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

Not Applicable

Not yet recruiting

• Conditions: Dilated Cardiomyopathy (DCM)

• Registration Number: NCT07137338
• Lead Sponsor: Rocket Pharmaceuticals Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-07-25
• Status Verified: 2025-08
• Study First Submitted QC: 2025-08-20
• Last Update Submitted: 2025-08-20
• Study First Posted: 2025-08-22
• Last Update Posted: 2025-08-22
• Study Start: 2026-06-01
• Primary Completion: 2029-06
• Study Completion: 2029-06-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 8

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 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-emergent Adverse Events (TEAE)	Baseline up to End of Study (up to 24 months post-infusion)	Number of participants with Adverse Events following a single IV dose of RP-A701
Incidence of Treatment-emergent Serious Adverse Events (SAE).	Baseline up to End of Study (up to 24 months post-infusion)	Number of participants with Serious Adverse Events (SAE) following a single IV dose of RP-A701
Incidence of Dose Limiting Toxicities (DLT).	Baseline up to End of Study (up to 24 months post-infusion)	Number of participants with Dose Limiting Toxicities (DLT) following a single IV dose of RP-A701

Secondary Outcome Measures

Name	Time	Method
To assess the impact of RP-A701 on features of cardiovascular function.	Baseline up to End of Study (up to 24 months post-infusion)	Change in 6-minute walk distance.
To assess the extent of RP-A701 transduction and protein expression.	Baseline up to End of Study (up to 24 months post-infusion)	Change in BAG3 myocardial protein expression
To assess the impact of RP-A701 on quality of life.	Baseline up to End of Study (up to 24 months post-infusion)	Change in Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score.
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)	Change in symptoms of HF assessed by New York Heart Association (NYHA) class.