MEK inhibitor for severe infantile heart muscle disease from RASopathy
Treatments studied
Part of Bones, joints & muscles, Genetic & congenital, Heart & circulation, Skin clinical trials.
This study tests a new medicine for babies with a genetic condition that causes a thickened heart muscle. The medicine may help improve heart function in infants who are very sick and need hospital care.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed genetic change (in a RASopathy gene) linked to Noonan, Costello, or cardiofaciocutaneous syndrome
- Your baby is between 28 days and 6 months old
- Your baby has been diagnosed with hypertrophic cardiomyopathy (thickened heart muscle) with a specific echo finding
- Your baby has severe heart failure symptoms (Ross class III or IV) and is currently hospitalized
- Your baby has not taken a MEK inhibitor before
- Your baby does not have a serious bleeding risk (low platelet count) or active cancer requiring treatment
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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