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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

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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

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

Latest Information Update: 09 Aug 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

    • 30 May 2024 According to Blueprint Medicines media release, data from this trial will be presented at European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024.
    • 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

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