(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

Description

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.

Conditions

Indolent Systemic Mastocytosis, Smoldering Systemic Mastocytosis

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Study Overview

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Study Details

Study overview

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.

A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis

(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

Condition
Indolent Systemic Mastocytosis
Intervention / Treatment

-

Contacts and Locations

Birmingham

University of Alabama at Birmingham, Birmingham, Alabama, United States, 35294

Palo Alto

Stanford Cancer Institute, Palo Alto, California, United States, 94305

Boston

Brigham and Women's Hospital, Boston, Massachusetts, United States, 02115

Ann Arbor

Michigan Medicine University of Michigan, Ann Arbor, Michigan, United States, 48109

Rochester

Mayo Clinic, Rochester, Minnesota, United States, 55902

Buffalo

Roswell Park Cancer Institute, Buffalo, New York, United States, 14263

New York

Columbia University Medical Center, New York, New York, United States, 10032

Cincinnati

University of Cincinnati Medical Center, Cincinnati, Ohio, United States, 45219

Houston

The University of Texas, MD Anderson Cancer Center, Houston, Texas, United States, 77030

Salt Lake City

Huntsman Cancer Institute, University of Utah, Salt Lake City, Utah, United States, 84112

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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.
  • * 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.

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Blueprint Medicines Corporation,

Study Record Dates

2032-09-30