Avapritinib for Systemic Mastocytosis

Recruiting at 31 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Blueprint Medicines Corporation

Must not be taking: Midostaurin, TKIs, Hydroxyurea, others

Disqualifiers: Eosinophilia, Primary malignancy, Seizure disorder, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 10 months

107 Participants Needed

Trial Summary

What is the purpose of this trial?

This is an open-label, single arm, Phase 2 study evaluating the efficacy and safety of avapritinib (BLU-285) in patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive SM (ASM), SM with associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL)

Eligibility Criteria

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.

Inclusion Criteria

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

Your blood test must show a level of tryptase higher than 20 ng/mL.

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

Exclusion Criteria

My kidney function is significantly reduced.

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.

I have high eosinophil levels and a positive FIP1L1-PDGFRA test, or no prior imatinib treatment failure.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive avapritinib (BLU-285) as an immediate release tablet, orally, continuously, in 28-day cycles

10 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term follow-up

Participants are monitored for progression-free survival and overall survival

Approximately 4 years

Treatment Details

Interventions

• Avapritinib

Trial Overview 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.

Participant Groups

Group I: Avapritinib Experimental Treatment 1 Intervention

Avapritinib will be administered as an immediate release tablet, orally, continuously, in 28-day cycles

Avapritinib is already approved in United States, European Union for the following indications:

Approved in United States as Ayvakit for:

• Unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations
• Advanced systemic mastocytosis
• Indolent systemic mastocytosis

Approved in European Union as Ayvakit for:

• Unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations
• Advanced systemic mastocytosis
• Indolent systemic mastocytosis

Who Is Running the Clinical Trial?

Blueprint Medicines Corporation

Lead Sponsor

Trials

31

Recruited

6,000+