Gene therapy for Danon disease in males
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial tests a gene therapy called RP-A501 for men with Danon disease, a heart condition. It aims to replace the faulty gene that causes the disease to improve heart function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed genetic mutation in the LAMP2 gene.
- Must be male and 8 years or older.
- Your heart shows thickening of the walls but pumps normally (ejection fraction of 50% or more).
- You should have mild to moderate heart failure symptoms (NYHA class II or III).
- A blood test must show high troponin levels, a marker of heart damage.
- You cannot have had a heart or other organ transplant, severe heart failure, blood clots, or strokes.
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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