Actively Recruiting
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Led by Rocket Pharmaceuticals Inc. · Updated on 2026-04-16
9
Participants Needed
3
Research Sites
313 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.
CONDITIONS
Official Title
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female 18 years or older at the time of signing informed consent
- Able and willing to provide signed informed consent
- Clinical diagnosis of Arrhythmogenic Cardiomyopathy by 2010 revised Task Force Criteria
- Documented pathogenic or likely pathogenic truncating variant in PKP2
- Implantable Cardioverter-Defibrillator (ICD) implanted at least 6 months before enrollment
- Premature ventricular contraction (PVC) frequency of 500 or more per 24 hours by ambulatory rhythm monitoring
- Left ventricular ejection fraction of 50% or higher by echocardiogram or cardiac MRI
You will not qualify if you...
- Neutralizing antibody titer against AAVrh.74 capsid greater than 1:40
- Cardiomyopathy caused by genetic factors other than PKP2 truncating variant
- Previous participation in gene transfer or gene editing studies
- Severe dysfunction of the right ventricle
- New York Heart Association Class IV heart failure
AI-Screening
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Trial Site Locations
Total: 3 locations
1
University of California, San Diego
La Jolla, California, United States, 92037
Actively Recruiting
2
Duke University
Durham, North Carolina, United States, 27710
Actively Recruiting
3
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Withdrawn
Research Team
C
Clinical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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