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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 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2026-05-07

No results posted yet for this 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.

Conditions

  • Dilated Cardiomyopathy (DCM)

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)

One-time treatment with a single ascending dose

Sponsors & Collaborators

  • Rocket Pharmaceuticals Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-30
Primary Completion
2029-06-30
Completion
2029-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07137338 on ClinicalTrials.gov