Status:
RECRUITING
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Lead Sponsor:
Blueprint Medicines Corporation
Conditions:
Indolent Systemic Mastocytosis
Smoldering Systemic Mastocytosis
Eligibility:
All Genders
18+ years
Phase:
PHASE2
PHASE3
Brief 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 w...
Eligibility Criteria
Inclusion
- Key
- 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 must have SDT for ISM symptom management stabilized for at least 14 days prior to starting screening procedures.
- For participants receiving corticosteroids, the dose must be ≤ 20 mg/day prednisone or equivalent, and the dose must be stable for ≥ 14 days.
- Part K:
- Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review
- Part S:
- Participant has confirmed diagnosis of SSM, confirmed by Central Pathology Review of BM biopsy and central review of B- and C-findings by WHO diagnostic criteria.
- Part 2:
- Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review
- Key
Exclusion
- Participant has been diagnosed with any of the following WHO systemic mastocytosis (SM) sub-classifications: cutaneous mastocytosis only, SM with an associated hematologic neoplasm of non-MC lineage (SM-AHN), aggressive SM, mast cell leukemia, or mast cell sarcoma.
- Participant has been diagnosed with another myeloproliferative disorder.
- Participant has organ damage attributable to SM.
- Participant has clinically significant, uncontrolled, cardiovascular disease
- Participant has a QT interval corrected using Fridericia's formula (QTcF) \> \> 470 milliseconds (msec) (for females) or \> 450 msec (for males).
- Participant has a history of a primary malignancy that has been diagnosed or required therapy within 3 years. The following prior malignancies are not exclusionary: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site.
- Time since any cytoreductive therapy including masitinib and midostaurin should be at least 5 half-lives or 14 days (whichever is longer), and for cladribine, interferon alpha, pegylated interferon, or antibody therapy \< 28 days or 5 half-lives of the drug (whichever is longer), before beginning the screening period.
- Participant has received radiotherapy or psoralen and ultraviolet A (PUVA) therapy \< 14 days before beginning the screening period.
- Other protocol-defined criteria apply.
Key Trial Info
Start Date :
November 30 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 30 2032
Estimated Enrollment :
534 Patients enrolled
Trial Details
Trial ID
NCT04910685
Start Date
November 30 2021
End Date
September 30 2032
Last Update
November 3 2025
Active Locations (54)
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1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
2
Stanford Cancer Institute
Palo Alto, California, United States, 94305
3
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
4
Michigan Medicine University of Michigan
Ann Arbor, Michigan, United States, 48109