Gene therapy for BAG3-related dilated cardiomyopathy
Part of Genetic & congenital, Heart & circulation clinical trials.
This trial tests a one-time gene therapy called RP-A701 for people with dilated cardiomyopathy caused by a BAG3 gene mutation. The goal is to see if it is safe and can improve heart function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 65 years old.
- You have a confirmed BAG3 gene mutation that causes heart muscle problems.
- Your heart's pumping strength is moderately reduced (ejection fraction between 25% and 45%).
- You have an implantable cardioverter-defibrillator (ICD) placed at least 3 months ago.
- You have mild to moderate heart failure symptoms (NYHA class II or III) and are on stable heart medications.
- You do not have severe coronary artery disease, uncontrolled high blood pressure, or significant valve disease as the cause of your heart condition.
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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