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

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There are two parts planned for this study, the Phase 1 that will test different doses of avapritinib to identify the recommended dose of avapritinib for the Phase 2. The Phase 2 will continue to test this recommended dose of avapritinib to determine if it is a tolerable, safe, and effective treatment for subjects with solid tumors dependent on KIT or PDGFRA function. The avapritinib dose should be administered once every day, at approximately the same time each day, ideally in the morning. Generally, it is expected that subjects will participate for minimum duration of approximately 3 months but there is no defined maximum duration.




- Patient must be 2 to <18 years of age at the time of signing the informed consent. - Diagnosis: a. Patient has confirmed diagnosis of a R/R solid tumor with a mutation (including non-synonymous point mutations, insertions, and deletions) in KIT or PDGFRA (confirmed by local mutational testing of tumor sample) that has progressed despite standard therapy and no alternative treatment option is available. OR b. Patient has confirmed diagnosis of H3K27M mutant glioma that has failed standard therapy or for which no standard therapy that may convey clinical benefit exists, as judged by the investigator. - Disease extent: All patients must have at least 1 measurable lesion as defined by RECIST v1.1 or RANO (for CNS tumors).

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Type of Study



Childrens Hospital Colorado

Principal Investigator
Photograph of Kathleen Dorris,  MD

Kathleen Dorris, MD

Study ID

Protocol Number: 21-4262

More information available at ClinicalTrials.gov: NCT04773782


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