An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)
Purpose
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.
Condition
- Indolent Systemic Mastocytosis
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Sex
- All
- Accepts 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.
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
| Participants With ISM | Patients who are currently being treated or plan to be treated for ISM |
Recruiting Locations
Brigham and Women's Allergy and Clinical Immunology, Mass General Brigham Healthcare Center (Chestnut Hill)
Chestnut Hill, Massachusetts 02467
Chestnut Hill, Massachusetts 02467
More Details
- Status
- Recruiting
- Sponsor
- Blueprint Medicines Corporation