NCT05885412 A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
| NCT ID | NCT05885412 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Rocket Pharmaceuticals Inc. |
| Condition | PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) |
| Study Type | INTERVENTIONAL |
| Enrollment | 9 participants |
| Start Date | 2023-08-29 |
| Primary Completion | 2028-09 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 9 participants in total. It began in 2023-08-29 with a primary completion date of 2028-09.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Key Inclusion Criteria: 1. Male or female ≥18 years at the time of signing the informed consent 2. Capable and willing to provide signed informed consent 3. Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC) 4. Documentation of a pathogenic or likely pathogenic truncating variant in PKP2 5. History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment 6. PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring 7. Left ventricular ejection fraction by echocardiogram or CMR ≥50% Key Exclusion Criteria: 1. Anti-AAVrh.74 capsid neutralizing antibody titer of \>1:40 2. Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant 3. Previous participation in a study of gene transfer or gene editing 4. Severe Right Ventricular (RV) dysfunction 5. New York Heart Association (NYHA) Class IV heart failure.
Contact & Investigator
Barry Greenberg, MD
PRINCIPAL INVESTIGATOR
University of California, San Diego
Frequently Asked Questions
Who can join the NCT05885412 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05885412 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05885412 currently recruiting?
Yes, NCT05885412 is actively recruiting participants. Contact the research team at clinicaltrials@rocketpharma.com for enrollment information.
Where is the NCT05885412 trial being conducted?
This trial is being conducted at La Jolla, United States, Durham, United States, Philadelphia, United States.
Who is sponsoring the NCT05885412 clinical trial?
NCT05885412 is sponsored by Rocket Pharmaceuticals Inc.. The principal investigator is Barry Greenberg, MD at University of California, San Diego. The trial plans to enroll 9 participants.