Summary

Eligibility
for people ages up to 25 years (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
completion around

Description

Summary

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.

Official Title

Pediatric Neuro-Oncology Consortium (PNOC)-002: Safety, Phase 0, and Pilot Efficacy Study of Vemurafenib, an Oral Inhibitor of BRAFV600E, in Children and Young Adults With Recurrent/Refractory BRAFV600E- or BRAF Ins T Mutant Brain Tumors

Details

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. Using the RP2D, the study team will then conduct a Phase 0 study in a pre-surgical cohort of 10 patients requiring debulking surgery at the time of recurrence. These patients will receive neo-adjuvant vemurafenib, thus allowing the study team to measure intra-tumoral drug concentrations and target inhibition. An expansion cohort will then be enrolled to allow the study team to preliminarily estimate efficacy. Participants are followed up for adverse events while receiving study treatment until resolution.

Keywords

Pediatric Recurrent/Refractory BRAFV600E-mutant Gliomas, recurrent/refractory BRAFV600E-mutant gliomas, pediatrics, children, neo-adjuvant vemurafenib, Glioma, Recurrence, Vemurafenib

Eligibility

Locations

  • Mattel Children's Hospital UCLA
    Los Angeles California 90095 United States
  • Children's Hospital Los Angeles
    Los Angeles California 90027 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
University of California, San Francisco
ID
NCT01748149
Phase
Phase 1 research study
Study Type
Interventional
Participants
About 40 people participating
Last Updated