Avapritinib for Systemic Mastocytosis
Palo Alto (17 mi)Age: 18+
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Waitlist Available
Sponsor: Blueprint Medicines Corporation
Prior Safety Data
Breakthrough Therapy
Approved in 2 jurisdictions
Trial Summary
What is the purpose of this trial?This trial is testing a new medication called avapritinib combined with the best possible care for patients with a condition called indolent systemic mastocytosis (ISM). These patients have symptoms that are not well managed by their current treatments. Avapritinib works by blocking certain proteins that cause abnormal cell growth and activity, aiming to improve symptom control.
Eligibility Criteria
This trial is for patients with indolent systemic mastocytosis (ISM) who haven't found relief from their symptoms through standard care. They must have a specific symptom score, confirmed ISM by pathology review, and be able to perform daily activities with minimal assistance. People can't join if they've had certain treatments recently, other SM subtypes, prior avapritinib use, or specific heart rhythm issues.Inclusion Criteria
I am on a stable dose of corticosteroids, not exceeding 20 mg/d prednisone or its equivalent, for at least 14 days.
My systemic mastocytosis was confirmed by a specialized lab review.
My symptoms are moderate to severe based on a recent symptom score.
I am able to care for myself and perform daily activities.
Exclusion Criteria
I have never been treated with avapritinib.
I have been diagnosed with a specific type of mast cell disorder.
Treatment Details
The study tests Avapritinib's effectiveness and safety against a placebo in ISM patients whose symptoms persist despite best supportive care. It's randomized and double-blind: neither doctors nor participants know who gets the real drug or placebo until the study ends. Everyone eventually receives Avapritinib during part three of the trial.
7Treatment groups
Experimental Treatment
Placebo Group
Group I: (Part 3) Avapritinib RP2D + BSCExperimental Treatment1 Intervention
Avapritinib will be administered orally in continuous 28-day cycles
Group II: (Part 2) Avapritinib RP2D + BSCExperimental Treatment1 Intervention
Avapritinib will be administered orally in continuous 28-day cycles
Group III: (Part 1) Avapritinib Dose 3 + BSCExperimental Treatment1 Intervention
Avapritinib will be administered orally in continuous 28-day cycles
Group IV: (Part 1) Avapritinib Dose 2 + BSCExperimental Treatment1 Intervention
Avapritinib will be administered orally in continuous 28-day cycles
Group V: (Part 1) Avapritinib Dose 1 + BSCExperimental Treatment1 Intervention
Avapritinib will be administered orally in continuous 28-day cycles
Group VI: (Part 2) Placebo + BSCPlacebo Group1 Intervention
Placebo will be administered orally in continuous 28-day cycles
Group VII: (Part 1) Placebo + BSCPlacebo Group1 Intervention
Placebo will be administered orally in continuous 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
Find a clinic near you
Research locations nearbySelect from list below to view details:
Tom Baker Cancer CentreCalgary, Canada
St. Michael's HospitalToronto, Canada
University of Alberta HospitalEdmonton, Canada
Virginia Commonwealth University Medical CenterRichmond, VA
More Trial Locations
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Who is running the clinical trial?
Blueprint Medicines CorporationLead Sponsor