Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/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
Query!
Scientific title
Treatment of Patients With Newly Diagnosed Medulloblastoma, Supratentorial Primitive Neuroectodermal Tumor, or Atypical Teratoid Rhabdoid Tumor
Query!
Secondary ID [1]
0
0
NCI-2011-01185
Query!
Secondary ID [2]
0
0
SJMB03
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Brain and Central Nervous System Tumors
0
0
Query!
Condition category
Condition code
Cancer
0
0
0
0
Query!
Neuroendocrine tumour (NET)
Query!
Cancer
0
0
0
0
Query!
Brain
Query!
Cancer
0
0
0
0
Query!
Children's - Brain
Query!
Cancer
0
0
0
0
Query!
Children's - Other
Query!
Cancer
0
0
0
0
Query!
Sarcoma (also see 'Bone') - soft tissue
Query!
Cancer
0
0
0
0
Query!
Kidney
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Other interventions - 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
Other interventions: 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.
Query!
Intervention code [1]
0
0
Other interventions
Query!
Intervention code [2]
0
0
Treatment: Drugs
Query!
Intervention code [3]
0
0
Treatment: Surgery
Query!
Intervention code [4]
0
0
Treatment: Other
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Progression-Free Survival (PFS) in ERBB2-Negative Tumors Compared to ERBB2-Positive Tumors
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
2 years after tumor cell analysis in 122 participants
Query!
Primary outcome [2]
0
0
Progression-Free Survival (PFS) Compared Between ERBB2 Assessment and Risk Group.
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
2 years after tumor cell analysis in 122 participants
Query!
Primary outcome [3]
0
0
Frequency of Mutations Associated With SHH and WNT Tumors
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
within 3.5 years following completion of accrual
Query!
Secondary outcome [1]
0
0
Reading Decoding Composite Scores in the Intervention and Standard of Care Groups
Query!
Assessment method [1]
0
0
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).
Query!
Timepoint [1]
0
0
5 years postdiagnosis
Query!
Secondary outcome [2]
0
0
Number of Average Risk Patients Whose Treatment Failure Included the Posterior Fossa
Query!
Assessment method [2]
0
0
To monitor for treatment failure in the posterior fossa of patients whose tumor bed receives a reduced volume of radiation.
Query!
Timepoint [2]
0
0
Annually for 6 years post irradiation
Query!
Secondary outcome [3]
0
0
Associative Memory for Two Risk Group at Enrollment
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
At enrollment
Query!
Secondary outcome [4]
0
0
Associative Memory for Two Risk Group at 5 Years After Enrollment
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
At 5 years after enrollment
Query!
Secondary outcome [5]
0
0
Processing Speed for Two Risk Group at Enrollment
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
At enrollment
Query!
Secondary outcome [6]
0
0
Processing Speed for Two Risk Group at 5 Years After Enrollment
Query!
Assessment method [6]
0
0
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.
Query!
Timepoint [6]
0
0
At 5 years after enrollment
Query!
Secondary outcome [7]
0
0
Perceptual Speed for Two Risk Group at Enrollment
Query!
Assessment method [7]
0
0
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.
Query!
Timepoint [7]
0
0
At enrollment
Query!
Secondary outcome [8]
0
0
Perceptual Speed for Two Risk Group at 5 Years After Enrollment
Query!
Assessment method [8]
0
0
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.
Query!
Timepoint [8]
0
0
At 5 years after enrollment
Query!
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
Query!
Minimum age
3
Years
Query!
Query!
Maximum age
21
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Non-randomised trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/08/2003
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
31/12/2023
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
416
Query!
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Query!
Recruitment hospital [1]
0
0
Sydney Children's Hospital - Randwick
Query!
Recruitment hospital [2]
0
0
Children's Hospital at Westmead - Westmead
Query!
Recruitment hospital [3]
0
0
Lady Cilento Children's Hospital, Brisbane - Brisbane
Query!
Recruitment hospital [4]
0
0
Royal Children's Hospital - Parkville
Query!
Recruitment postcode(s) [1]
0
0
2031 - Randwick
Query!
Recruitment postcode(s) [2]
0
0
2145 - Westmead
Query!
Recruitment postcode(s) [3]
0
0
4029 - Brisbane
Query!
Recruitment postcode(s) [4]
0
0
3052 - Parkville
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
North Carolina
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Pennsylvania
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Tennessee
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Texas
Query!
Country [5]
0
0
Canada
Query!
State/province [5]
0
0
Ontario
Query!
Funding & Sponsors
Primary sponsor type
Other
Query!
Name
St. Jude Children's Research Hospital
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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).
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT00085202
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Amar Gajjar, MD
Query!
Address
0
0
St. Jude Children's Research Hospital
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT00085202
Download to PDF