An Observational Study In Participants With Indolent Systemic Mastocytosis (ISM)

Overview

About this study

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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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.

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 01/20/2026. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Thanai Pongdee, M.D.

Contact us for the latest status

Contact information:

Kay Bachman

5072845689

bachman.kay@mayo.edu

More information

Publications

Publications are currently not available