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Phase 1 Gene Therapy for BAG3 Dilated Cardiomyopathy

This Phase 1 study is testing a new gene therapy called RP-A701 for people with Dilated Cardiomyopathy (DCM) caused by a problem with the BAG3 gene. RP-A701 is a one-time treatment given through an IV. The main goals of this study are to see how safe RP-A701 is and if it causes any side effects. Researchers will also look for early signs that the treatment might be working. You may be able to join if you are between 18 and 65 years old and have a clinical diagnosis of DCM. The study plans to enroll 8 participants.

Study design
This is a Phase 1, open-label study, meaning both you and the study team will know what treatment you are receiving. It will involve a small number of participants (8 planned) to test different doses of RP-A701.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
You will be followed for up to 24 months after receiving the treatment to monitor for any side effects.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07137338

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

Recruiting
PHASE1Ages 18–65InterventionalTreatment
Rocket Pharmaceuticals Inc.
~8 participants
Updated 2026-06-30 on ClinicalTrials.gov
What's 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)

At a glance

Recruiting sites
3 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of Treatment-emergent Adverse Events (TEAE)
Measured over Baseline up to End of Study (up to 24 months post-infusion)
+2 more outcomes measured

Conditions

Dilated Cardiomyopathy (DCM)

Explore related trials

Dilated CardiomyopathyBAG3 Gene

Where it's being run

3 sites across 3 states
California1
Minnesota1
South Carolina1

Who to contact

Clinical Information
[email protected]
646-627-0033
Email the study team

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What this trial measures

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