RecruitingPhase 1

A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

United States9 participantsStarted 2023-08-29

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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Inclusion 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%

Exclusion criteria

✕. 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.

What they're measuring

Evaluation of safety associated with RP-A601

Timeframe: 12 months post-infusion

Trial details

NCT IDNCT05885412

SponsorRocket Pharmaceuticals Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-09

Contact for this trial

Clinical Information

646-627-0033 clinicaltrials@rocketpharma.com

View on ClinicalTrials.gov More PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) trials