Dilated Cardiomyopathy (DCM)Phase 1NCT07137338
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
A Phase 1 study testing 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) in people with Dilated Cardiomyopathy (DCM), run by Rocket Pharmaceuticals Inc. at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Dilated Cardiomyopathy (DCM)
- What is being tested?
- 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)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 3 sitesUnited States
- Study dates
- 17 Apr 2026to Jun 2029
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects are eligible for inclusion into the study only if all the following criteria apply:
- 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
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- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
Clinical Res, 843-792-2300, andrewcl@musc.edu
Daniel Judge, MD
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Clinical Research Coordinator, 507-538-1500, slade.sierra@mayo.edu
John Giudicessi, MD, PhD
- Recruiting
University of California, San Diego
San Diego, California, 92037
Clinical Research Coordinator, 858-822-1722, emoyacespedes@health.ucsd.edu
Barry Greenberg, MD
About this study
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)
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)
- Being tested
- 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)
- 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
- Medical subject headings
- Cardiomyopathy, Dilated, Cardiomyopathies, Dilatation, Pathologic, Heart Failure, Cardiovascular Diseases, Heart Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-emergent Adverse Events (TEAE)
Number of participants with Adverse Events following a single IV dose of RP-A701
Measured: 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
Measured: 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
Measured: 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.
Change in measures of LV systolic function, including LV ejection fraction (LVEF), LV global longitudinal strain.
Measured: Baseline up to End of Study (up to 24 months post-infusion)
To assess the impact of RP-A701 on features of cardiovascular function.
Change in peak oxygen consumption (VO2) or ventilatory efficiency (VE/VCO2 slope) by cardiopulmonary exercise test (CPET)
Measured: Baseline up to End of Study (up to 24 months post-infusion)
To assess the impact of RP-A701 on features of cardiovascular function.
Change in 6-minute walk distance.
Measured: Baseline up to End of Study (up to 24 months post-infusion)
To assess the extent of RP-A701 transduction and protein expression.
Change in BAG3 myocardial protein expression
Measured: 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).
Change in symptoms of HF assessed by New York Heart Association (NYHA) class.
Measured: Baseline up to End of Study (up to 24 months post-infusion)
To assess the impact of RP-A701 on quality of life.
Change in Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score.
Measured: Baseline up to End of Study (up to 24 months post-infusion)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2025
- Study start17 Apr 2026
- Primary completionJun 2029 (estimated)
- Study completionJun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dilated Cardiomyopathy (DCM)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- The single-group study will be composed of up to two sequential dose cohorts
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07137338
- Sponsor's protocol ID
- RP-A701-0125
- Status last verified
- Sep 2026
- First submitted
- 25 Jul 2025
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No