Avapritinib in Tumors with KIT or PDGFRA alterations or Glioma with H3K27M mutations (BLU-285-3101)
A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients with Solid Tumors Dependent on KIT or PDGFRA Signaling (BLU-285-3101)
Recruiting
Summary
This is a Phase 1/2, multicenter, open-label trial of avapritinib in patients aged 2 to less than 18 years of age with with relapsed/refractory (R/R) tumors with mutations (including non-synonymous point mutations, insertions, and deletions) in KIT or PDGFRA, or gliomas with the H3K27M mutation, and no available alternative treatment options. This is a single-arm trial in which all patients will receive avapritinib. The study consists of 2 parts: dose confirmation, safety, and PK (Part 1) and initial efficacy, safety, and PK at the Part 2 recommended dose (Part 2).
Avapritinib tablets are administered orally daily for 28 day cycles.
Main inclusion criteria
- Recurrent or refractory tumor with a in KIT or PDGFRA or H3K27M mutant glioma
- Age: 2-18 years
- at least one measurable lesion
- Karnofsky oder Lansky > 50
Sponsor
Blueprint Medicines Corporation