This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by SDT. Parts 1 and 2 will enroll participants with ISM. Participants enrolled in Part 2 will roll over onto Part 3 to receive treatment with elenestinib in an open-label fashion following completion of the earlier Part. Part K will enroll participants with ISM who have previously received an approved selective KIT inhibitor. The study also includes pharmacokinetic (PK) groups that will enroll participants with ISM.
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Part 1: Number of participants with Treatment-emergent Adverse Events (TEAEs)
Timeframe: Up to 12 weeks
Part 1: Mean change from baseline in ISM-Symptom in Assessment Form (ISM-SAF) Total Symptom Score (TSS)
Timeframe: Baseline, Week 13
Part 2: Mean change from baseline in ISM-SAF TSS
Timeframe: Baseline, Week 49
Part 3: Number of participants with Adverse Events (AEs)
Timeframe: Up to 5 years
Part 3: Change from baseline in ISM-SAF TSS
Timeframe: Baseline up to 5 years