Gene therapy for BAG3-related heart failure
Part of Genetic & congenital, Heart & circulation clinical trials.
This study tests an experimental gene therapy called ALXN2350 for people with dilated cardiomyopathy caused by a specific genetic change in the BAG3 gene. It aims to see if the therapy can improve heart function and slow disease progression.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed change (mutation) in the BAG3 gene that is known to cause heart muscle disease.
- You have been diagnosed with dilated cardiomyopathy (a condition where the heart is enlarged and struggles to pump blood).
- You have had chronic heart failure for at least 3 months.
- Your heart's pumping strength (ejection fraction) is between 15% and 45%, measured by an echocardiogram (ultrasound of the heart).
- You are taking a stable combination of heart failure medications.
- You have not had any untreated serious heart valve problems.
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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