Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
RECRUITING
NCT05885412
PHASE1

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

Sponsor: Rocket Pharmaceuticals Inc.

View on ClinicalTrials.gov

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.

Official title: 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)

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

9

Start Date

2023-08-29

Completion Date

2026-09

Last Updated

2024-10-04

Healthy Volunteers

No

Interventions

GENETIC

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)

Locations (3)

University of California, San Diego

La Jolla, California, United States

Duke University

Durham, North Carolina, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States