NCT07264959Recruiting

An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)

Blueprint Medicines Corporation

Start Date

4/15/2026

Completion Date

12/1/2032

Summary

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 Criteria

Age Range: 18 years to No maximum

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.

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Conditions

Indolent Systemic Mastocytosis

Locations

Brigham and Women's Allergy and Clinical Immunology, Mass General Brigham Healthcare Center (Chestnut Hill)

Chestnut Hill, Massachusetts 02467

United States