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

Purpose

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.

Condition

  • Dilated Cardiomyopathy (DCM)

Eligibility

Eligible Ages
Between 18 Years and 65 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 1
Study Type
Interventional
Allocation
N/A
Intervention Model
Sequential Assignment
Intervention Model Description
The single-group study will be composed of up to two sequential dose cohorts
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Single ascending dose of RP-A701 in up to 2 consecutive cohorts
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)
    One-time treatment with a single ascending dose

Recruiting Locations

University of California, San Diego
San Diego, California 92037
Contact:
Clinical Research Coordinator
858-246-5442
c2olandt@health.ucsd.edu

Mayo Clinic
Rochester, Minnesota 55905
Contact:
Clinical Research Coordinator
507-538-1500
slade.sierra@mayo.edu

Medical University of South Carolina
Charleston, South Carolina 29425
Contact:
Clinical Res
843-792-2300
andrewcl@musc.edu

More Details

Status
Recruiting
Sponsor
Rocket Pharmaceuticals Inc.

Study Contact

Clinical Information
646-627-0033
clinicaltrials@rocketpharma.com