Avapritinib for Systemic Mastocytosis
Trial Summary
Will I have to stop taking my current medications?
The trial does not specify if you must stop all current medications, but it does require that you have not taken certain treatments like cytoreductive therapy or investigational agents for a specific period before starting the trial. If you are on corticosteroids, your dose must be stable for at least 14 days.
What data supports the effectiveness of the drug Avapritinib for treating systemic mastocytosis?
How is the drug Avapritinib unique in treating systemic mastocytosis?
Avapritinib is unique because it is a highly selective inhibitor of the KIT D816V mutation, which is present in most patients with advanced systemic mastocytosis. It has shown high response rates and can lead to complete remission, making it a potent option for patients who have undergone previous treatments.12467
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
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Part 1 Treatment
Participants receive avapritinib or placebo in continuous 28-day cycles to determine optimal dosing
Part 2 Treatment
Participants receive avapritinib or placebo in continuous 28-day cycles to assess efficacy and safety
Part 3 Treatment
All participants receive avapritinib in continuous 28-day cycles
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Avapritinib
- Placebo
Avapritinib is already approved in United States, European Union for the following indications:
- 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
- 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
Who Is Running the Clinical Trial?
Blueprint Medicines Corporation
Lead Sponsor