Vismodegib in Treating Patients With Recurrent or Refractory Medulloblastoma

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:
NCT00939484
First received: July 14, 2009
Last updated: January 28, 2016
Last verified: March 2015
Results First Received: December 10, 2015  
Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Adult Medulloblastoma
Interventions: Other: Pharmacological Study
Drug: Vismodegib

  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
This study was distributed to the sites on June 18, 2009, and received the first IRB approval on July 20, 2009. The study was closed to accrual on December 07, 2012. Thirty two (32) patients have been enrolled to the study, Last patient went off treatment in October 2013.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Total 32 patients were registered on this study, 8 in Stratum A, 21 in Stratum B and 3 in Stratum C. One patient in Stratum B was declared ineligible.

Reporting Groups
  Description
Stratum A (PTCH/SHH Pathway Inactivated) Adult patients with recurrent or refractory medulloblastoma who have tumors without evidence of PTCH/SHH pathway activation
Stratum B (PTCH/SHH Pathway Activated) Adult patients with recurrent or refractory medulloblastoma who have tumors with evidence of activation of the PTCH/SHH pathway
Stratum C (Unknown PTCH/SHH Pathway Activation) Adult patients with recurrent or refractory medulloblastoma registered on protocol whose tumor assay is inconclusive as to whether or not the PTCH/SHH pathway is activated or whose tissue sample is inadequate to assess the PTCH/SHH pathway status

Participant Flow:   Overall Study
    Stratum A (PTCH/SHH Pathway Inactivated)     Stratum B (PTCH/SHH Pathway Activated)     Stratum C (Unknown PTCH/SHH Pathway Activation)  
STARTED     8     20     3  
COMPLETED     0     0     0  
NOT COMPLETED     8     20     3  
Progression/Relapse                 8                 20                 3  



  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.
One patient in Stratum B was ineligible for the study and thus was not included in the baseline table.

Reporting Groups
  Description
Stratum A (PTCH/SHH Pathway Inactivated) Adult patients with recurrent or refractory medulloblastoma who have tumors without evidence of PTCH/SHH pathway activation
Stratum B (PTCH/SHH Pathway Activated) Adult patients with recurrent or refractory medulloblastoma who have tumors with evidence of activation of the PTCH/SHH pathway
Stratum C (Unknown PTCH/SHH Pathway Activation) Adult patients with recurrent or refractory medulloblastoma registered on protocol whose tumor assay is inconclusive as to whether or not the PTCH/SHH pathway is activated or whose tissue sample is inadequate to assess the PTCH/SHH pathway status
Total Total of all reporting groups

Baseline Measures
    Stratum A (PTCH/SHH Pathway Inactivated)     Stratum B (PTCH/SHH Pathway Activated)     Stratum C (Unknown PTCH/SHH Pathway Activation)     Total  
Number of Participants  
[units: participants]
  8     20     3     31  
Age [1]
[units: years]
Median (Full Range)
  23.8   (22.4 to 40.6)     32.0   (23.5 to 51.9)     32.9   (25.3 to 38.6)     30.3   (22.4 to 51.9)  
Gender  
[units: participants]
       
Female     2     9     2     13  
Male     6     11     1     18  
Ethnicity (NIH/OMB)  
[units: participants]
       
Hispanic or Latino     0     1     1     2  
Not Hispanic or Latino     7     18     2     27  
Unknown or Not Reported     1     1     0     2  
Race (NIH/OMB)  
[units: participants]
       
American Indian or Alaska Native     0     0     0     0  
Asian     0     0     0     0  
Native Hawaiian or Other Pacific Islander     0     0     0     0  
Black or African American     0     3     0     3  
White     7     16     3     26  
More than one race     0     0     0     0  
Unknown or Not Reported     1     1     0     2  
Region of Enrollment  
[units: participants]
       
United States     8     20     3     31  
Diagnosis  
[units: participants]
       
Desmoplastic medulloblastoma     0     1     0     1  
Medulloblastoma, NOS     8     18     3     29  
Primitive neuroectodermal tumor     0     1     0     1  
[1] Age at study enrollment



  Outcome Measures
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1.  Primary:   Objective Response (CR+PR) Sustained for ≥ 8 Weeks   [ Time Frame: Up to 12 months ]

2.  Secondary:   Progression-free Survival   [ Time Frame: From start of treatment up to 2 years ]

3.  Secondary:   Duration of Objective Response   [ Time Frame: From start of treatment up to 2 years ]

4.  Secondary:   Pharmacokinetic Parameters of Vismodegib, CSF Penetration   [ Time Frame: up to 12 month ]


  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
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Tong Lin (Biostatistician)
Organization: St. Jude Children's Research Hospital
phone: 9015952048
e-mail: Tong.Lin@stjude.org


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00939484     History of Changes
Other Study ID Numbers: NCI-2012-03020
NCI-2012-03020 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
PBTC-025B ( Other Identifier: Pediatric Brain Tumor Consortium )
PBTC-025B ( Other Identifier: CTEP )
U01CA081457 ( US NIH Grant/Contract Award Number )
Study First Received: July 14, 2009
Results First Received: December 10, 2015
Last Updated: January 28, 2016
Health Authority: United States: Food and Drug Administration