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

NCT04773782 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 29

Last updated 2026-01-13

No results posted yet for this study

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

DRUG

avapritinib

oral administration

Sponsors & Collaborators

  • Blueprint Medicines Corporation

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-02-24
Primary Completion
2025-11-21
Completion
2025-11-21
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Austria
  • Canada
  • France
  • Germany
  • Italy
  • South Korea
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04773782 on ClinicalTrials.gov