NCT07255638

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
80

participants targeted

Target at P50-P75 for all trials

Timeline
31mo left

Started Dec 2025

Typical duration for all trials

Geographic Reach
1 country

8 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress14%
Dec 2025Dec 2028

First Submitted

Initial submission to the registry

November 20, 2025

Completed
11 days until next milestone

First Posted

Study publicly available on registry

December 1, 2025

Completed
8 days until next milestone

Study Start

First participant enrolled

December 9, 2025

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2028

Last Updated

April 20, 2026

Status Verified

April 1, 2026

Enrollment Period

3 years

First QC Date

November 20, 2025

Last Update Submit

April 17, 2026

Conditions

Indolent Systemic Mastocytosis

Keywords

ISMIndolent systemic mastocytosisAvapritinibBLU-285Selective KIT mutation-targeted tyrosine kinase inhibitorTyrosine kinase inhibitorMastocytosisNeoplastic mast cells

Outcome Measures

Primary Outcomes (1)

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

    Baseline, Month 6

Secondary Outcomes (19)

  • 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)

  • +14 more secondary outcomes

Study Arms (1)

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

AvapritinibDRUG

Avapritinib will be administered as an oral tablet.

Also known as: Ayvakyt, BLU-285
Avapritinib

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

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.

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Charité Universitätmedizin Berlin, Klinik für Dermatologie, Venerologie und Allergologie

Berlin, 10117, Germany

RECRUITING

Katholisches Klinikum Bochum

Bochum, 44791, Germany

RECRUITING

MVZ Klinikum Deggendorf

Deggendorf, 94469, Germany

RECRUITING

Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie

Dresden, 01307, Germany

RECRUITING

University Medical Center Goettingen - Georg August University of Goettingen

Göttingen, 37075, Germany

RECRUITING

Universitats-Hautklinik Tubingen, Studienzentrum lmmundermatologie

Tübingen, 72076, Germany

RECRUITING

HNO-Gemeinschaftspraxis Wiesbaden, GEKA Gesellschaft für Experimentelle und Klinische Atemwegsforschung mbH, Zentrum für Rhinologie und Allergologie

Wiesbaden, 65205, Germany

RECRUITING

Helios Universitätsklinikum Wuppertal

Wuppertal, 42283, Germany

RECRUITING

MeSH Terms

Conditions

Mastocytosis, SystemicMastocytosis

Interventions

avapritinib

Condition Hierarchy (Ancestors)

Neoplasms, Connective TissueNeoplasms, Connective and Soft TissueNeoplasms by Histologic TypeNeoplasmsMast Cell Activation DisordersImmune System Diseases

Central Study Contacts

Blueprint Medicines, EU Contact

CONTACT

+31 85 064 4001medinfoeurope@blueprintmedicines.com

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 20, 2025

First Posted

December 1, 2025

Study Start

December 9, 2025

Primary Completion (Estimated)

December 1, 2028

Study Completion (Estimated)

December 1, 2028

Last Updated

April 20, 2026

Record last verified: 2026-04

Locations

DE(8)