MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies
Recruiting
Phase 2
Interventional Study
Cardiomegaly
Noonan Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 1 Day – 18
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 40 (estimated)
- Sponsor
- Medical University of Warsaw · Other
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About This Trial
The goal of this study is to evaluate the effectiveness of trametinib treatment in patients with Hyperthropic cardiomyopathy and a genetic mutation in the RAS/MAPK pathway.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* patient with diagnosed RASopathy
* patient with diagnosed hypertrophic cardiomyopathy
* signed innform consent
Exclusion Criteria:
* contraindications to treatment with propranolol (drug hypersensitivity, atrioventricular block, severe bradycardia) disopyramide (drug hyperse…
Contacts