Indolent Systemic MastocytosisNCT07255638
A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany
An observational study of Indolent Systemic Mastocytosis, run by Blueprint Medicines Corporation at 12 sites in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Indolent Systemic Mastocytosis
- What is being tested?
- Avapritinib
- Type of treatment
- Pill or oral medicine
- How many people
- 80 participants
- Where
- 12 sitesGermany
- Study dates
- 9 Dec 2025to 1 Dec 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
12 sites in 1 country. Contact the study team or a site near you to ask about taking part.
12 sites in 1 country
Germany12 sites, 12 recruiting
- Recruiting
Charité Universitätmedizin Berlin, Institut für Allergieforschung
Berlin, 12203
- Recruiting
Katholisches Klinikum Bochum
Bochum, 44791
- Recruiting
MVZ Klinikum Deggendorf
Deggendorf, 94469
- Recruiting
Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie
Dresden, 01307
- Recruiting
Universitatsmedizin Freiburg, Klinik fur Innere Medizin
Freiburg im Breisgau, 79106
- Recruiting
University Medical Center Goettingen - Georg August University of Goettingen
Göttingen, 37075
- Recruiting
Klinik fur Hamatologie Universistatklinik Leipzig
Leipzig, 04103
- Recruiting
Technische Universitat Munchen, Studienzentrum der Klinik fur Dermatologie am Biedertein
Munich, 65119
- Recruiting
Universitats-Hautklinik Tubingen, Studienzentrum lmmundermatologie
Tübingen, 72076
- Recruiting
Helios Dr. Horst Schmidt Kliniken Wiesbaden
Wiesbaden, 65199
- Recruiting
HNO-Gemeinschaftspraxis Wiesbaden, GEKA Gesellschaft für Experimentelle und Klinische Atemwegsforschung mbH, Zentrum für Rhinologie und Allergologie
Wiesbaden, 65205
- Recruiting
Helios Universitätsklinikum Wuppertal
Wuppertal, 42283
About this study
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
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.
- Conditions
- Indolent Systemic Mastocytosis
- Being tested
- Avapritinib
- Keywords
- ISM, Indolent systemic mastocytosis, Avapritinib, BLU-285, Selective KIT mutation-targeted tyrosine kinase inhibitor, Tyrosine kinase inhibitor, Mastocytosis, Neoplastic mast cells
- Medical subject headings
- Mastocytosis, Systemic, Mastocytosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvapritinibDrug
Also called Ayvakyt, BLU-285
Avapritinib will be administered as an oral tablet.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Drug: Avapritinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Mastocytosis Control-quality of Life (MC-QoL) Questionnaire Score at Month 6
Measured: Baseline, Month 6
Secondary outcomes (19)
Socio-demographic Variable: Participant Age
Measured: Baseline (Day -30 to Day -1)
Socio-demographic Variable: Participant Gender
Measured: Baseline (Day -30 to Day -1)
Socio-demographic Variable: Participant Ethnicity
Measured: Baseline (Day -30 to Day -1)
Socio-demographic Variable: Participant Race
Measured: Baseline (Day -30 to Day -1)
Baseline Serum Tryptase and KIT D816V Variant Allele Fraction (VAF)
Measured: Baseline (Day -30 to Day -1)
Number of Participants With Prior Medical History of Anaphylaxis
Measured: Baseline (Day -30 to Day -1)
Number of Participants with Comorbidity
Measured: 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
Measured: Baseline up to Month 24
Number of Participants Prescribed a Life Support Kit
Measured: Baseline up to Month 24
Change From Baseline in Mastocytosis Activity Score (MAS) at Months 3, 6, 12, 18, and 24
Measured: Baseline, Months 3, 6, 12, 18, and 24
Change From Baseline in MC-QoL Questionnaire Score at Months 3, 6, 12, 18, and 24
Measured: 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
Measured: Baseline, Months 3, 6, 12, 18, and 24
Time to Response as Assessed by MAS
Measured: Baseline up to Month 24
Change From Baseline in Serum Tryptase at Months 3, 6, 12, 18, and 24
Measured: 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
Measured: 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)
Measured: Baseline up to Month 24
Number of Participants With ISM-related Hospital Visits and ER Visits
Measured: Baseline up to Month 24
Number of Participants With Changes in ISM Symptom-directed Therapies
Measured: Baseline up to Month 24
Number of Participants With Dose Modifications and Study/Treatment Discontinuations
Measured: Baseline up to Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Dec 2025
- Study start9 Dec 2025
- Primary completion1 Dec 2028 (estimated)
- Study completion1 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Indolent Systemic MastocytosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07255638
- Sponsor's protocol ID
- BLU-285-2407
- Status last verified
- Oct 2026
- First submitted
- 20 Nov 2025
- Last updated
- 7 Oct 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07255638
- Acronym / sponsor ID
- BLU-285-2407
- Conditions
- Indolent Systemic Mastocytosis
- Keywords
- ISM, Indolent systemic mastocytosis, Avapritinib, BLU-285, Selective KIT mutation-targeted tyrosine kinase inhibitor, Tyrosine kinase inhibitor, Mastocytosis, Neoplastic mast cells
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- Study start
- 9 Dec 2025 (actual)
- Primary completion
- 1 Dec 2028 (estimated)
- Study completion
- 1 Dec 2028 (estimated)
- First posted
- 1 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Avapritinib | – | Drug: AvapritinibParticipants will receive avapritinib as part of their treatment plan with their healthcare practitioner (HCP) and in accordance with the Summary of Product Characteristics (SmPC). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (1 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AvapritinibAlso: Ayvakyt, BLU-285Avapritinib will be administered as an oral tablet. | Drug | Avapritinib |
Primary outcome
Change From Baseline in Mastocytosis Control-quality of Life (MC-QoL) Questionnaire Score at Month 6
Time frame: Baseline, Month 6
19 secondary outcomes
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
Time to Response as Assessed by MAS
Time frame: 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
Number of Participants With Anaphylaxis Requiring Hospitalization, Emergency Room (ER) Visit, Epinephrine Autoinjector Use, or Other Medication
Time frame: 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
Number of Participants With ISM-related Hospital Visits and ER Visits
Time frame: Baseline up to Month 24
Number of Participants With Changes in ISM Symptom-directed Therapies
Time frame: Baseline up to Month 24
Number of Participants With Dose Modifications and Study/Treatment Discontinuations
Time frame: Baseline up to Month 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2026.
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(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Indolent Systemic Mastocytosis, run by Blueprint Medicines Corporation at 12 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 7 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Blueprint Medicines, EU Contact (+31 85 064 4001, medinfoeurope@blueprintmedicines.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Mastocytosis Control-quality of Life (MC-QoL) Questionnaire Score at Month 6 (time frame: Baseline, Month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Oct 2026.