RP-A701 gene therapy for BAG3-related dilated cardiomyopathy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label, dose-escalation Phase 1 trial gives a single IV dose of RP-A701, an AAVrh.74 vector carrying the BAG3 transgene, to adults with genetically confirmed BAG3-DCM. The primary goal is to characterize safety and tolerability across ascending dose cohorts, with preliminary efficacy measured by changes in LVEF, heart failure symptoms, and clinical events. Eligible participants have LVEF 25–45%, NYHA class II–III symptoms, a pathogenic or likely pathogenic BAG3 variant, and an implanted ICD for at least 3 months. Participants are enrolled at multiple U.S. centers and will undergo serial clinical, imaging, and laboratory monitoring over planned follow-up visits.

Why it matters

Who can join: the full rules

Inclusion Criteria:

Subjects are eligible for inclusion into the study only if all the following criteria apply:

1. Male or female between 18 and 65 years of age at the time of signing the informed consent
2. Capable of and willing to provide signed informed consent
3. Clinical diagnosis of DCM defined as and requiring each of the following:

   1. Mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) by echocardiography or CMR performed within 3 months of enrollment.
   2. Absence of severe coronary artery disease (\>70% stenosis) or active myocardial ischemia as the etiology of LV systolic dysfunction
   3. Absence of uncontrolled hypertension, significant cardiac valve disease (i.e., greater than moderate in severity), infiltrative disorder, or systemic disease known to cause cardiomyopathy.
4. Documentation of a pathogenic or likely pathogenic variant in BAG3
5. History of ICD implantation ≥ 3 months prior to enrollment
6. NYHA Class II or III HF symptoms with stable HF therapeutic guideline-directed medical regimen for 30 days prior to enrollment

Exclusion Criteria:

1. CV disease that may be related to a genetic etiology other than a BAG3 pathogenic or likely pathogenic variant.
2. Previous participation in a study of gene transfer or gene editing.
3. I.V. inotropic, vasodilator, or diuretic therapy ≤ 30 days prior to enrollment.
4. History of intracardiac thrombosis or arterial thromboembolic events
5. Severe RV dysfunction assessed by echocardiogram or CMR ≤ 12 months prior to screening
6. LVEF \< 25% by echocardiogram or CMR at ≤ 3 months prior to screening
7. NYHA Class I or IV HF

Where it's running

• University of California, San Diego
  San Diego, California, United States Recruiting
• Mayo Clinic
  Rochester, Minnesota, United States Recruiting
• Medical University of South Carolina
  Charleston, South Carolina, United States Recruiting

Study contacts

• Study coordinator: Clinical Information
• Email: clinicaltrials@rocketpharma.com
• Phone: 646-627-0033

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.