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A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
NCT07137338 · Rocket Pharmaceuticals Inc.
In plain English
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Official title
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)
About this study
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.
Eligibility criteria
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
Enrollment target: 8 participants
Allocation: na
Masking: none
Age groups: adult, older_adult
Timeline
Starts: 2026-06
Estimated completion: 2029-06
Last updated: 2026-04-02
Interventions
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)
Primary outcomes
- • Incidence of Treatment-emergent Adverse Events (TEAE) (Baseline up to End of Study (up to 24 months post-infusion))
- • Incidence of Treatment-emergent Serious Adverse Events (SAE). (Baseline up to End of Study (up to 24 months post-infusion))
- • Incidence of Dose Limiting Toxicities (DLT). (Baseline up to End of Study (up to 24 months post-infusion))
Sponsor
Rocket Pharmaceuticals Inc. · industry
Contacts & investigators
ContactClinical Information · contact · clinicaltrials@rocketpharma.com · 646-627-0033
All locations (3)
University of California, San DiegoRecruiting
San Diego, California, United States
Mayo ClinicNot Yet Recruiting
Rochester, Minnesota, United States
Medical University of South CarolinaNot Yet Recruiting
Charleston, South Carolina, United States