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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorBlueprint Medicines Corporation

About this trial

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.

Eligibility criteria

Qualifiers

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.

Disqualifiers

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.

Trial design

Treatments tested in this trial

  • Not listed

Trial groups

150 Participants
are grouped into 1 trial group