Avapritinib for Systemic Mastocytosis

Phase 2

Waitlist Available

Research Sponsored by Blueprint Medicines Corporation

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patient must have a diagnosis of aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) or mast cell leukemia (MCL) based on World Health Organization diagnostic criteria. Before enrollment, the Study Steering Committee must confirm the diagnosis of AdvSM (based on Central Pathology Laboratory assessment of bone marrow).

Patient must have Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 3.

Must not have

Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m2 or creatinine > 1.5 × ULN.

Patient has received any cytoreductive therapy (including midostaurin and other TKIs, hydroxyurea, azacitidine) or an investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon and any antibody therapy (eg, brentuximab vedotin) less than 28 days before obtaining screening BM biopsy for this study.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up approximately 4 years after the first subjected enrolled

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug, avapritinib, for patients with advanced mastocytosis, which is a rare disease where mast cells accumulate in the body.

Who is the study for?

This trial is for adults with advanced systemic mastocytosis, including aggressive forms and mast cell leukemia. Participants must have a serum tryptase level of at least 20 ng/mL and an ECOG performance status of 0-3. They should not have had recent cytoreductive therapy or certain other treatments, nor should they have severe kidney issues, seizure disorders, brain cancer, low platelet counts, a history of intracranial bleeding, abnormal heart rhythm or liver enzymes due to causes other than mastocytosis.

What is being tested?

The PATHFINDER study is testing the effectiveness and safety of Avapritinib (BLU-285), which targets specific mutations in tyrosine kinase involved in systemic mastocytosis. It's an open-label Phase 2 trial where all participants receive the drug without being compared to another treatment or placebo.

What are the potential side effects?

Potential side effects from Avapritinib may include nausea, vomiting, swelling around the eyes or on the skin (periorbital edema), fatigue, muscle pain (myalgia), cognitive changes like memory problems or confusion (cognitive effects), hair color changes and fluid retention.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with a severe form of mastocytosis confirmed by a specialized committee.

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I can care for myself but may not be able to do active work.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My kidney function is significantly reduced.

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I haven't taken any cancer-reducing drugs or experimental treatments within the last 14 days, or specific drugs like cladribine or antibody therapies within the last 28 days.

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I have epilepsy or need medication to prevent seizures.

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My platelet count is below 50,000 or I need platelet transfusions.

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I have a brain tumor or cancer that has spread to my brain.

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I have been treated with avapritinib before.