A Prospective Non-Interventional Study to Describe the Effectiveness of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Patients With Indolent Systemic Mastocytosis and Symptoms Not Adequately Controlled With Symptomatic Treatments in Real-World Settings

What this Trial Is About

Researchers are evaluating the effectiveness of avapritinib (BLU-285) in treating people with indolent systemic mastocytosis (ISM) who have symptoms not well controlled by other treatments. This non-interventional study aims to fill gaps in understanding the natural history and management of ISM in real-world settings in Germany. Each participant will be followed for up to 24 months to observe treatment outcomes. Participants will receive avapritinib as an oral tablet prescribed by their healthcare provider, following standard treatment guidelines. The study does not assign treatment but observes participants who are starting avapritinib as part of their usual care. This approach allows researchers to gather data on how avapritinib is used and its effects in typical clinical practice. During the study, participants will undergo assessments including questionnaires to measure quality of life and mastocytosis symptoms at multiple time points up to 24 months. Researchers will collect information on demographics, medical history, medication changes, hospital visits, and safety events such as anaphylaxis. The main outcome is the change in quality of life measured at 6 months, with ongoing monitoring throughout the study period.

A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany