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

Browse trials for

• Mastocytosis, Systemic 56
• Mastocytosis 49

Keywords

• ISM
• Indolent systemic mastocytosis
• Avapritinib
• BLU-285
• Selective KIT mutation-targeted tyrosine kinase inhibitor
• Tyrosine kinase inhibitor
• Mastocytosis
• Neoplastic mast cells

Mastocytosis, Systemic

56 studies on the registry are indexed under Mastocytosis, Systemic; 16 are open to participants now.

This study's planned enrollment of 80 is below the median of 155 across 18 observational studies indexed under Mastocytosis, Systemic.

Lead sponsor

Blueprint Medicines Corporation is the lead sponsor of 32 studies on the registry; 6 are open to participants now.

Of its 14 completed or terminated interventional studies of FDA-regulated products, 3 (21%) have results posted.

First submitted after Blueprint Medicines Corporation became part of Sanofi.

Study population

Groups and cohorts

• 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).

  Drug: Avapritinib

Interventions

• DrugAvapritinib

  Avapritinib will be administered as an oral tablet.

  Also known as: Ayvakyt, BLU-285

Primary outcomes

1. Change From Baseline in Mastocytosis Control-quality of Life (MC-QoL) Questionnaire Score at Month 6

   Time frame: Baseline, Month 6

Secondary outcomes

1. Socio-demographic Variable: Participant Age

   Time frame: Baseline (Day -30 to Day -1)

2. Socio-demographic Variable: Participant Gender

   Time frame: Baseline (Day -30 to Day -1)

3. Socio-demographic Variable: Participant Ethnicity

   Time frame: Baseline (Day -30 to Day -1)

4. Socio-demographic Variable: Participant Race

   Time frame: Baseline (Day -30 to Day -1)

5. Baseline Serum Tryptase and KIT D816V Variant Allele Fraction (VAF)

   Time frame: Baseline (Day -30 to Day -1)

6. Number of Participants With Prior Medical History of Anaphylaxis

   Time frame: Baseline (Day -30 to Day -1)

7. Number of Participants with Comorbidity

   Time frame: Baseline up to Month 24

8. Number of Participants Initiating a New ISM-directed Medication, Discontinuing an ISM-directed Medication, and With Dose Increases or Decreases for ISM-directed Medications

   Time frame: Baseline up to Month 24

9. Number of Participants Prescribed a Life Support Kit

   Time frame: Baseline up to Month 24

10. Change From Baseline in Mastocytosis Activity Score (MAS) at Months 3, 6, 12, 18, and 24

    Time frame: Baseline, Months 3, 6, 12, 18, and 24

11. Change From Baseline in MC-QoL Questionnaire Score at Months 3, 6, 12, 18, and 24

    Time frame: Baseline, Months 3, 6, 12, 18, and 24

12. Change From Baseline in Mastocytosis Control Test (MCT) Score at Months 3, 6, 12, 18, and 24

    Time frame: Baseline, Months 3, 6, 12, 18, and 24

13. Time to Response as Assessed by MAS

    Time frame: Baseline up to Month 24

14. Change From Baseline in Serum Tryptase at Months 3, 6, 12, 18, and 24

    Time frame: Baseline, Months 3, 6, 12, 18, and 24

15. Number of Participants With Anaphylaxis Requiring Hospitalization, Emergency Room (ER) Visit, Epinephrine Autoinjector Use, or Other Medication

    Time frame: Baseline up to Month 24

16. Number of Participants Meeting the Definition of Polypharmacy (Use of Multiple Medications Simultaneously; Typically, 3 or More Symptom-directed Prescription Drugs)

    Time frame: Baseline up to Month 24

17. Number of Participants With ISM-related Hospital Visits and ER Visits

    Time frame: Baseline up to Month 24

18. Number of Participants With Changes in ISM Symptom-directed Therapies

    Time frame: Baseline up to Month 24

19. Number of Participants With Dose Modifications and Study/Treatment Discontinuations

    Time frame: Baseline up to Month 24