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

Official Title

A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumour Activity of Avapritinib in Pediatric Patients With Solid Tumours Dependent on KIT or PDGFRA Signaling

Summary:

This is a Phase 1/2, multicentre, open-label trial of avapritinib in patients aged 2 to less than 18 years of age with with relapsed/refractory (R/R) solid tumours 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).

Trial Description

Primary Outcome:

  • Determination of recommended Part 2 dose (Part 1)
  • Overall Response Rate (Part 2)
Secondary Outcome:
  • Rate and severity of adverse events (Part 1 and Part 2)
  • Overall Response Rate (Part 2)
  • Duration of Response (Part 2)
  • Progression-free survival (Part 2)
  • Disease control rate (Part 2)
  • Cmax (Part 1 and Part 2)
  • AUC(0-24) (Part 1 and Part 2)
  • Tmax (Part 1)
  • T 1/2 (Part 1)
  • Ctrough (Part 2)

View this trial on ClinicalTrials.gov

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Resources

Canadian Cancer Society

These resources are provided in partnership with the Canadian Cancer Society