Actively Recruiting
An Observational Study in Patients With Indolent Systemic Mastocytosis
Led by Blueprint Medicines Corporation · Updated on 2026-04-22
150
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This observational study focuses on adults diagnosed with Indolent Systemic Mastocytosis (ISM), aiming to gather detailed information about the clinical features, outcomes, and medical care of people living with ISM in everyday settings. The research will explore demographic and clinical aspects including episodes of anaphylaxis and bone-related issues. Participants' quality of life and how well their disease is controlled will be assessed through questionnaires. The study includes participants who are currently receiving or plan to receive treatments directed at ISM symptoms, including the medication avapritinib. As an observational study, there are no assigned treatments or placebos; instead, researchers will monitor participants' real-world management of ISM over time. Participants will be involved for up to 61 months, during which their baseline characteristics will be recorded. Researchers will regularly collect patient-reported outcomes, blood samples to measure specific markers like serum KIT D816V variant allele frequency and serum tryptase levels, and track changes in symptom-directed therapies. This long-term monitoring helps understand how ISM and its treatments affect patients over time, along with safety and disease progression.
CONDITIONS
Brief Title
An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female adult participants (18 years of age or older) diagnosed with Indolent Systemic Mastocytosis (ISM) according to WHO criteria
- Currently being treated or planning to be treated with symptom-directed therapies and/or avapritinib for ISM
You will not qualify if you...
- Diagnosed with advanced systemic mastocytosis (AdvSM) or another associated hematologic neoplasm
- Diagnosed with smoldering systemic mastocytosis
- Currently participating in interventional studies in systemic mastocytosis at enrollment
- Receiving treatment with a KIT inhibitor other than avapritinib at enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 61 months
Participants are observed to track changes in symptoms, treatments, and laboratory measures over time.
Regular assessments over up to 61 months
Trial Site Locations
Total: 1 location
1
Brigham and Women's Allergy and Clinical Immunology, Mass General Brigham Healthcare Center (Chestnut Hill)
Chestnut Hill, Massachusetts, United States, 02467
Actively Recruiting
Research Team
B
Blueprint Medicines
B
Blueprint Medicines, EU Contact
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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