Observing avapritinib treatment in people with indolent systemic mastocytosis in Germany
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 That Are Not Adequately Controlled With Symptomatic Treatments in Real-World Settings
Blueprint Medicines Corporation · NCT07255638
This study will see if avapritinib helps manage symptoms in people with indolent systemic mastocytosis who are starting the drug as part of routine care in Germany.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 80 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Blueprint Medicines Corporation (industry) |
| Drugs / interventions | avapritinib |
| Locations | 8 sites (Berlin and 7 other locations) |
| Trial ID | NCT07255638 on ClinicalTrials.gov |
What this trial studies
This is a non-interventional, real-world observational study following participants for up to 24 months to document how avapritinib is used and its effects in routine clinical practice. Participants are enrolled when their treating clinician starts avapritinib for ISM with moderate to severe symptoms not controlled by symptomatic therapy, and treatment follows the approved product information. Data collected will include clinical outcomes, safety events, and elements of the natural history and management of ISM. The study is conducted at several German centers, including Charité in Berlin and regional hospital sites in Bochum and Deggendorf.
Who should consider this trial
Good fit: Ideal candidates are people with indolent systemic mastocytosis who are about to start avapritinib for moderate to severe symptoms not adequately controlled with symptomatic treatment and who meet the product labeling and clinic eligibility.
Not a fit: People with a history or risk of intracranial hemorrhage, recent cerebrovascular events, severe thrombocytopenia, or prior exposure to avapritinib are excluded and unlikely to benefit from participating.
Why it matters
Potential benefit: If successful, the study could provide real-world evidence on effectiveness and safety of avapritinib in ISM, helping clinicians make better treatment and management decisions.
How similar studies have performed: Avapritinib has shown clinical activity in systemic mastocytosis, including responses in advanced forms, but real-world data specifically in indolent systemic mastocytosis remain limited.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Berlin and 7 other locations
- Charité Universitätmedizin Berlin, Klinik für Dermatologie, Venerologie und Allergologie — Berlin, Germany (RECRUITING)
- Katholisches Klinikum Bochum — Bochum, Germany (RECRUITING)
- MVZ Klinikum Deggendorf — Deggendorf, Germany (RECRUITING)
- Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie — Dresden, Germany (RECRUITING)
- University Medical Center Goettingen - Georg August University of Goettingen — Göttingen, Germany (RECRUITING)
- Universitats-Hautklinik Tubingen, Studienzentrum lmmundermatologie — Tübingen, Germany (RECRUITING)
- HNO-Gemeinschaftspraxis Wiesbaden, GEKA Gesellschaft für Experimentelle und Klinische Atemwegsforschung mbH, Zentrum für Rhinologie und Allergologie — Wiesbaden, Germany (RECRUITING)
- Helios Universitätsklinikum Wuppertal — Wuppertal, Germany (RECRUITING)
Study contacts
- Study coordinator: Blueprint Medicines, EU Contact
- Email: medinfoeurope@blueprintmedicines.com
- Phone: +31 85 064 4001
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Indolent Systemic Mastocytosis, ISM, Indolent systemic mastocytosis, Avapritinib, BLU-285, Selective KIT mutation-targeted tyrosine kinase inhibitor, Tyrosine kinase inhibitor, Mastocytosis