(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Recruiting
Phase 2
Phase 3
Interventional Study
Indolent Systemic Mastocytosis
Smoldering Systemic Mastocytosis
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 and older
- Sex
- Any
- Trial phase
- Phase 2/3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 534 (estimated)
- Sponsor
- Blueprint Medicines Corporation · Industry
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About This Trial
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 …
Trial Locations
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Eligibility Criteria
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 adequ…
Contacts