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Gene Therapy

RP-A601 for Arrhythmogenic Cardiomyopathy

Phase 1
Recruiting
Led By Barry Greenberg, MD
Research Sponsored by Rocket Pharmaceuticals Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
Male or female ≥18 years at the time of signing the informed consent
Must not have
Severe Right ventricular (RV) dysfunction
New York Heart Association (NYHA) Class IV heart failure
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months post-infusion
Awards & highlights
No Placebo-Only Group

Summary

This trial will test the safety and effectiveness of a new drug in high-risk adults with a specific condition.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Since this is an early-phase trial for RP-A601, potential side effects are being studied; however, common risks may include reactions at the infusion site, flu-like symptoms, immune responses or worsening of underlying conditions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with ACM according to the 2010 criteria.
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I am 18 years old or older.
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My tests show a specific genetic change in the PKP2 gene.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My heart's right side is not working properly.
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I have the most severe form of heart failure.
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My heart condition is genetic but not caused by the PKP2 gene.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months post-infusion
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months post-infusion for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Evaluation of safety associated with RP-A601
Secondary study objectives
Preliminary efficacy of RP-A601 - Cardiac biomarkers
Preliminary efficacy of RP-A601 - Myocardial PKP2 protein expression
Preliminary efficacy of RP-A601 - Ventricular ectopy and arrhythmia

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: RP-A601Experimental Treatment1 Intervention
Single ascending dose of RP-A601 in 2 consecutive cohorts

Find a Location

Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor
16 Previous Clinical Trials
417 Total Patients Enrolled
Barry Greenberg, MDPrincipal InvestigatorUniversity of California, San Diego
6 Previous Clinical Trials
487 Total Patients Enrolled

Media Library

RP-A601 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05885412 — Phase 1
Arrhythmogenic Cardiomyopathy Clinical Trial 2023: RP-A601 Highlights & Side Effects. Trial Name: NCT05885412 — Phase 1
RP-A601 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05885412 — Phase 1
Arrhythmogenic Cardiomyopathy Research Study Groups: RP-A601
~3 spots leftby Sep 2025
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