Menu
Recruiting NCT07255638

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

— Observational Indolent Systemic Mastocytosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Avapritinib.
Who it may be relevant to
Registry conditions: Indolent Systemic Mastocytosis. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Germany
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official 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

Overview

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

  • Drug Avapritinib
    Avapritinib will be administered as an oral tablet.

Primary outcome measures

  • Change From Baseline in Mastocytosis Control-quality of Life (MC-QoL) Questionnaire Score at Month 6 [Time frame: Baseline, Month 6]
Secondary outcome measures (12)
  • Socio-demographic Variable: Participant Age [Time frame: Baseline (Day -30 to Day -1)]
  • Socio-demographic Variable: Participant Gender [Time frame: Baseline (Day -30 to Day -1)]
  • Socio-demographic Variable: Participant Ethnicity [Time frame: Baseline (Day -30 to Day -1)]
  • Socio-demographic Variable: Participant Race [Time frame: Baseline (Day -30 to Day -1)]
  • Baseline Serum Tryptase and KIT D816V Variant Allele Fraction (VAF) [Time frame: Baseline (Day -30 to Day -1)]
  • Number of Participants With Prior Medical History of Anaphylaxis [Time frame: Baseline (Day -30 to Day -1)]
  • Number of Participants with Comorbidity [Time frame: 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 [Time frame: Baseline up to Month 24]
  • Number of Participants Prescribed a Life Support Kit [Time frame: Baseline up to Month 24]
  • 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]
  • 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]
  • 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]

Eligibility 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Other

Study locations

Germany · 8 centers
  • Charité Universitätmedizin Berlin, Klinik für Dermatologie, Venerologie und Allergologie — Berlin
  • Katholisches Klinikum Bochum — Bochum
  • MVZ Klinikum Deggendorf — Deggendorf
  • Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie — Dresden
  • University Medical Center Goettingen - Georg August University of Goettingen — Göttingen
  • Universitats-Hautklinik Tubingen, Studienzentrum lmmundermatologie — Tübingen
  • HNO-Gemeinschaftspraxis Wiesbaden, GEKA Gesellschaft für Experimentelle und Klinische Atem — Wiesbaden
  • Helios Universitätsklinikum Wuppertal — Wuppertal

Identifiers

NCT: NCT07255638 · BLU-285-2407

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗