Using MEK inhibitors to treat hypertrophic cardiomyopathy in children with RASopathies

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to evaluate the effectiveness of trametinib, a MEK kinase inhibitor, in treating hypertrophic cardiomyopathy (HCM) in children with genetic mutations in the RAS/MAPK pathway, such as those seen in Noonan syndrome. It is a randomized, open-label trial involving patients aged 0 to 18 who have been diagnosed with both RASopathy and HCM. Participants will be divided into two groups: one receiving trametinib alongside standard treatment and the other receiving only standard treatment. The study will assess the effectiveness of trametinib over a three-month period, with potential continuation based on results.

Why it matters

Who can join: the full rules

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 hypersensitivity, WPW syndrome, atrioventricular block, QT prolongation) trametinib (drug hypersensitivity)
* lack of consent of the child's guardians to participate in the study

Where it's running

• Department of Paediatrics, The Medical University of Warsaw, Poland
  Warsaw, Poland Recruiting

Study contacts

• Principal investigator: MAciej Kołodziej, MD — Medical University of Warsaw
• Study coordinator: Maciej Kołodziej, MD
• Email: maciej.kolodziej@wum.edu.pl
• Phone: 22-317-95-37

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Therapy to help caregivers of children with genetic disorders manage stress

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.