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New treatment option for PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Official title A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
ClinicalTrials.gov ID: NCT05885412
What this study is testing
What is RP-A601?
RP-A601 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for pkp2 arrhythmogenic cardiomyopathy (pkp2-acm).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Male or female ≥18 years at the time of signing the informed consent
- Capable and willing to provide signed informed consent
- Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
- Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
- History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment
You likely can't join if
- Anti-AAVrh.74 capsid neutralizing antibody titer of \>1:40
- Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
- Previous participation in a study of gene transfer or gene editing
- Severe Right Ventricular (RV) dysfunction
- New York Heart Association (NYHA) Class IV heart failure.
See the full eligibility criteria
- Male or female ≥18 years at the time of signing the informed consent
- Capable and willing to provide signed informed consent
- Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
- Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
- History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment
- PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring
- Left ventricular ejection fraction by echocardiogram or CMR ≥50% Key
- Anti-AAVrh.74 capsid neutralizing antibody titer of \>1:40
- Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
- Previous participation in a study of gene transfer or gene editing
- Severe Right Ventricular (RV) dysfunction
- New York Heart Association (NYHA) Class IV heart failure.
The study team makes the final eligibility decision.
Where it's taking place
- La Jolla, California, United States
- Durham, North Carolina, United States
- Philadelphia, Pennsylvania, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include La Jolla, California, United States; Durham, North Carolina, United States; Philadelphia, Pennsylvania, United States. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.