Indolent Systemic Mastocytosis
Conditions
Keywords
ISM, Indolent systemic mastocytosis, Avapritinib, BLU-285, Selective KIT mutation-targeted tyrosine kinase inhibitor, Tyrosine kinase inhibitor, Mastocytosis, Neoplastic mast cells
Brief summary
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.
Interventions
DRUGAvapritinib
Avapritinib will be administered as an oral tablet.
Sponsors
Blueprint Medicines Corporation
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change From Baseline in Mastocytosis Control-quality of Life (MC-QoL) Questionnaire Score at Month 6 | Baseline, Month 6 |
Secondary
| Measure | 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 | 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 |
Countries
Germany
Contacts
CONTACTBlueprint Medicines, EU Contact
Outcome results
None listed