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Treatment of Patients With Newly Diagnosed Medulloblastoma, Supratentorial Primitive Neuroectodermal Tumor, or Atypical Teratoid Rhabdoid Tumor

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00085202
First received: June 10, 2004
Last updated: January 9, 2014
Last verified: January 2013
Results First Received: November 4, 2013  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Brain and Central Nervous System Tumors
Interventions: Biological: filgrastim
Drug: cisplatin
Drug: cyclophosphamide
Drug: vincristine
Procedure: autologous hematopoietic stem cell transplantation
Radiation: radiation therapy

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
416 participants were enrolled between 9/9/2003 and 3/7/2013.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Of the 416 participants enrolled, three were ineligible and taken off study.

Reporting Groups
  Description
Average-Risk Group Participants assigned to the average-risk arm had localized tumor without overt evidence of invasion beyond the posterior fossa (or supratentorial compartment for PNET's/ATRT). Participants with T4 disease met the following criteria: gross total resection defined as residual tumor or imaging abnormality whose size was <1.5 cm^2 on postoperative CT or MR images, no evidence of CNS or extraneural metastasis, and brain stem invasion by the tumor in the absence of imaging evidence of residual tumor (tumor size <1.5 cm^2).
High-Risk Group Participants assigned to the high-risk arm were determined to have the presence of metastatic disease within the neuraxis (i.e., evidence of subarachnoid dissemination), OR presence of residual disease (≥1.5 cm^2) at the primary site after surgery.

Participant Flow:   Overall Study
    Average-Risk Group     High-Risk Group  
STARTED     269     144  
COMPLETED     251     120  
NOT COMPLETED     18     24  
Still on therapy                 2                 0  
Bone involvement confirmed by bone scan                 0                 1  
Excessive toxicity                 6                 5  
Non-specified                 3                 5  
Withdrawal by Subject                 2                 1  
Physician Decision                 4                 1  
Progressive disease                 1                 11  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Participants had a diagnosis of medulloblastoma, supratentorial primitive neuroectodermal tumor (PNET), PNET variants (ependymoblastoma, pineoblastoma, CNS neuroblastoma), or atypical teratoid rhabdoid tumor (ATRT).

Reporting Groups
  Description
Average-Risk Group Participants assigned to the average-risk arm had localized tumor without overt evidence of invasion beyond the posterior fossa (or supratentorial compartment for PNET's/ATRT). Participants with T4 disease met the following criteria: gross total resection defined as residual tumor or imaging abnormality whose size was <1.5 cm^2 on postoperative CT or MR images, no evidence of CNS or extraneural metastasis, and brain stem invasion by the tumor in the absence of imaging evidence of residual tumor (tumor size <1.5 cm^2).
High-Risk Group Participants assigned to the high-risk arm were determined to have the presence of metastatic disease within the neuraxis (i.e., evidence of subarachnoid dissemination), OR presence of residual disease (≥1.5 cm^2) at the primary site after surgery.
Total Total of all reporting groups

Baseline Measures
    Average-Risk Group     High-Risk Group     Total  
Number of Participants  
[units: participants]
  269     144     413  
Age  
[units: Years]
Mean ± Standard Deviation
  9.15  ± 4.08     8.66  ± 3.93     8.98  ± 4.03  
Age  
[units: years]
Median ( Full Range )
  8.42  
  ( 3.06 to 21.56 )  
  7.87  
  ( 3.13 to 20.43 )  
  8.33  
  ( 3.06 to 21.56 )  
Gender  
[units: participants]
     
Female     105     56     161  
Male     164     88     252  



  Outcome Measures
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1.  Primary:   Progression-Free Survival (PFS) in ERBB2-Negative Tumors Compared to ERBB2-Positive Tumors   [ Time Frame: 2 years after tumor cell analysis in 122 participants ]

2.  Primary:   Progression-Free Survival (PFS) Compared Between ERBB2 Assessment and Risk Group.   [ Time Frame: 2 years after tumor cell analysis in 122 participants ]

3.  Primary:   Frequency of Mutations Associated With SHH and WNT Tumors   [ Time Frame: within 18 months following completion of accrual ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

4.  Secondary:   Reading Decoding Composite Scores in the Intervention and Standard of Care Groups   [ Time Frame: Measurements will be made at time of randomization, at 3 months from initiation of treatment, and yearly thereafter for 5 years ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

5.  Secondary:   Number of Average Risk Patients Whose Treatment Failure Included the Posterior Fossa   [ Time Frame: Annually for 6 years post irradiation ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

6.  Secondary:   Mean RT Dose to Specified Target Tissue Volume by Rate and Pattern of Failure, e.g. Local Failure, Distant Failure, Etc.   [ Time Frame: Once all patients have been followed for 2 years ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The study is ongoing. Participant flow and adverse events will be updated as additional outcomes are reported.


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Amar Gajjar, MD
Organization: St. Jude Children's Research Hospital
phone: 866-278-5833
e-mail: info@stjude.org


No publications provided


Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00085202     History of Changes
Other Study ID Numbers: SJMB03
Study First Received: June 10, 2004
Results First Received: November 4, 2013
Last Updated: January 9, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board