Study on heart disease in children with RASopathy
Part of Heart & circulation clinical trials.
This study looks at medical records of children with a genetic condition called RASopathy who developed thickened heart muscle and needed hospital care for heart failure in their first six months of life. The goal is to understand how the heart condition progresses over time.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed genetic diagnosis of a RASopathy (a group of conditions caused by changes in the RAS-MAPK pathway).
- You had a heart ultrasound showing thickened heart muscle (more than normal for your age).
- You were admitted to the hospital for congestive heart failure between January 2015 and June 2019, or developed worsening heart failure during a hospital stay in your first six months of life.
- You are not currently taking an mTOR inhibitor or MEK inhibitor (specific types of targeted therapy).
- Your doctor can find or calculate your Ross score (a measure of heart failure) from your medical records during your first six months of life.
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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