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

Blueprint Medicines

Status: Enrolling

Conditions: Indolent Systemic Mastocytosis

Treatments: Drug: Avapritinib

Study type: Observational

Funder types: Industry

Identifiers

NCT07255638

BLU-285-2407

Details and patient eligibility

About

This is a non-interventional study assessing the effectiveness of avapritinib (BLU-285) in the management of ISM in real-world settings in Germany. The study also seeks to address the existing data gap in the natural history and management of participants with ISM.

The study is designed to follow each participant up to a maximum of 24 months.

Enrollment: 80 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participants is starting avapritinib treatment at the HCP's discretion as part of their routine care (for ISM with moderate to severe symptoms inadequately controlled with symptomatic treatment) and in accordance with approved SmPC.

Exclusion criteria

• Participant with a potential increased risk for intracranial hemorrhage including those with a history of vascular aneurysm, intracranial hemorrhage, cerebrovascular accident within the prior year, or severe thrombocytopenia.
• Participant who has previously taken avapritinib as a commercial drug or as part of a clinical study.

Trial design

80 participants in 1 patient group

Avapritinib

Description:

Participants will receive avapritinib as part of their treatment plan with their healthcare practitioner (HCP) and in accordance with the Summary of Product Characteristics (SmPC).

Treatment:

Drug: Avapritinib