Title: 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

Study Director: Blueprint Medicines Corporation

Description: Researchers are conducting a phase 1/2 trial of avapritinib in pediatric patients who have relapsed or refractory solid tumors with mutations in KIT or PDGFRA or who have H3K27M-mutant glioma.

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In the first part of the study, researchers will determine the recommended phase 2 dose. In the second part, researchers will assess the antitumor activity, safety, and pharmacokinetics of the recommended dose.

In both phases, patients will receive oral avapritinib daily in 28-day cycles. The primary outcome measures are the recommended phase 2 dose and the overall response rate.

This trial is enrolling patients who are 2 to 17 years of age. Eligible patients will have a relapsed/refractory solid tumor or central nervous system tumor, with a mutation in KIT or PDGFRA, that has progressed despite standard therapy and for which there is no alternative treatment option. 

Patients are also eligible if they have H3K27M-mutant glioma that has failed standard therapy or for which there is no standard therapy that may provide a clinical benefit. For a full list of eligibility criteria, please see the reference. 

Status: Recruiting

This study is sponsored by Blueprint Medicines Corporation.


ClinicalTrials.gov. A study of avapritinib in pediatric patients with solid tumors dependent on KIT or PDGFRA signaling. NCT04773782. Accessed July 20, 2022.