(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Status:
Recruiting
Trial end date:
2028-06-01
Target enrollment:
Participant gender:
Summary
This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the
efficacy and safety of BLU-263 + best supportive care (BSC) with placebo + BSC in patients
with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by
BSC. The study will be conducted in 4 parts. Parts 1 and 2 will enroll patients with ISM.
Patients enrolled in Part 1 or Part 2 will roll over onto Part 3 to receive treatment with
BLU-263 in an open-label fashion following completion of the earlier Part. Part M will enroll
patients with monoclonal mast cell activation syndrome (mMCAS). The study also includes
optional PK groups that may enroll patients with ISM.