Trials / Recruiting
RecruitingNCT05885412
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
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)
- Status
- Recruiting
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 9 (estimated)
- Sponsor
- Rocket Pharmaceuticals Inc. · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| 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) |
Timeline
- Start date
- 2023-08-29
- Primary completion
- 2028-09-01
- Completion
- 2029-09-01
- First posted
- 2023-06-02
- Last updated
- 2026-04-16
Locations
3 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05885412. Inclusion in this directory is not an endorsement.