A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis
Blueprint Medicines Corporation
Summary
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.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: All Participants: -Participant must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2. Part 1 and PK groups: * Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review * Participant must have failed to achieve adequate symptom control for 1 or more Baseline symptoms, as determined by the Investigator, with at least 2 of the following symptom-directed therapies administered: H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab. * Participants…
Interventions
- DrugElenestinib
Elenestinib oral tablet
- DrugPlacebo
Placebo oral tablet
Locations (64)
- University of Alabama at BirminghamBirmingham, Alabama
- David Geffen School of Medicine at UCLALos Angeles, California
- Stanford Cancer InstitutePalo Alto, California
- UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical CampusAurora, Colorado
- Winship Cancer Institute, Emory UniversityAtlanta, Georgia
- Brigham and Women's HospitalBoston, Massachusetts