This is a Phase 4, non-interventional, observational study to collect detailed data on the clinical characteristics, clinical outcomes and medical management of ISM in real-world settings. The study will describe the demographic and clinical characteristics of ISM participants, including anaphylaxis and bone manifestations in ISM. Quality of life and disease control will be assessed through participant questionnaires. The study will also evaluate real world ISM treatment management, including use of avapritinib.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Male or female adult participants (≥ 18 years of age) with a diagnosis of ISM according to the World Health Organization (WHO) diagnostic criteria
* Participant is currently being treated or plans to be treated with symptom-directed therapies and/or avapritinib for ISM.
Exclusion Criteria:
* Participants with advanced systemic mastocytosis (AdvSM) or another associated hematologic neoplasm
* Participants with smoldering systemic mastocytosis
* Ongoing participation in interventional studies in systemic mastocytosis (SM) at the time of enrollment
* Participants currently receiving treatment with a KIT inhibitor other than avapritinib at the time of enrollment.
Primary outcome measure(s)
Baseline Demographics — Baseline (Month 1)
Change From Baseline in Patient-Reported Outcomes Scores — Baseline up to Month 61
Change From Baseline in Serum KIT D816V Variant Allele Frequency (VAF) — Baseline up to Month 61
Change From Baseline in Serum Tryptase — Baseline up to Month 61
Change From Baseline in Indolent Systemic Mastocytosis (ISM) Symptom-Directed Therapies — Baseline up to Month 61
Trial sites (7)
Facility
City
Region
Status
University of Colorado
Aurora
Colorado
Recruiting
Midwest Allergy Sinus Asthma
Normal
Illinois
Recruiting
Institute for Allergy and Asthma
Wheaton
Maryland
Recruiting
Brigham and Women's Allergy and Clinical Immunology, Mass General Brigham Healthcare Center (Chestnut Hill)
Chestnut Hill
Massachusetts
Recruiting
University of Michigan Allergy Clinic
Ann Arbor
Michigan
Recruiting
Washington University of St. Louis
St Louis
Missouri
Recruiting
University of Utah
Salt Lake City
Utah
Recruiting
More Blueprint Medicines Corporation trials in the USA
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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