Recently added Catalysts

RP-A701

Phase 1

Dilated Cardiomyopathy (DCM) | Gene therapy | Cardiovascular |Rocket Pharmaceuticals, Inc.|Last Updated: Jun 30, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC

Total Trials1

Total Enrollment8

FDA Designations

No designations recorded

Clinical trial landscape

RP-A701 · 1 trial · 1 indication

Phase 1 1

NCT07137338A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated CardiomyopathyDilated Cardiomyopathy (DCM)

RECRUITING8 Analytics

PHASE1RECRUITING

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

Dilated Cardiomyopathy (DCM)

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Study Endpoints

Primary Endpoints

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

Secondary Endpoints

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

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

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

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).

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

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Study Design & Arms

AllocationNA

MaskingNONE

ModelSEQUENTIAL

PurposeTREATMENT

Treatment Arms

Arm	Type	Description
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

Interventions

Name	Type	Description
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

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Eligibility Criteria

Age Range18 Years to 65 Years

SexALL

Healthy VolunteersNo

Study Sites3

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 informed consent 2. Capable of and willing to provide signed informed consent 3. Clinical diagnosis of DCM de...

Countries:United States

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Recent Changes (Last 90 Days)

LOWJun 30, 2026NCT07137338lastUpdatePostDate: changed

LOWJun 30, 2026NCT07137338lastUpdatePostDate: changed

LOWJun 30, 2026NCT07137338lastUpdatePostDate: changed