Familial dilated cardiomyopathy – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Tue, 02 Jun 2026 04:15:44 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Familial dilated cardiomyopathy – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study of ALXN2350 gene therapy in adults with BAG3 mutation-associated dilated cardiomyopathy: A safety and efficacy trial https://clinicaltrials.eu/trial/study-of-alxn2350-gene-therapy-in-adults-with-bag3-mutation-associated-dilated-cardiomyopathy-a-safety-and-efficacy-trial/ Thu, 30 Apr 2026 13:08:10 +0000 https://clinicaltrials.eu/trial/study-of-alxn2350-gene-therapy-in-adults-with-bag3-mutation-associated-dilated-cardiomyopathy-a-safety-and-efficacy-trial/ This clinical trial studies BAG3 Mutation-Associated Dilated Cardiomyopathy, a heart condition where the heart muscle becomes enlarged and weak due to a specific genetic mutation. The study will test a new gene therapy called ALXN2350, which is given as a single intravenous infusion. The therapy uses a modified virus called adeno-associated virus serotype 9 to deliver a corrective gene to heart cells.

The study aims to determine if this treatment is safe and can help improve heart function in adults with this specific type of heart disease. During the trial, participants will receive either the gene therapy or standard heart failure medications including sirolimus, prednisone, and co-trimoxazole. The medications will be given in tablet form or as oral solutions.

Throughout the study, doctors will monitor participants’ heart health using various tests including blood samples and heart measurements. They will check for changes in heart function and track important markers of heart health in the blood. The study will also measure how the body responds to the treatment and watch for any side effects.

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