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)
Name: Avapritinib
Description: 0 - 80 points (higher value represents worse symptom outcomes)
Measure: Mean Change from Baseline in Advanced Systemic Mastocytosis-Symptom Assessment Form (AdvSM-SAF) Total Symptom Score Time: 10 MonthsDescription: Months
Measure: Time-to-response (TTR) Time: 10 MonthsDescription: Months
Measure: Duration of Response (DOR) Time: 10 MonthsDescription: Months
Measure: Progression-free Survival (PFS) Time: 10 MonthsDescription: Months
Measure: Overall Survival (OS) Time: 10 MonthsDescription: percentage
Measure: Changes in bone marrow mast cells Time: 10 MonthsDescription: ng/mL
Measure: Change in serum tryptase Time: 10 MonthsDescription: percentage
Measure: Change in V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) mutation burden Time: 10 MonthsDescription: mL
Measure: Change in liver volume by imaging Time: 10 MonthsDescription: mL
Measure: Change in spleen volume by imaging Time: 10 MonthsDescription: 0 - 10 points (higher value represents worse symptom outcomes)
Measure: Change in PGIS Time: 10 MonthsDescription: 0 - 100 points (lower value represents worse quality of life)
Measure: Change in EORTC QLQ-C30 Time: 10 MonthsDescription: CTCAE version 4.0
Measure: Safety of Avapritinib as assessed by incidence of adverse events Time: 10 MonthsDescription: h•ng/mL
Measure: Area Under Curve (0 to Tau) for Avapritinib Time: 4 MonthsAllocation: N/A
Single Group Assignment
There is one SNP
Change in V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) mutation burden. --- D816V ---