NCT04773782

A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: Blueprint Medicines Corporation · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 1/2, multicenter, open-label trial of avapritinib in participants 2 to \< 18 years of age with advanced relapsed/refractory (R/R) solid tumors, including central nervous system (CNS) tumors, that harbor a PDGFRA and/or KIT mutation (including non-synonymous point mutations, insertions, and deletions) or amplification, or DMG-H3K27a who have no available curative treatment options. This is a single-arm trial in which all participants 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).

Conditions

• Solid Tumor, Unspecified, Child
• Relapsed Solid Neoplasm
• CNS Tumor

Interventions

Type	Name	Description
DRUG	avapritinib	oral administration

Timeline

• Start date: 2022-02-24
• Primary completion: 2025-11-21
• Completion: 2025-11-21
• First posted: 2021-02-26
• Last updated: 2026-01-13

Locations

26 sites across 9 countries: United States, Australia, Austria, Canada, France, Germany, Italy, South Korea, United Kingdom

Regulatory

• FDA-regulated drug study