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A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard Therapy

Trial Profile

A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard Therapy

Status: Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 27 Mar 2024

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At a glance

  • Drugs Avapritinib (Primary)
  • Indications Systemic mastocytosis
  • Focus Adverse reactions; Proof of concept; Registrational; Therapeutic Use
  • Acronyms PIONEER
  • Sponsors Blueprint Medicines
  • Most Recent Events

    • 26 Feb 2024 Results assessing disease burden and prognostic tools that predict overall survival (OS) in patients with ISM presented at the 2024 Annual Meeting of the American Academy of Allergy, Asthma and Immunology
    • 26 Feb 2024 Results assessing Reductions in Polypharmacy for Patients with Indolent Systemic Mastocytosis on Avapritinib presented at the 2024 Annual Meeting of the American Academy of Allergy, Asthma and Immunology
    • 26 Feb 2024 Results assessing biomarkers of disease burden and improved symptoms versus placebo presented at the 2024 Annual Meeting of the American Academy of Allergy, Asthma and Immunology
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