Observational Study of Adults With Indolent Systemic Mastocytosis Receiving Symptom Treatments or Avapritinib

What this Trial Is About

Researchers are conducting a Phase 4 observational study to collect detailed information about people with Indolent Systemic Mastocytosis ISM. The study focuses on understanding the clinical features, outcomes, and medical care of ISM in real-world settings. It also aims to describe the demographics and symptoms such as anaphylaxis and bone problems, and assess quality of life and disease control through patient questionnaires. Participants in this study include those currently treated or planning to be treated for ISM, including treatment with avapritinib or other symptom-directed therapies. This is a non-interventional study, meaning treatments are not assigned by the study but are observed as they occur naturally. Data will be collected over time to understand how ISM is managed and how patients respond to treatments. During the study, participants will provide information about their health and treatments through questionnaires and clinical evaluations. Researchers will measure changes in patient-reported outcomes, serum KIT D816V variant allele frequency, serum tryptase levels, and the use of symptom-directed therapies from baseline up to 61 months. The study will continue until December 2032, allowing long-term observation of participants health and treatment effects.

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