NCT03580655

(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis

An Open-label, Single Arm, Phase 2 Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 107 (actual)
• Sponsor: Blueprint Medicines Corporation · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, single arm, Phase 2 study evaluating the efficacy and safety of avapritinib (BLU-285) in patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive SM (ASM), SM with associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL)

Conditions

• Advanced Systemic Mastocytosis
• Aggressive Systemic Mastocytosis
• Systemic Mastocytosis With an Associated Hematologic Neoplasm
• Mast Cell Leukemia

Interventions

Type	Name	Description
DRUG	Avapritinib	Avapritinib tablet

Timeline

• Start date: 2018-11-21
• Primary completion: 2024-12-18
• Completion: 2024-12-18
• First posted: 2018-07-09
• Last updated: 2025-04-30

Locations

32 sites across 12 countries: United States, Austria, Canada, Denmark, France, Germany, Italy, Netherlands, Norway, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study