Clinical trial

A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)

Name

RP-A601-0323

Description

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.

Trial arms

Trial start

2023-08-29

Estimated PCD

2025-09-01

Trial end

2026-09-01

Status

Recruiting

Phase

Early phase I

Treatment

RP-A601

RP-A601 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, human plakophilin 2 (PKP2), transcript variant 2a (PKP2a)

Arms:

RP-A601

Size

Primary endpoint

Incidence of treatment emergent adverse events (TEAEs)

12 months post-infusion

Incidence of Serious Adverse Events (SAEs)

12 months post-infusion

Identification of Dose Limiting Toxicities (DLTs)

12 months post-infusion

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. Anti-AAVrh.74 capsid neutralizing antibody assay ≤1:40 6. History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment Key Exclusion Criteria: 1. Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant 2. Previous participation in a study of gene transfer or gene editing 3. Severe Right ventricular (RV) dysfunction 4. Left ventricular ejection fraction by echocardiogram ≤50% 5. New York Heart Association (NYHA) Class IV heart failure.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 9, 'type': 'ESTIMATED'}}

Updated at

2023-10-30

1 organization

1 product

1 indication

Product

RP-A601

Organization

Rocket Pharmaceuticals

Indication

PKP2-ACM