(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

Official Title

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

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)

Trial Description

Primary Outcome:

• Objective response rate (ORR) based on modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (IWG-MRT-ECNM) response criteria

• Mean Change from Baseline in Advanced Systemic Mastocytosis-Symptom Assessment Form (AdvSM-SAF) Total Symptom Score
• Time-to-response (TTR)
• Duration of Response (DOR)
• Progression-free Survival (PFS)
• Overall Survival (OS)
• Changes in bone marrow mast cells
• Change in serum tryptase
• Change in V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) mutation burden
• Change in liver volume by imaging
• Change in spleen volume by imaging
• Clinical benefit based on modified IWG-MRT-ECNM consensus criteria
• Change in PGIS
• Change in EORTC QLQ-C30
• Safety of Avapritinib as assessed by incidence of adverse events
• Area Under Curve (0 to Tau) for Avapritinib

View this trial on ClinicalTrials.gov