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RecruitingNCT07137338Updated Oct 1, 2026

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

A Phase 1 interventional study of 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 Dilated Cardiomyopathy (DCM), sponsored by Rocket Pharmaceuticals Inc.. Recruiting at 3 sites in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-10-01.

Sponsored by Rocket Pharmaceuticals Inc. · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Started Apr 2026; still recruiting 6 months later.
Updated Oct 1, 2026Start date movedGo to Updates ↓
Phase
Phase 1
Study type
Interventional
Enrollment
8
Allocation
Not applicable
Ages
18 Years to 65 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Dilated Cardiomyopathy (DCM)

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
03

In context

Cardiomyopathy, Dilated

255 studies on the registry are indexed under Cardiomyopathy, Dilated; 66 are open to participants now.

This study's planned enrollment of 8 is below the median of 43 across 145 interventional studies indexed under Cardiomyopathy, Dilated.

Browse Cardiomyopathy, Dilated studies →

Lead sponsor

Rocket Pharmaceuticals Inc. is the lead sponsor of 19 studies on the registry; 7 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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
  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 ( ≥ 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. Class II or III HF symptoms with stable HF therapeutic guideline-directed medical regimen for 30 days prior to enrollment

Exclusion criteria

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. \< 25% by echocardiogram or CMR at ≤ 3 months prior to screening
  7. Class I or IV HF
Terms in these criteria, in plain language(3)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

LVEF (left ventricular ejection fraction)

The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.

NYHA class (heart failure)

The New York Heart Association’s four classes of how much heart failure limits physical activity.

I
No limits: ordinary activity causes no undue tiredness or breathlessness.
II
Slight limits: comfortable at rest, ordinary activity causes symptoms.
III
Marked limits: comfortable at rest, less than ordinary activity causes symptoms.
IV
Symptoms even at rest; any activity makes them worse.

Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
8 participants (estimated)

Study arms

  • 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)

Interventions

  • GeneticRP-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

06

What researchers measure

Primary outcomes

  1. Incidence of Treatment-emergent Adverse Events (TEAE)

    Number of participants with Adverse Events following a single IV dose of RP-A701

    Time frame: Baseline up to End of Study (up to 24 months post-infusion)

  2. Incidence of Treatment-emergent Serious Adverse Events (SAE).

    Number of participants with Serious Adverse Events (SAE) following a single IV dose of RP-A701

    Time frame: Baseline up to End of Study (up to 24 months post-infusion)

  3. Incidence of Dose Limiting Toxicities (DLT).

    Number of participants with Dose Limiting Toxicities (DLT) following a single IV dose of RP-A701

    Time frame: Baseline up to End of Study (up to 24 months post-infusion)

Secondary outcomes

  1. 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.

    Time frame: Baseline up to End of Study (up to 24 months post-infusion)

  2. 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)

    Time frame: Baseline up to End of Study (up to 24 months post-infusion)

  3. To assess the impact of RP-A701 on features of cardiovascular function.

    Change in 6-minute walk distance.

    Time frame: Baseline up to End of Study (up to 24 months post-infusion)

  4. To assess the extent of RP-A701 transduction and protein expression.

    Change in BAG3 myocardial protein expression

    Time frame: Baseline up to End of Study (up to 24 months post-infusion)

  5. 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.

    Time frame: Baseline up to End of Study (up to 24 months post-infusion)

  6. To assess the impact of RP-A701 on quality of life.

    Change in Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score.

    Time frame: Baseline up to End of Study (up to 24 months post-infusion)

07

Study locations

3 of 3 sites recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedDate before it movedWhere the date was before it moved

Started Apr 2026 (actual). Primary completion is estimated for Jun 2029, 38 months after the start.

Start moved once since we started tracking on Sep 25, 2026: Jun 2026 → Apr 2026, on Oct 1, 2026.

Earlier changes are on the record history on ClinicalTrials.gov ↗

How these figures are worked out, and which studies are left out of them.

Registered
Jul 25, 2025
First posted
Aug 22, 2025
StartActual
Apr 17, 2026was Jun 2026
Primary completionEst.
Jun 2029
CompletionEst.
Jun 2029
Last update
Oct 1, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Start date
Jun 2026→Apr 17, 2026 (actual)
Oct 1, 2026
Show all 1 update
  1. Oct 1, 2026
    Start date Jun 2026→Apr 17, 2026 (now actual)
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07137338
Lead sponsor
Rocket Pharmaceuticals Inc.
Responsible party
Sponsor
First posted
Aug 22, 2025
Start date
Apr 17, 2026
Primary completion
Jun 2029 (estimated)
Completion
Jun 2029 (estimated)
Last update
Oct 1, 2026

Study contacts

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

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Rocket Pharmaceuticals Inc.

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