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Clinical Trial 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).


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT04773782
Study type Interventional
Source Blueprint Medicines Corporation
Contact Blueprint Medicines
Phone 617-714-6707
Email medinfo@blueprintmedicines.com
Status Recruiting
Phase Phase 1/Phase 2
Start date February 24, 2022
Completion date April 30, 2025

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