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
- Are there results?
- Not yet: the study is under way
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.
- More trials run by Rocket Pharmaceuticals Inc.The sponsor's other registered studies.
- 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
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT07137338
- Acronym / sponsor ID
- RP-A701-0125
- Conditions
- Dilated Cardiomyopathy (DCM)
- Keywords
- Cardiomyopathy, Dilated, BAG3 Protein, human, dilated cardiomyopathy, DCM, BAG3, BCL2-associated Athanogene 3
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Rocket Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Apr 2026 (actual)
- Primary completion
- Jun 2029 (estimated)
- Study completion
- Jun 2029 (estimated)
- First posted
- 22 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The single-group study will be composed of up to two sequential dose cohorts
| Arm | Type | Receives |
|---|---|---|
| Single ascending dose of RP-A701 in up to 2 consecutive cohorts | Experimental | 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)Participants will receive a single intravenous dose of RP-A701 on Day 0 and will be followed for up to two years |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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:
- Documentation of a pathogenic or likely pathogenic variant in BAG3
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Genetic | Single ascending dose of RP-A701 in up to 2 consecutive cohorts |
Primary outcome
Incidence of Treatment-emergent Adverse Events (TEAE)
Time frame: 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).
Time frame: 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).
Time frame: 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
6 secondary outcomes
To assess the impact of RP-A701 on features of cardiovascular function.
Time frame: Baseline up to End of Study (up to 24 months post-infusion)
To assess the impact of RP-A701 on features of cardiovascular function.
Time frame: Baseline up to End of Study (up to 24 months post-infusion)
To assess the impact of RP-A701 on features of cardiovascular function.
Time frame: Baseline up to End of Study (up to 24 months post-infusion)
To assess the extent of RP-A701 transduction and protein expression.
Time frame: 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).
Time frame: Baseline up to End of Study (up to 24 months post-infusion)
To assess the impact of RP-A701 on quality of life.
Time frame: Baseline up to End of Study (up to 24 months post-infusion)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Information (646-627-0033, clinicaltrials@rocketpharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-emergent Adverse Events (TEAE) (time frame: Baseline up to End of Study (up to 24 months post-infusion)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2026; AzTrials last refreshed its copy on 6 Nov 2026.