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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorBlueprint Medicines Corporation

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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 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.

Trial design

Design model

Other

Time perspective

Prospective

Treatments tested in this trial

  • Avapritinib

    Drug

    Avapritinib will be administered as an oral tablet.

Treatment groups

80 Participants
are divided into 1 treatment group
Group A: Avapritinib1 intervention

Trial outcomes

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)

Other outcomes

Sponsors and contacts

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