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

April 17, 2026 updated by: Blueprint Medicines Corporation

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
80

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Germany
      • Berlin, Germany, 10117
        • Recruiting
        • Charité Universitätmedizin Berlin, Klinik für Dermatologie, Venerologie und Allergologie
      • Bochum, Germany, 44791
        • Recruiting
        • Katholisches Klinikum Bochum
      • Deggendorf, Germany, 94469
        • Recruiting
        • MVZ Klinikum Deggendorf
      • Dresden, Germany, 01307
        • Recruiting
        • Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie
      • Göttingen, Germany, 37075
        • Recruiting
        • University Medical Center Goettingen - Georg August University of Goettingen
      • Tübingen, Germany, 72076
        • Recruiting
        • Universitats-Hautklinik Tubingen, Studienzentrum lmmundermatologie
      • Wiesbaden, Germany, 65205
        • Recruiting
        • HNO-Gemeinschaftspraxis Wiesbaden, GEKA Gesellschaft für Experimentelle und Klinische Atemwegsforschung mbH, Zentrum für Rhinologie und Allergologie
      • Wuppertal, Germany, 42283
        • Recruiting
        • Helios Universitätsklinikum Wuppertal

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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
Avapritinib will be administered as an oral tablet.
Other Names:
  • BLU-285
  • Ayvakyt

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change From Baseline in Mastocytosis Control-quality of Life (MC-QoL) Questionnaire Score at Month 6
Time Frame: Baseline, Month 6
Baseline, Month 6

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Socio-demographic Variable: Participant Age
Time Frame: Baseline (Day -30 to Day -1)
Baseline (Day -30 to Day -1)
Socio-demographic Variable: Participant Gender
Time Frame: Baseline (Day -30 to Day -1)
Baseline (Day -30 to Day -1)
Socio-demographic Variable: Participant Ethnicity
Time Frame: Baseline (Day -30 to Day -1)
Baseline (Day -30 to Day -1)
Socio-demographic Variable: Participant Race
Time Frame: Baseline (Day -30 to Day -1)
Baseline (Day -30 to Day -1)
Baseline Serum Tryptase and KIT D816V Variant Allele Fraction (VAF)
Time Frame: Baseline (Day -30 to Day -1)
Baseline (Day -30 to Day -1)
Number of Participants With Prior Medical History of Anaphylaxis
Time Frame: Baseline (Day -30 to Day -1)
Baseline (Day -30 to Day -1)
Number of Participants with Comorbidity
Time Frame: Baseline up to Month 24
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
Baseline up to Month 24
Number of Participants Prescribed a Life Support Kit
Time Frame: Baseline up to Month 24
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
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
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
Baseline, Months 3, 6, 12, 18, and 24
Time to Response as Assessed by MAS
Time Frame: Baseline up to Month 24
Baseline up to Month 24
Change From Baseline in Serum Tryptase at Months 3, 6, 12, 18, and 24
Time Frame: Baseline, 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
Time Frame: Baseline up to Month 24
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)
Time Frame: Baseline up to Month 24
Baseline up to Month 24
Number of Participants With ISM-related Hospital Visits and ER Visits
Time Frame: Baseline up to Month 24
Baseline up to Month 24
Number of Participants With Changes in ISM Symptom-directed Therapies
Time Frame: Baseline up to Month 24
Baseline up to Month 24
Number of Participants With Dose Modifications and Study/Treatment Discontinuations
Time Frame: Baseline up to Month 24
Baseline up to Month 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Blueprint Medicines Corporation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 9, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 20, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 20, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 1, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 20, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 17, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • BLU-285-2407

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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