RP-A601 for Arrhythmogenic Cardiomyopathy

Recruiting at 2 trial locations

Overseen By Clinical Information

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Rocket Pharmaceuticals Inc.

Disqualifiers: Severe RV dysfunction, NYHA Class IV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 13

Duration: 1 day

9 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the safety and initial effectiveness of a new treatment called RP-A601 for people with arrhythmogenic cardiomyopathy (ACM). It specifically targets those whose ACM is linked to a gene problem in PKP2. Participants should have had an Implantable Cardioverter-Defibrillator (a device that helps prevent dangerous heart rhythms) for at least six months and experience frequent irregular heartbeats. The study involves administering a single dose of RP-A601 through an IV to observe the body's reaction and its potential benefits for the condition. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive it.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that RP-A601 is likely to be safe for humans?

Research has shown that RP-A601 is generally well-tolerated. In earlier studies, patients received a specific dose without experiencing severe side effects that would have halted the trial. Most reported side effects were mild. Some patients were monitored for up to 12 months, and safety remained positive during this period. This suggests that RP-A601 may be safe for humans, but further studies are needed to confirm these results. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard of care for arrhythmogenic cardiomyopathy, which often includes medications like beta-blockers, antiarrhythmics, or even implantable devices, RP-A601 is unique because it utilizes a novel gene therapy approach. Researchers are excited about RP-A601 because it targets the genetic root of the disease, potentially offering a more precise and long-lasting solution. By delivering a single dose that could modify the underlying genetic cause, this treatment has the potential to transform how we manage this challenging condition.

What evidence suggests that RP-A601 might be an effective treatment for Arrhythmogenic Cardiomyopathy?

Research has shown that RP-A601, the investigational treatment in this trial, may help treat arrhythmogenic cardiomyopathy, a disease affecting the heart muscle. In a previous study, all three patients who received this treatment experienced improved or stable heart function. This early evidence suggests that RP-A601 could effectively manage the condition. The treatment was generally well-tolerated, with no serious side effects reported. These findings offer hope for RP-A601 in combating this heart disease. ^{1 2 3 5 6}

Who Is on the Research Team?

Barry Greenberg, MD

Principal Investigator

University of California, San Diego

Are You a Good Fit for This Trial?

Adults over 18 with a specific heart condition called PKP2-ACM, who have had an ICD implanted for at least 6 months can join. They must have a certain genetic variant and low levels of antibodies against the treatment virus. People with severe heart issues, other genetic causes of cardiomyopathy, or those in advanced stages of heart failure cannot participate.

Inclusion Criteria

I have been diagnosed with ACM according to the 2010 criteria.

I am willing and able to sign the consent form.

My tests show a specific genetic change in the PKP2 gene.

Exclusion Criteria

My heart's right side is not working properly.

I have the most severe form of heart failure.

My heart condition is genetic but not caused by the PKP2 gene.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose intravenous infusion of RP-A601

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and preliminary efficacy, including cardiac biomarkers and myocardial PKP2 protein expression

12 months

Regular visits (in-person and virtual) over 12 months

What Are the Treatments Tested in This Trial?

Interventions

• RP-A601

Trial Overview The trial is testing RP-A601, given as a one-time intravenous infusion to see if it's safe and might help patients with PKP2-ACM. It starts with small doses that increase gradually to find the right balance between effectiveness and safety.

How Is the Trial Designed?

Group I: RP-A601 Experimental Treatment 1 Intervention

Single ascending dose of RP-A601 in 2 consecutive cohorts

RP-A601 is already approved in European Union, United States for the following indications:

Approved in European Union as RP-A601 for:

• PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) - Orphan Medicinal Product Designation

Approved in United States as RP-A601 for:

• PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) - Fast Track and Orphan Drug Designations

Who Is Running the Clinical Trial?

Rocket Pharmaceuticals Inc.

Lead Sponsor

Trials

17

Recruited

430+

Citations

1. ir.rocketpharma.com ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-presents-preliminary-data-phase-1

News Release Details RP-A601 was generally well-tolerated at a dose of 8.0E13 GC/kg with no dose-limiting toxicities in all three patients with up to 12 months follow-up.

2. academic.oup.com academic.oup.com/eurheartj/article/45/Supplement_1/ehae666.3643/7838341

Phase I gene therapy clinical trial design of RP-A601 in adult ... This Phase I trial will evaluate the safety and preliminary efficacy of RP-A601 and was initiated based on robust preclinical efficacy and safety data.

3. ir.rocketpharma.com ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-receives-fda-regenerative-medicine-3

News Release Details The safety profile was favorable, with RP-A601 generally well-tolerated, no dose-limiting toxicities, and most adverse events being mild or ...

4. cgtlive.com cgtlive.com/view/rocket-pkp2-arrhythmogenic-cardiomyopathy-gene-therapy-rp-a601-improves-stabilizes-heart-function-phase-1-trial

Rocket's PKP2-Arrhythmogenic Cardiomyopathy Gene ... All 3 patients received the same dose of RP-A601, 8x10 13 GC/kg. As of the April 2025 data cut off for efficacy, all 3 patients had shown ...

5. firstwordpharma.com firstwordpharma.com/story/5987427

Rocket Pharmaceuticals Reports Second Quarter 2025 ... Initial Phase 1 data demonstrating encouraging safety and preliminary efficacy outcomes of RP-A601 for PKP2-ACM were presented at ASGCT in May ...

6. finance.yahoo.com finance.yahoo.com/news/rocket-pharmaceuticals-presents-preliminary-data-191500936.html

Rocket Pharmaceuticals Presents Preliminary Data from ... RP-A601 was generally well-tolerated at a dose of 8.0E13 GC/kg with no dose-limiting toxicities in all three patients with up to 12 months ...