Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma
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|ClinicalTrials.gov Identifier: NCT01774253|
Recruitment Status : Terminated (Lack of enrollment and commercial availability of drug)
First Posted : January 23, 2013
Results First Posted : October 28, 2016
Last Update Posted : February 28, 2017
The purpose of this research study is to evaluate an investigational drug (Vismodegib) for Pontine Glioma that is growing or has come back (reoccurred). This study will look at the tumors response to the study drug, Vismodegib, and will also look at the safety and tolerability of Vismodegib.
Vismodegib has been tested in multiple adult clinical trials and one pediatric trial. Laboratory testing in pontine gliomas suggests that this drug may be effective in treating this disease.
|Condition or disease||Intervention/treatment||Phase|
|Pontine Glioma||Drug: Vismodegib||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II, Open-label, Multi-center Study of Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma.|
|Study Start Date :||May 2013|
|Actual Primary Completion Date :||October 2015|
|Actual Study Completion Date :||October 2015|
Vismodegib will be dosed at 150mg-300mg orally (max dose: 300mg) once a day on days 1 to 28 of a 28-day cycle. In the absence of unacceptable toxicity or disease progression, treatment may continue for as long as tolerated.
Other Name: Erivedge
- Number of Days Participants Experienced Progression Free Survival (PFS) [ Time Frame: 5 years ]Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or the appearance of new lesions.
- Number of Participants With Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 2 years ]To determine the safety and tolerability of Vismodegib as a single agent in pediatric and young adult patients with refractory or recurrent pontine glioma
- Determine the Median Overall Survival (OS) of Participants [ Time Frame: 2 years ]Overall Survival (OS) and clinical benefit (ORR + stable disease, SD)
- Evaluate the Impact of Quality of Life of Children Receiving Vismodegib Using PedsQL Questionnaires [ Time Frame: 2 years ]Evaluate the impact of Quality of Life of children receiving Vismodegib using PedsQL questionnaires
- Determine the Response Rates of Participants Based on Activation (or no Activation) of Their Hedgehog Signaling Pathway [ Time Frame: 3 years ]To determine the objective response rates (partial and complete response) for patients without and with evidence of activation of Hedgehog signaling pathway in their tumors
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01774253
|United States, Arizona|
|Phoenix Children's Hospital|
|Phoenix, Arizona, United States, 85016|
|United States, Michigan|
|Helen DeVos Children's Hospital|
|Grand Rapids, Michigan, United States, 49503|
|Study Chair:||Giselle Sholler, MD||The Spectrum Health Group|
|Principal Investigator:||Albert Cornelius, MD||Helen DeVos Children's Hospital|