Tests RP-A701 gene therapy in adults with BAG3 dilated cardiomyopathy
Tests RP-A701, given once through a vein, in adults with BAG3 heart disease.
Plain-language title generated from the registry record
Registry title: A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Key facts
- Where
- Charleston, Rochester, and San Diego, United StatesSee the 3 sites
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 65 years old?
Source: NCT07137338, who can take part
Where in United States
- Medical University of South CarolinaCharleston, United StatesTaking people
- Mayo ClinicRochester, United StatesTaking people
- University of California San DiegoSan Diego, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Rocket Pharmaceuticals Inc.
- Trial code
- NCT07137338
- Record updated
- 1 October 2026
- Original description
This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV administration in high-risk adult patients with BAG3-DCM.Read full original descriptionShow less
A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human BCL2-associated Athanogene 3 (BAG3) Gene Coding Sequence (RP-A701) in Subjects With Dilated Cardiomyopathy Arising From Pathogenic BAG3 Variants (BAG3-DCM)
This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV administration in high-risk adult patients with BAG3-DCM.
Inclusion: Subjects are eligible for inclusion into the study only if all the following criteria apply:; Male or female between 18 and 65 years of age at the time of signing the informed consent; Capable of and willing to provide signed informed consent; Clinical diagnosis of DCM defined as and requiring each of the following:; Mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) by echocardiography or CMR performed within 3 months of enrollment; Absence of severe coronary artery disease (>70% stenosis) or active myocardial ischemia as the etiology of LV systolic dysfunction; Absence of uncontrolled hypertension, significant cardiac valve disease (i.e., greater than moderate in severity), infiltrative disorder, or systemic disease known to cause cardiomyopathy; Documentation of a pathogenic or likely pathogenic variant in BAG3; History of ICD implantation ≥ 3 months prior to enrollment; NYHA Class II or III HF symptoms with stable HF therapeutic guideline-directed medical regimen for 30 days prior to enrollment.
Exclusion: Previous participation in a study of gene transfer or gene editing; I.V. inotropic, vasodilator, or diuretic therapy ≤ 30 days prior to enrollment; Severe RV dysfunction assessed by echocardiogram or CMR ≤ 12 months prior to screening; CV disease that may be related to a genetic etiology other than a BAG3 pathogenic or likely pathogenic variant; History of intracardiac thrombosis or arterial thromboembolic events; LVEF < 25% by echocardiogram or CMR at ≤ 3 months prior to screening; NYHA Class I or IV HF.