A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

NCT07137338 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: RP-A701-0125
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Dilated Cardiomyopathy (DCM)

Keywords

Cardiomyopathy; Dilated; BAG3 Protein; human; dilated cardiomyopathy; DCM; BAG3; BCL2-associated Athanogene 3; Genetic Cardiomyopathy; Gene Therapy; Heart Failure; Ventricular Arrhythmia; Cardiovascular Diseases; Cardiomyopathies; Heart Diseases; Inherited heart disease; AAVrh.74

Outcome Measures

Primary Outcomes (3)

• Incidence of Treatment-emergent Adverse Events (TEAE)

  Number of participants with Adverse Events following a single IV dose of RP-A701

  Baseline up to End of Study (up to 24 months post-infusion)

• Incidence of Treatment-emergent Serious Adverse Events (SAE).

  Number of participants with Serious Adverse Events (SAE) following a single IV dose of RP-A701

  Baseline up to End of Study (up to 24 months post-infusion)

• Incidence of Dose Limiting Toxicities (DLT).

  Number of participants with Dose Limiting Toxicities (DLT) following a single IV dose of RP-A701

  Baseline up to End of Study (up to 24 months post-infusion)

Secondary Outcomes (6)

• To assess the impact of RP-A701 on features of cardiovascular function.

  Baseline up to End of Study (up to 24 months post-infusion)

• To assess the impact of RP-A701 on features of cardiovascular function.

  Baseline up to End of Study (up to 24 months post-infusion)

• To assess the impact of RP-A701 on features of cardiovascular function.

  Baseline up to End of Study (up to 24 months post-infusion)

• To assess the extent of RP-A701 transduction and protein expression.

  Baseline up to End of Study (up to 24 months post-infusion)

• To assess the impact of RP-A701 on features of heart failure (HF).

  Baseline up to End of Study (up to 24 months post-infusion)

Study Arms (1)

Single ascending dose of RP-A701 in up to 2 consecutive cohorts

EXPERIMENTAL

Participants will receive a single intravenous dose of RP-A701 on Day 0 and will be followed for up to two years

Genetic: RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)

Interventions

RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3) GENETIC

One-time treatment with a single ascending dose

Single ascending dose of RP-A701 in up to 2 consecutive cohorts

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Rocket Pharmaceuticals Inc.: lead

Study Sites (3)

University of California, San Diego

San Diego, California, 92037, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

MeSH Terms

Conditions

Cardiomyopathy, Dilated; Cardiomyopathies; Dilatation, Pathologic; Heart Failure; Cardiovascular Diseases; Heart Diseases

Condition Hierarchy (Ancestors)

Cardiomegaly; Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Clinical Information

CONTACT

646-627-0033; clinicaltrials@rocketpharma.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The single-group study will be composed of up to two sequential dose cohorts

Study Record Dates

• First Submitted: July 25, 2025
• First Posted: August 22, 2025
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): June 1, 2029
• Last Updated: May 7, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)