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 males with Danon disease, a genetic condition that affects the heart. The treatment aims to fix the faulty gene to help improve heart function and slow disease progression.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are male and at least 8 years old.
- You have a confirmed genetic change (mutation) in the LAMP2 gene linked to Danon disease.
- Your heart shows thickening of the left ventricle (the main pumping chamber) and your heart's pumping function is still relatively good (ejection fraction of 50% or higher).
- You have mild to moderate heart failure symptoms (NYHA class II or III).
- You have had a blood test showing elevated troponin (a marker of heart damage) within the last 12 months.
- You do not have severe heart failure, a history of blood clots or strokes, or a prior organ transplant.
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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