Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma
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.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II, Open-label, Multi-center Study of Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma.|
- Determine the Progression Free Survival (PFS) of Participants using days until progression [ Time Frame: 5 years ] [ Designated as safety issue: No ]To determine the activity of Vismodegib based on Progression Free Survival (PFS) in pediatric and adolescent subjects with refractory or recurrent pontine glioma.
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]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 ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]To evaluate the impact of QOL of children receiving Vismodegib
- Determine the response rates of Participants based on activation (or no activation) of their hedgehog signaling pathway [ Time Frame: 3 years ] [ Designated as safety issue: No ]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
|Study Start Date:||May 2013|
|Estimated Study Completion Date:||April 2021|
|Estimated Primary Completion Date:||April 2016 (Final data collection date for primary outcome measure)|
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
|Contact: Genevieve Bergendahl, RNfirstname.lastname@example.org|
|Contact: Alyssa VanderWerff||(616) email@example.com|
|United States, Arizona|
|Phoenix Children's Hospital||Recruiting|
|Phoenix, Arizona, United States, 85016|
|Contact: Sam Chimienti 602-546-0188 firstname.lastname@example.org|
|Principal Investigator: Amy Rosenfeld, MD|
|United States, Michigan|
|Helen DeVos Children's Hospital||Recruiting|
|Grand Rapids, Michigan, United States, 49503|
|Contact: Shannon Mackeigan 616-267-1162 email@example.com|
|Principal Investigator: Albert Cornelius, MD|
|Principal Investigator: Giselle Sholler, MD|
|Study Chair:||Giselle Sholler, MD||The Spectrum Health Group|
|Principal Investigator:||Albert Cornelius, MD||Helen DeVos Children's Hospital|