Real-world care and outcomes for people with indolent systemic mastocytosis (ISM)
Official title: An Observational Study in Patients With Indolent Systemic Mastocytosis
Run by Blueprint Medicines Corporation · NCT07264959 · Data updated 2026-11-12
This study will see how people with ISM are treated and how they are doing in everyday care, including those taking avapritinib.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 150 (estimated) |
| Sponsor | Blueprint Medicines Corporation Industry-sponsored |
| Drugs / interventions | avapritinib |
| Locations | 7 sites (Aurora, Colorado and 6 other locations) |
| Status | Recruiting |
| Started | April 15, 2026 |
| Main results expected | December 1, 2032 |
| Study ends | December 1, 2032 |
| Registry record updated | September 4, 2026 |
| Trial ID | NCT07264959 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adults aged 18 or older with a WHO diagnosis of indolent systemic mastocytosis who are currently being treated or plan to be treated with symptom-directed therapies and/or avapritinib are ideal candidates.
✕Probably not a fit
People with advanced systemic mastocytosis, smoldering systemic mastocytosis, another associated hematologic neoplasm, those enrolled in interventional SM trials, or those receiving a KIT inhibitor other than avapritinib are excluded and unlikely to benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This Phase 4, non-interventional observational program will collect detailed clinical and treatment data from adults with WHO-diagnosed indolent systemic mastocytosis. Investigators will record demographics, clinical features such as anaphylaxis and bone manifestations, treatments used in routine practice, and participant-reported quality of life and disease control via questionnaires. The study specifically includes participants treated or planning treatment with symptom-directed therapies and/or avapritinib and excludes those with advanced or smoldering disease, other hematologic neoplasms, or use of non-avapritinib KIT inhibitors. Data will be gathered from medical records and participant questionnaires to characterize real-world management and outcomes over time.
Why it matters
Potential benefit: If successful, the study could improve understanding of how ISM is managed in real life and help guide treatment decisions and supportive care for patients.
How similar studies have gone: Observational registries and real-world studies in mast cell disorders have provided useful clinical insights and safety signals, and clinical trials have shown benefit of KIT-targeted agents in related forms, but prospective ISM-specific real-world data remain limited.
Who can join: the full rules
Show full inclusion / exclusion criteria
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.
Where it's running
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University of Colorado
Aurora, Colorado, United States Recruiting -
Midwest Allergy Sinus Asthma
Normal, Illinois, United States Recruiting -
Institute for Allergy and Asthma
Wheaton, Maryland, United States Recruiting -
Brigham and Women's Allergy and Clinical Immunology, Mass General Brigham Healthcare Center (Chestnut Hill)
Chestnut Hill, Massachusetts, United States Recruiting -
University of Michigan Allergy Clinic
Ann Arbor, Michigan, United States Recruiting -
Washington University of St. Louis
St Louis, Missouri, United States Recruiting
Show 1 more location
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University of Utah
Salt Lake City, Utah, United States Recruiting
Study contacts
- Study coordinator: Blueprint Medicines
- Email: medinfo@blueprintmedicines.com
- Phone: 1-888-258-7768
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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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.
-
Angioedema biomarker tests to help diagnose recurrent swelling
This study tests multiple blood-based diagnostic tests to see if they can better identify the cause of recurrent angioedema in patients compared with current tests.
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Evaluating BLU-263 for Indolent Systemic Mastocytosis
This study is testing if a new medication called elenestinib can help people with indolent systemic mastocytosis who aren't getting enough relief from their current treatments.
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Italian Mastocytosis Registry
This registry collects medical data from adults with systemic mastocytosis across 27 Italian centers to see how the disease progresses and how care varies over time.
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Peripheral blood KIT-D816V testing in adults with systemic mastocytosis
This will test whether a sensitive blood test (ddPCR) can find the KIT-D816V mutation in adults with systemic mastocytosis so they might avoid a bone marrow biopsy.
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Tracking mastocytosis from childhood to adulthood
This hospital registry will collect clinical, laboratory, and genetic information from children and adults with cutaneous or systemic mastocytosis seen in Pavia to see how the disease changes over time and what predicts outcomes.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.