Clinical Trials:
Dilated Cardiomyopathy (DCM)

RP-A701 is an investigational gene therapy for the treatment of BAG3-associated Dilated Cardiomyopathy (BAG3-DCM), an inherited heart disease caused by mutations in the BAG3 gene. The genetic mutation leads to an accumulation of misfolded and damaged proteins, which can impair the heart’s ability to contract, causing impaired cardiac function, heart failure, and even premature death.
Rocket has received approval from the FDA to initiate a Phase 1 study of RP-A701. The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. Initial study participants will include adults with implantable cardioverter defibrillators (ICDs) and advanced disease at high risk for heart failure progression and cardiac death. Participants will receive a single dose of RP-A701, and the trial will assess BAG3 protein expression, changes in cardiac biomarkers, and clinical predictors of disease progression.
Natural History Study
Rocket is also sponsoring a multi-center, global, non-interventional natural history study designed to collect longitudinal prospective and retrospective clinical information on patients with Danon Disease. The overarching goal of the study is to characterize the natural history of Danon Disease by collecting de-identified information from male patients living with the condition. Initial trial sites for the natural history study in the U.S. and EU have been identified, and patient enrollment will begin pending approval from each center’s institutional review board.
Natural History Trial Sites
- Texas Children’s Hospital, Texas, USA
- UC San Diego Health, California, USA
- Children’s Hospital Colorado, Colorado, USA
- Children’s Hospital of Philadelphia, Pennsylvania, USA
- Phoenix Children’s Hospital, Arizona, USA
- German Heart Center, Munich, Germany
- Great Ormond Street Hospital & UCL Institute of Cardiovascular Science, London, England
- Meyer Children’s Hospital, Firenze FI, Italy
- Sheba Medical Center, Ramat Gan, Israel
For more details about the Danon natural history study, visit clinicaltrials.gov.
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