251 Participants Needed

Avapritinib for Systemic Mastocytosis

Recruiting at 48 trial locations
BM
Overseen ByBlueprint Medicines
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 2 JurisdictionsThis treatment is already approved in other countries

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?

Research shows that Avapritinib is effective in treating advanced systemic mastocytosis, with studies reporting an overall response rate of 71-75% and significant reductions in disease markers. The drug was generally well tolerated, with most side effects being mild to moderate.12345

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

My current treatment hasn't relieved my symptoms.
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.
See 2 more

Exclusion Criteria

I haven't had radiotherapy or PUVA therapy in the last 14 days.
I have never been treated with avapritinib.
I haven't had specific cancer treatments in the required time before the trial starts.
See 3 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Part 1 Treatment

Participants receive avapritinib or placebo in continuous 28-day cycles to determine optimal dosing

6 months
Monthly visits (in-person)

Part 2 Treatment

Participants receive avapritinib or placebo in continuous 28-day cycles to assess efficacy and safety

6 months
Monthly visits (in-person)

Part 3 Treatment

All participants receive avapritinib in continuous 28-day cycles

6 months
Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 5 years

Treatment Details

Interventions

  • Avapritinib
  • Placebo
Trial Overview 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.
Participant Groups
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

Who Is Running the Clinical Trial?

Blueprint Medicines Corporation

Lead Sponsor

Trials
31
Recruited
6,000+

Findings from Research

In a phase I trial of avapritinib for advanced systemic mastocytosis, 72% of patients showed a positive response, with 56% achieving complete or partial responses, indicating strong efficacy.
The treatment was well-tolerated, with no patients stopping due to adverse events, which were mostly mild to moderate, suggesting a favorable safety profile.
Rapid Responses to Avapritinib (BLU-285) in Mastocytosis.[2019]
Avapritinib, a selective oral inhibitor targeting the mutated tyrosine kinase in systemic mastocytosis, has shown promising safety and efficacy in early Phase I and II trials, even for patients who did not respond to previous therapies.
Current studies are primarily focused on patients with aggressive forms of systemic mastocytosis, with new trials beginning for those with indolent forms, indicating a potential expansion of treatment options in the future.
Avapritinib in the Treatment of Systemic Mastocytosis: an Update.Below, S., Michaelis, LC.[2021]
Avapritinib, a selective inhibitor for the KIT D816V mutation, showed a high overall response rate of 71% in patients with advanced systemic mastocytosis (AdvSM) who had previously received systemic therapy, with a median time to response of 2.3 months.
The treatment was generally well tolerated, with 94% of patients experiencing treatment-related adverse events, mostly mild (grade 1/2), and 81% remaining on treatment after 6 months, indicating its safety profile in this patient population.
Efficacy and safety of avapritinib in previously treated patients with advanced systemic mastocytosis.Reiter, A., Schwaab, J., DeAngelo, DJ., et al.[2022]

References

Rapid Responses to Avapritinib (BLU-285) in Mastocytosis. [2019]
Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial. [2022]
Avapritinib in the Treatment of Systemic Mastocytosis: an Update. [2021]
Efficacy and safety of avapritinib in previously treated patients with advanced systemic mastocytosis. [2022]
Safety and efficacy of avapritinib in advanced systemic mastocytosis: the phase 1 EXPLORER trial. [2022]
The clinical experience of compassionate use program for avapritinib: implications for drug positioning in the therapeutic scenario of systemic mastocytosis. [2023]
Avapritinib for advanced systemic mastocytosis. [2023]
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