RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2021-11-30
Study Completion:2032-09-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04910685

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.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review
  2. * 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.
  3. * Participants must have SDT for ISM symptom management stabilized for at least 14 days prior to starting screening procedures.
  4. * For participants receiving corticosteroids, the dose must be ≤ 20 mg/day prednisone or equivalent, and the dose must be stable for ≥ 14 days.
  1. * 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.
  2. * Participant has been diagnosed with another myeloproliferative disorder.
  3. * Participant has organ damage attributable to SM.
  4. * Participant has clinically significant, uncontrolled, cardiovascular disease
  5. * Participant has a QT interval corrected using Fridericia's formula (QTcF) \> \> 470 milliseconds (msec) (for females) or \> 450 msec (for males).
  6. * 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.
  7. * 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.
  8. * Participant has received radiotherapy or psoralen and ultraviolet A (PUVA) therapy \< 14 days before beginning the screening period.

Contacts and Locations

Study Contact

Blueprint Medicines
CONTACT
617-714-6707
medinfo@blueprintmedicines.com

Study Locations (Sites)

University of Alabama at Birmingham
Birmingham, Alabama, 35294
United States
Stanford Cancer Institute
Palo Alto, California, 94305
United States
Brigham and Women's Hospital
Boston, Massachusetts, 02115
United States
Michigan Medicine University of Michigan
Ann Arbor, Michigan, 48109
United States
Mayo Clinic
Rochester, Minnesota, 55902
United States
Roswell Park Cancer Institute
Buffalo, New York, 14263
United States
Columbia University Medical Center
New York, New York, 10032
United States
University of Cincinnati Medical Center
Cincinnati, Ohio, 45219
United States
The University of Texas, MD Anderson Cancer Center
Houston, Texas, 77030
United States
Huntsman Cancer Institute, University of Utah
Salt Lake City, Utah, 84112
United States

Collaborators and Investigators

Sponsor: Blueprint Medicines Corporation

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-11-30
Study Completion Date2032-09-30

Study Record Updates

Study Start Date2021-11-30
Study Completion Date2032-09-30

Terms related to this study

Keywords Provided by Researchers

  • ISM
  • SSM

Additional Relevant MeSH Terms

  • Indolent Systemic Mastocytosis
  • Smoldering Systemic Mastocytosis