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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Avapritinib | 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). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Avapritinib | Drug | Avapritinib will be administered as an oral tablet. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Mastocytosis Control-quality of Life (MC-QoL) Questionnaire Score at Month 6 | Baseline, Month 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Socio-demographic Variable: Participant Age | Baseline (Day -30 to Day -1) | |
| Socio-demographic Variable: Participant Gender | Baseline (Day -30 to Day -1) | |
| Socio-demographic Variable: Participant Ethnicity |
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Inclusion Criteria:
Exclusion Criteria:
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Adult participants with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment, who are initiating treatment with commercial avapritinib according to the German approved label.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Blueprint Medicines, EU Contact | Contact | +31 85 064 4001 | medinfoeurope@blueprintmedicines.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Charité Universitätmedizin Berlin, Klinik für Dermatologie, Venerologie und Allergologie | Recruiting | Berlin | 10117 | Germany |
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| ID | Term |
|---|---|
| D034721 | Mastocytosis, Systemic |
| D008415 | Mastocytosis |
| ID | Term |
|---|---|
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| C000707147 | avapritinib |
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| Baseline (Day -30 to Day -1) |
| Socio-demographic Variable: Participant Race | Baseline (Day -30 to Day -1) |
| Baseline Serum Tryptase and KIT D816V Variant Allele Fraction (VAF) | Baseline (Day -30 to Day -1) |
| Number of Participants With Prior Medical History of Anaphylaxis | Baseline (Day -30 to Day -1) |
| Number of Participants with Comorbidity | Baseline up to Month 24 |
| Number of Participants Initiating a New ISM-directed Medication, Discontinuing an ISM-directed Medication, and With Dose Increases or Decreases for ISM-directed Medications | Baseline up to Month 24 |
| Number of Participants Prescribed a Life Support Kit | Baseline up to Month 24 |
| Change From Baseline in Mastocytosis Activity Score (MAS) at Months 3, 6, 12, 18, and 24 | Baseline, Months 3, 6, 12, 18, and 24 |
| Change From Baseline in MC-QoL Questionnaire Score at Months 3, 6, 12, 18, and 24 | Baseline, Months 3, 6, 12, 18, and 24 |
| Change From Baseline in Mastocytosis Control Test (MCT) Score at Months 3, 6, 12, 18, and 24 | Baseline, Months 3, 6, 12, 18, and 24 |
| Time to Response as Assessed by MAS | Baseline up to Month 24 |
| Change From Baseline in Serum Tryptase at Months 3, 6, 12, 18, and 24 | Baseline, Months 3, 6, 12, 18, and 24 |
| Number of Participants With Anaphylaxis Requiring Hospitalization, Emergency Room (ER) Visit, Epinephrine Autoinjector Use, or Other Medication | Baseline up to Month 24 |
| Number of Participants Meeting the Definition of Polypharmacy (Use of Multiple Medications Simultaneously; Typically, 3 or More Symptom-directed Prescription Drugs) | Baseline up to Month 24 |
| Number of Participants With ISM-related Hospital Visits and ER Visits | Baseline up to Month 24 |
| Number of Participants With Changes in ISM Symptom-directed Therapies | Baseline up to Month 24 |
| Number of Participants With Dose Modifications and Study/Treatment Discontinuations | Baseline up to Month 24 |
| Katholisches Klinikum Bochum | Recruiting | Bochum | 44791 | Germany |
| MVZ Klinikum Deggendorf | Recruiting | Deggendorf | 94469 | Germany |
| Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie | Recruiting | Dresden | 01307 | Germany |
| University Medical Center Goettingen - Georg August University of Goettingen | Recruiting | Göttingen | 37075 | Germany |
| Universitats-Hautklinik Tubingen, Studienzentrum lmmundermatologie | Recruiting | Tübingen | 72076 | Germany |
| HNO-Gemeinschaftspraxis Wiesbaden, GEKA Gesellschaft für Experimentelle und Klinische Atemwegsforschung mbH, Zentrum für Rhinologie und Allergologie | Recruiting | Wiesbaden | 65205 | Germany |
| Helios Universitätsklinikum Wuppertal | Recruiting | Wuppertal | 42283 | Germany |
| D000090362 | Mast Cell Activation Disorders |
| D007154 | Immune System Diseases |