Vemurafenib for Pediatric Brain Cancer

Recruiting in Palo Alto (17 mi)

+20 other locations

Overseen by Sabine Mueller, MD, PhD, MAS

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase < 1

Waitlist Available

Sponsor: University of California, San Francisco

No Placebo Group

Breakthrough Therapy

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

This is a multicenter, safety and pharmacokinetic trial to determine the MTD and/or select a recommended phase 2 dose (RP2D) of vemurafenib in children with recurrent or refractory gliomas containing the BRAFV600E or BRAF Ins T mutation.

Eligibility Criteria

This trial is for children with recurrent or refractory gliomas that have a specific BRAFV600E mutation. They must be able to swallow tablets, not have had certain treatments recently, and their organs need to function well. Kids under 18 are eligible for the safety study, while those up to 25 can join other parts of the trial if they meet additional criteria.

Inclusion Criteria

Cardiac: Corrected QT (QTc) interval <450 msec on EKG

I have had all suspicious skin lesions removed.

Signed informed consent according to institutional guidelines must be obtained

Exclusion Criteria

History of allergic reactions attributed to compounds of similar chemical or biologic composition to vemurafenib

I am not on any other cancer treatments or experimental drugs.

I do not have uncontrolled seizures.

Participant Groups

The trial is testing Vemurafenib's safety and proper dosing in kids with brain tumors that came back or didn't respond to treatment. It aims to find the maximum tolerable dose (MTD) and/or recommend a phase 2 dose (RP2D). The drug concentration in tumor tissue will also guide eligibility for later phases.

Group I: Vemurafenib Experimental Treatment 1 Intervention

Vemurafenib should be swallowed whole with 8 oz (1 cup) of water. Pharmacokinetic studies will determine if vemurafenib can be "crushed". If patients receiving "crushed" tablets are felt to receive adequate exposure, then they will be allowed to participate in the expansion cohort. \[Patients approved to take "crushed" tablets should use a pill crusher and mix pill with 3-5 ml apple sauce\]. If not, then only patients able to swallow whole pills will be eligible. The patient will be requested to maintain a medication diary of each dose of medication. The medication diary will be returned to clinic staff at the end of each cycle.

Vemurafenib is already approved in United States, European Union, Canada for the following indications:

🇺🇸 Approved in United States as Zelboraf for:

• Unresectable or metastatic melanoma with BRAF V600E mutation
• Erdheim-Chester Disease (ECD) with BRAF V600 mutation

🇪🇺 Approved in European Union as Zelboraf for:

• Adults with BRAF V600E mutation positive unresectable or metastatic melanoma

🇨🇦 Approved in Canada as Zelboraf for:

• Unresectable or metastatic melanoma with BRAF V600E mutation

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

UCSF Medical Center-Mount Zion San Francisco, CA

Rady Children's Hospital - San Diego San Diego, CA

UCSF Medical Center-Parnassus San Francisco, CA

University of Florida Health Science Center - Gainesville Gainesville, FL

More Trial Locations

Who Is Running the Clinical Trial?

University of California, San Francisco Lead Sponsor

Genentech, Inc. Industry Sponsor

References