Evaluating BLU-263 for Indolent Systemic Mastocytosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study is a randomized, double-blind, placebo-controlled trial assessing the efficacy and safety of elenestinib (BLU-263) in patients with indolent systemic mastocytosis (ISM) who have not achieved adequate symptom control with standard therapies. Participants will be enrolled in multiple parts, with those in Part 2 rolling over to receive open-label treatment in Part 3. The study also includes pharmacokinetic groups for further analysis of the drug's effects. The goal is to determine if elenestinib can provide better symptom management compared to placebo.

Why it matters

Who can join: the full rules

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 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 Criteria:

* 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.

Where it's running

• University of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting
• Orso Health, Modena Allergy and Asthma Inc
  La Jolla, California, United States Recruiting
• David Geffen School of Medicine at UCLA
  Los Angeles, California, United States Recruiting
• Stanford Cancer Institute
  Palo Alto, California, United States Recruiting
• UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus
  Aurora, Colorado, United States Recruiting
• Winship Cancer Institute, Emory University
  Atlanta, Georgia, United States Recruiting

+38 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Blueprint Medicines
• Email: medinfo@blueprintmedicines.com
• Phone: 617-714-6707

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Real-world care and outcomes for people with indolent systemic mastocytosis (ISM)

  This study will see how people with ISM are treated and how they are doing in everyday care, including those taking avapritinib.

  Recruiting at 7 sites Blueprint Medicines Corporation · Aurora, Colorado and 6 other locations · NCT07264959

• Observing avapritinib treatment in people with indolent systemic mastocytosis in Germany

  This study will see if avapritinib helps manage symptoms in people with indolent systemic mastocytosis who are starting the drug as part of routine care in Germany.

  Recruiting at 12 sites Blueprint Medicines Corporation · Berlin and 11 other locations · NCT07255638

• Angioedema biomarker tests to help diagnose recurrent swelling

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  Recruiting Foundation For Rare Disease Research · Wheaton, Maryland · NCT06210698

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.