Actively Recruiting
PKP2-ACM Natural History Study
Led by Rocket Pharmaceuticals Inc. · Updated on 2026-02-27
36
Participants Needed
3
Research Sites
265 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The goal of this study is to describe the natural history and clinical events for patients who have Arrhythmogenic Cardiomyopathy with Pathogenic Plakophilin-2 Variants (PKP2-ACM) managed with standard of care.
CONDITIONS
Official Title
PKP2-ACM Natural History Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female age 12 years or older at the time of providing informed consent.
- Able and willing to provide signed informed consent or assent and comply with study requirements.
- Clinical diagnosis of arrhythmogenic cardiomyopathy (ACM).
- Documentation of a pathogenic or likely pathogenic variant in PKP2 by a certified genetic test.
- History of implantable cardioverter defibrillator (ICD) implantation 6 months or more before consent.
- Left ventricular ejection fraction of 50% or greater by echocardiogram or cardiac MRI within 12 months before consent.
You will not qualify if you...
- Genetic testing shows the arrhythmia or cardiomyopathy may be caused by a gene other than PKP2 truncating variant.
- Currently participating in another clinical trial involving investigational agents that could affect this study.
- Previous participation in gene transfer or gene editing studies.
- New York Heart Association (NYHA) Class IV heart failure.
- Need for mechanical circulatory support or heart transplantation within 6 months of enrollment.
- Prior cardiac or other organ transplantation.
- Pacemaker-dependent rhythm within 12 months before enrollment.
- Positive test for human immunodeficiency virus (HIV) antibody.
- Unwillingness or inability to comply with study procedures or follow-up.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 3 locations
1
Boston Children's Hospital
Boston, Massachusetts, United States, 02116
Actively Recruiting
2
Duke University
Durham, North Carolina, United States, 27710
Actively Recruiting
3
Amsterdam UMC
Amsterdam, Netherlands, 1105 AZ
Not Yet Recruiting
Research Team
C
Clinical Information
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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