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NCT Number: NCT07255638

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

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.

Recruiting

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Key information

Conditions

Indolent Systemic Mastocytosis Immune System Diseases Mast Cell Activation Disorders Mastocytosis Mastocytosis, Systemic Neoplasms Neoplasms by Histologic Type Neoplasms, Connective Tissue Neoplasms, Connective and Soft Tissue

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Charité Universitätmedizin Berlin, Klinik für Dermatologie, Venerologie und Allergologie, Berlin, Germany

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Avapritinib

Drug

Avapritinib will be administered as an oral tablet.

Other names: 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

Study contacts

Contact information is provided by the study sponsor or research team.

Blueprint Medicines, EU Contact

CONTACT

[email protected]

+31 85 064 4001

Sponsors and collaborators

Lead sponsor

Blueprint Medicines Corporation

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2028
Study completion
2028
First posted
Dec 1, 2025
Registry last updated
Apr 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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