop F, Klimo P, Sabin ND, Patay Z, Merchant TE, Stewart CF, Orr BA, Korbel JO, Jones DTW, Sharma T, Lichter P, Kool M, Korshunov A, Pfister SM, Gilbertson RJ, Sanders RP, Onar-Thomas A, Ellison DW, Gajjar A, Northcott PA. Risk-adapted therapy for young children with medulloblastoma (SJYC07): therapeutic and molecular outcomes from a multicentre, phase 2 trial. Lancet Oncol. 2018 Jun;19(6):768-784. doi: 10.1016/S1470-2045(18)30204-3. Epub 2018 May 16.