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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT00085202
Registration number
NCT00085202
Ethics application status
Date submitted
10/06/2004
Date registered
11/06/2004
Date last updated
8/02/2024
Titles & IDs
Public title
Treatment of Patients With Newly Diagnosed Medulloblastoma, Supratentorial Primitive Neuroectodermal Tumor, or Atypical Teratoid Rhabdoid Tumor
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Scientific title
Treatment of Patients With Newly Diagnosed Medulloblastoma, Supratentorial Primitive Neuroectodermal Tumor, or Atypical Teratoid Rhabdoid Tumor
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Secondary ID [1]
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NCI-2011-01185
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Secondary ID [2]
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SJMB03
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Brain and Central Nervous System Tumors
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Condition category
Condition code
Cancer
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Neuroendocrine tumour (NET)
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Cancer
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Children's - Brain
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Cancer
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Brain
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Cancer
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Children's - Other
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Cancer
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Sarcoma (also see 'Bone') - soft tissue
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Cancer
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Kidney
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Other - filgrastim
Treatment: Drugs - cisplatin
Treatment: Drugs - cyclophosphamide
Treatment: Drugs - vincristine
Treatment: Surgery - autologous hematopoietic stem cell transplantation
Treatment: Other - radiation therapy
Experimental: Stratum 1 (high-risk group) - Patients undergo craniospinal radiotherapy once daily 5 days a week for 6 weeks. Six weeks after the completion of radiotherapy, patients receive high-dose chemotherapy followed by autologous stem cell transplantation (SCT) and filgrastim (G-CSF) with post-transplantation vincristine. High-dose chemotherapy and autologous SCT repeat every 4 weeks for 3 additional courses in the absence of unacceptable toxicity.
Interventions: vincristine, cisplatin, cyclophosphamide, autologous hematopoietic stem cell transplantation, filgrastim, radiation therapy
Experimental: Stratum 2 (average-risk group) - Patients undergo craniospinal radiotherapy as in stratum 1, but at a lower dose. Patients receive high-dose chemotherapy, autologous SCT, G-CSF, and post-transplantation vincristine as in stratum 1.
Interventions: vincristine, cisplatin, cyclophosphamide, autologous hematopoietic stem cell transplantation, filgrastim, radiation therapy
Treatment: Other: filgrastim
Given subcutaneously
Treatment: Drugs: cisplatin
Given IV
Treatment: Drugs: cyclophosphamide
Given IV
Treatment: Drugs: vincristine
Given IV
Treatment: Surgery: autologous hematopoietic stem cell transplantation
Patients undergo autologous stem cell transplantation
Treatment: Other: radiation therapy
Patients undergo craniospinal radiotherapy once daily 5 days a week for 6 weeks.
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Intervention code [1]
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Treatment: Other
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Intervention code [2]
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Treatment: Drugs
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Intervention code [3]
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Treatment: Surgery
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Progression-Free Survival (PFS) in ERBB2-Negative Tumors Compared to ERBB2-Positive Tumors
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Assessment method [1]
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The relationship between ERBB2 protein expression in tumors and progression-free survival was assessed in 122 participants with a diagnosis of medulloblastoma and with ERBB2 protein assessments. If the ERBB2 value was greater than zero, the ERBB2 was defined as positive for the participant. If the ERBB2 value was zero, the ERBB2 was defined as negative. Progression-free survival was calculated from the date of diagnosis to the date of disease progression/relapse, the date of death, or the date of last contact. The log-rank test was used to compare the PFS distributions of ERBB2 groups.
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Timepoint [1]
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2 years after tumor cell analysis in 122 participants
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Primary outcome [2]
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Progression-Free Survival (PFS) Compared Between ERBB2 Assessment and Risk Group.
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Assessment method [2]
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122 participants with a diagnosis of medulloblastoma were grouped by ERBB2 positive/negative assessment and risk group into 4 groups. Progression-free survival was calculated from the date of diagnosis to the date of disease progression/relapse, the date of death, or the date of last contact. The log-rank test was used to compare the PFS distributions of ERBB2 groups.
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Timepoint [2]
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2 years after tumor cell analysis in 122 participants
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Primary outcome [3]
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Frequency of Mutations Associated With SHH and WNT Tumors
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Assessment method [3]
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The frequency of mutations for the main genes associated with SHH and WNT tumors identified via targeted sequencing based on formalin fixed paraffin embedded material is provided.
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Timepoint [3]
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within 3.5 years following completion of accrual
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Secondary outcome [1]
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Reading Decoding Composite Scores in the Intervention and Standard of Care Groups
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Assessment method [1]
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SOC is standard-of-Care control group. Patients randomly assigned to the control group received the current standard of care. RI is Reading Intervention Group. Patients randomly assigned to Reading Intervention Group which is with The Fast ForWord program. Assessment of reading decoding was completed using the Woodcock Johnson, Third Edition (WJIII) Tests of Achievement (Woodcock, McGraw, \& Mather, 2001), with particular attention given to the reading and reading-related abilities. Two subtests were completed: (1) Letter-Word Identification, and (2) Word Attack, a test requiring the patient to read phonologically regular nonwords. The combination of these two subtests provided a standardized composite score of overall reading decoding ability with a population mean of 100 and a standard deviation of 15. Scores of 90-110 are considered to be in the average range, while those 80-89 are considered low-average (refer: Journal of Pediatric Psychology 39(4) pp. 450-458, 2014).
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Timepoint [1]
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5 years postdiagnosis
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Secondary outcome [2]
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Number of Average Risk Patients Whose Treatment Failure Included the Posterior Fossa
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Assessment method [2]
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To monitor for treatment failure in the posterior fossa of patients whose tumor bed receives a reduced volume of radiation.
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Timepoint [2]
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Annually for 6 years post irradiation
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Secondary outcome [3]
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Associative Memory for Two Risk Group at Enrollment
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Assessment method [3]
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Assessment of associative memory at enrollment. Associate memory score is a representative measurement of learning and recalling pictograph representations of words. It measures associative memory (Long-Term Retrieval). Mean=100, SD=15, Average Range 85-115. Higher is better.
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Timepoint [3]
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At enrollment
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Secondary outcome [4]
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Associative Memory for Two Risk Group at 5 Years After Enrollment
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Assessment method [4]
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Assessment of associative memory at 5 years after enrollment. Assessment of associative memory score at enrollment. Associate memory score is a representative measurement of learning and recalling pictograph representations of words. It measures associative memory (Long-Term Retrieval). Mean=100, SD=15, Average Range 85-115. Higher is better.
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Timepoint [4]
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At 5 years after enrollment
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Secondary outcome [5]
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Processing Speed for Two Risk Group at Enrollment
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Assessment method [5]
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Assessment of Processing Speed at enrollment. This score measure of Processing Speed based on Visual Matching and Decision Speed tests. Mean=100, SD=15, Average Range 85-115. Higher is better.
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Timepoint [5]
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At enrollment
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Secondary outcome [6]
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Processing Speed for Two Risk Group at 5 Years After Enrollment
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Assessment method [6]
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Assessment of Processing Speed at 5 years after enrollment. This score measure of Processing Speed based on Visual Matching and Decision Speed tests. Mean=100, SD=15, Average Range 85-115. Higher is better.
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Timepoint [6]
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At 5 years after enrollment
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Secondary outcome [7]
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Perceptual Speed for Two Risk Group at Enrollment
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Assessment method [7]
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Assessment of Perceptual Speed at enrollment. It measures rapidly locating and circling identical numbers from a set of numbers which reflects perceptual speed. Mean=100, SD=15, Average Range 85-115. Higher is better.
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Timepoint [7]
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At enrollment
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Secondary outcome [8]
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Perceptual Speed for Two Risk Group at 5 Years After Enrollment
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Assessment method [8]
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Assessment of Perceptual Speed at 5 years after enrollment. It measures rapidly locating and circling identical numbers from a set of numbers which reflects perceptual speed. Mean=100, SD=15, Average Range 85-115. Higher is better.
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Timepoint [8]
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At 5 years after enrollment
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Eligibility
Key inclusion criteria
DISEASE CHARACTERISTICS:
* Histologically confirmed diagnosis of 1 of the following:
* Medulloblastoma
* Supratentorial primitive neuroectodermal tumor (PNET)
* PNET variants (ependymoblastoma, pineoblastoma, CNS neuroblastoma)
* Atypical teratoid rhabdoid tumor (ATRT)
* Definitive surgery for CNS tumor within the past 31 days
* Meets one of the following risk criteria:
* Average-risk disease
* Localized disease with no overt evidence of invasion beyond the posterior fossa (or supratentorial compartment for PNET or ATRT) by intraoperative observations of the neurosurgeon AND postoperative CT scan or MRI
* T4 disease eligible if all of the following are true:
* Gross total resection determined by intraoperative observations of the neurosurgeon AND postoperative CT scan or MRI
* Residual tumor or imaging abnormality whose size is < 1.5 cm^2
* No evidence of CNS or extraneural metastasis by MRI of the spine (with and without contrast agent) or CT-based myelogram AND by cytologic examination of the lumbar cerebral spinal fluid (CSF) 14-28 days after surgery
* Brain stem invasion allowed in the absence of residual tumor (tumor < 1.5 cm^2 by imaging)
* High-risk disease meeting one of the following criteria:
* Metastatic disease within the neuraxis (i.e., evidence of subarachnoid dissemination by imaging and/or cytologic examination of CSF)
* Presence of residual disease > 1.5 cm^2 at the primary site after surgery
PATIENT CHARACTERISTICS:
Age
* 3 to 21 at diagnosis
Performance status
* Lansky 30-100% (< 10 years old)
* Karnofsky 30-100% (= 10 years old) (except for posterior fossa syndrome)
Life expectancy
* Not specified
Hematopoietic
* Hemoglobin > 8 g/dL
* WBC > 2,000/mm^3
* Absolute neutrophil count > 500/mm^3
* Platelet count > 50,000/mm^3
Hepatic
* ALT < 5 times normal
* Bilirubin < 3.0 mg/dL
Renal
* Creatinine < 2.0 mg/dL OR
* Creatinine clearance > 70 mL/min
Other
* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY:
Biologic therapy
* Not specified
Chemotherapy
* No prior chemotherapy
Endocrine therapy
* Prior corticosteroid therapy allowed
Radiotherapy
* No prior radiotherapy
Surgery
* See Disease Characteristics
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Minimum age
3
Years
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Maximum age
21
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/08/2003
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
31/12/2023
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Sample size
Target
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Accrual to date
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Final
416
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
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Recruitment hospital [1]
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Sydney Children's Hospital - Randwick
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Recruitment hospital [2]
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Children's Hospital at Westmead - Westmead
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Recruitment hospital [3]
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Lady Cilento Children's Hospital, Brisbane - Brisbane
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Recruitment hospital [4]
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Royal Children's Hospital - Parkville
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Recruitment postcode(s) [1]
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2031 - Randwick
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Recruitment postcode(s) [2]
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2145 - Westmead
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Recruitment postcode(s) [3]
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4029 - Brisbane
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Recruitment postcode(s) [4]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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North Carolina
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Country [2]
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United States of America
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State/province [2]
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Pennsylvania
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Country [3]
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United States of America
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State/province [3]
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Tennessee
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Country [4]
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United States of America
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State/province [4]
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Texas
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Country [5]
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Canada
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State/province [5]
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Ontario
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Funding & Sponsors
Primary sponsor type
Other
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Name
St. Jude Children's Research Hospital
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
Drugs used in chemotherapy, such as vincristine, cisplatin, and cyclophosphamide, work in different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage tumor cells. Combining radiation therapy with chemotherapy may kill more tumor cells. Autologous stem cell transplant may be able to replace blood-forming cells that were destroyed by chemotherapy or radiation therapy. It is not yet known which radiation therapy regimen combined with chemotherapy and donor stem cell transplant is more effective in treating medulloblastoma, supratentorial primitive neuroectodermal tumor, or atypical teratoid rhabdoid tumor. This phase III trial is studying two different regimens of radiation therapy when given together with chemotherapy and autologous stem cell transplant to see how well they work in treating patients with newly diagnosed medulloblastoma, supratentorial primitive neuroectodermal tumor, or atypical teratoid rhabdoid tumor. PRIMARY OBJECTIVE: * To assess the relationship between ERBB2 protein expression in tumors and progression-free survival probability for patients with medulloblastoma. * To estimate the frequency of mutations associated with SHH and WNT tumors (as defined by gene expression profiling) via targeted sequencing performed in an independent cohort of WNT and SHH tumors (also defined by gene expression profiling).
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Trial website
https://clinicaltrials.gov/study/NCT00085202
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Trial related presentations / publications
Acharya S, Guo Y, Patni T, Li Y, Wang C, Gargone M, Ashford JM, Wilson L, Faught A, Reddick WE, Patay Z, Gajjar A, Conklin HM, Merchant TE. Association Between Brain Substructure Dose and Cognitive Outcomes in Children With Medulloblastoma Treated on SJMB03: A Step Toward Substructure-Informed Planning. J Clin Oncol. 2022 Jan 1;40(1):83-95. doi: 10.1200/JCO.21.01480. Epub 2021 Oct 29. Partanen M, Anghelescu DL, Hall L, Schreiber JE, Rossi M, Gajjar A, Jacola LM. Longitudinal associations between exposure to anesthesia and neurocognitive functioning in pediatric medulloblastoma. Eur J Cancer. 2021 May;148:103-111. doi: 10.1016/j.ejca.2021.02.010. Epub 2021 Mar 17. 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. Gajjar A, Robinson GW, Smith KS, Lin T, Merchant TE, Chintagumpala M, Mahajan A, Su J, Bouffet E, Bartels U, Schechter T, Hassall T, Robertson T, Nicholls W, Gururangan S, Schroeder K, Sullivan M, Wheeler G, Hansford JR, Kellie SJ, McCowage G, Cohn R, Fisher MJ, Krasin MJ, Stewart CF, Broniscer A, Buchhalter I, Tatevossian RG, Orr BA, Neale G, Klimo P Jr, Boop F, Srinivasan A, Pfister SM, Gilbertson RJ, Onar-Thomas A, Ellison DW, Northcott PA. Outcomes by Clinical and Molecular Features in Children With Medulloblastoma Treated With Risk-Adapted Therapy: Results of an International Phase III Trial (SJMB03). J Clin Oncol. 2021 Mar 1;39(7):822-835. doi: 10.1200/JCO.20.01372. Epub 2021 Jan 6. Xu H, Robinson GW, Huang J, Lim JY, Zhang H, Bass JK, Broniscer A, Chintagumpala M, Bartels U, Gururangan S, Hassall T, Fisher M, Cohn R, Yamashita T, Teitz T, Zuo J, Onar-Thomas A, Gajjar A, Stewart CF, Yang JJ. Common variants in ACYP2 influence susceptibility to cisplatin-induced hearing loss. Nat Genet. 2015 Mar;47(3):263-6. doi: 10.1038/ng.3217. Epub 2015 Feb 9. Erratum In: Nat Genet. 2015 Apr;47(4):423. doi: 10.1038/ng0415-423.
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Public notes
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Contacts
Principal investigator
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Amar Gajjar, MD
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Address
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St. Jude Children's Research Hospital
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Contact person for public queries
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT00085202
Download to PDF