An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM) (NCT07264959) | Clinical Trial Compass
RecruitingNot Applicable
An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)
United States150 participantsStarted 2026-04-15
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Baseline Demographics
Timeframe: Baseline (Month 1)
2
Change From Baseline in Patient-Reported Outcomes Scores
Timeframe: Baseline up to Month 61
3
Change From Baseline in Serum KIT D816V Variant Allele Frequency (VAF)
Timeframe: Baseline up to Month 61
4
Change From Baseline in Serum Tryptase
Timeframe: Baseline up to Month 61
5
Change From Baseline in Indolent Systemic Mastocytosis (ISM) Symptom-Directed Therapies