An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Indolent Systemic Mastocytosis. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Observational Study in Patients With Indolent Systemic Mastocytosis
Overview
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.
Primary outcome measures
- Baseline Demographics [Time frame: Baseline (Month 1)]
- Change From Baseline in Patient-Reported Outcomes Scores [Time frame: Baseline up to Month 61]
- Change From Baseline in Serum KIT D816V Variant Allele Frequency (VAF) [Time frame: Baseline up to Month 61]
- Change From Baseline in Serum Tryptase [Time frame: Baseline up to Month 61]
- Change From Baseline in Indolent Systemic Mastocytosis (ISM) Symptom-Directed Therapies [Time frame: Baseline up to Month 61]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 3 centers
- Institute for Allergy and Asthma — Wheaton
- Brigham and Women's Allergy and Clinical Immunology, Mass General Brigham Healthcare Cente — Chestnut Hill
- University of Michigan Allergy Clinic — Ann Arbor
Identifiers
NCT: NCT07264959 · BLU-285-2406