NCT05885412

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9

participants targeted

Target at below P25 for phase_1

Timeline
40mo left

Started Aug 2023

Longer than P75 for phase_1

Geographic Reach
1 country

3 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress45%
Aug 2023Sep 2029

First Submitted

Initial submission to the registry

May 22, 2023

Completed
11 days until next milestone

First Posted

Study publicly available on registry

June 2, 2023

Completed
3 months until next milestone

Study Start

First participant enrolled

August 29, 2023

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2028

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2029

Last Updated

April 16, 2026

Status Verified

April 1, 2026

Enrollment Period

5 years

First QC Date

May 22, 2023

Last Update Submit

April 14, 2026

Conditions

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Keywords

Arrhythmogenic cardiomyopathyArrhythmogenic Right Ventricular CardiomyopathyArrhythmogenic Right Ventricular DysplasiaSudden Cardiac DeathGenetic cardiomyopathyGene therapyPKP2ARVCARVDACMCardiac ArrestVentricular Arrhythmia

Outcome Measures

Primary Outcomes (1)

  • Evaluation of safety associated with RP-A601

    Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)

    12 months post-infusion

Secondary Outcomes (3)

  • Preliminary efficacy of RP-A601 - Myocardial PKP2 protein expression

    12 months post-infusion

  • Preliminary efficacy of RP-A601 - Ventricular ectopy and arrhythmia

    12 months post-infusion

  • Preliminary efficacy of RP-A601 - Cardiac biomarkers

    12 months post-infusion

Study Arms (1)

RP-A601

EXPERIMENTAL

Single ascending dose of RP-A601 in 2 consecutive cohorts

Genetic: RP-A601

Interventions

RP-A601GENETIC

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)

RP-A601

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify 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
  • PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring
  • Left ventricular ejection fraction by echocardiogram or CMR ≥50%

You may not qualify 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

University of California, San Diego

La Jolla, California, 92037, United States

RECRUITING

Duke University

Durham, North Carolina, 27710, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

WITHDRAWN

MeSH Terms

Conditions

Arrhythmogenic Right Ventricular DysplasiaDeath, Sudden, CardiacHeart Arrest

Condition Hierarchy (Ancestors)

Heart Defects, CongenitalCardiovascular AbnormalitiesCardiovascular DiseasesCardiomyopathiesHeart DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDeath, SuddenDeathPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Barry Greenberg, MD

    University of California, San Diego

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Clinical Information

CONTACT

646-627-0033clinicaltrials@rocketpharma.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 22, 2023

First Posted

June 2, 2023

Study Start

August 29, 2023

Primary Completion (Estimated)

September 1, 2028

Study Completion (Estimated)

September 1, 2029

Last Updated

April 16, 2026

Record last verified: 2026-04

Locations

US(3)