Avapritinib for Pediatric Solid Cancers

Recruiting in Palo Alto (17 mi)

+25 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Blueprint Medicines Corporation

No Placebo Group

Breakthrough Therapy

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

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).

Research Team

Eligibility Criteria

This trial is for children and teenagers aged 2 to less than 18 with advanced solid tumors, including brain tumors that have specific genetic changes (mutations in KIT or PDGFRA) or a type of glioma called DMG-H3K27a. They should have tried standard treatments without success and not be eligible for other curative options. Participants need to be stable on certain medications, able to perform daily activities at least minimally, and agree to use contraception.

Inclusion Criteria

Participant agrees to utilize contraception consistent with local regulations.

My brain disease treatment with steroids has been stable or reduced for a week.

My diagnosis is DMG-H3K27a and standard treatments have not worked or are not available.

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Exclusion Criteria

My platelet count is low and I haven't had a transfusion in the last 14 days.

Participant has International normalized ratio or prothrombin time (PT) > ULN.

My liver enzymes are high, but not over five times the normal limit if my cancer affects my liver.

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Treatment Details

Interventions

Avapritinib (Tyrosine Kinase Inhibitor)

Trial OverviewThe study tests avapritinib, a medication targeting cancers with certain genetic mutations. It's an open-label trial where all participants receive the drug. The first part confirms the dose and assesses safety; the second part looks at how effective it is at a recommended dose while continuing safety assessments.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: avapritinibExperimental Treatment1 Intervention

Avapritinib tablets for oral administration. Avapritinib will be dosed daily for 28 day cycles.