Gene therapy trial for Friedreich ataxia heart disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial tests a one-time gene therapy called LX2006 for heart problems caused by Friedreich ataxia. The goal is to see if it can improve heart function and structure.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 6 years old.
- You must have a confirmed diagnosis of Friedreich ataxia with symptoms starting by age 25.
- Your heart must show thickening (left ventricular hypertrophy) and abnormal mass.
- Your heart's pumping ability must be at least 30%.
- You cannot have other causes of heart failure or have had gene therapy before.
- You must not have poorly controlled diabetes or active cancer.
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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