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

NCTID	NCT05885412 (View at clinicaltrials.gov)
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. (Show More)
Indication	Arrhythmogenic Right Ventricular Cardiomyopathy
Compound Name	RP-A601 (AAVrh.74-PKP2a)
Sponsor	Rocket Pharmaceuticals Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	9

Target Gene/Variant	PKP2A
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh.74
Editor Type
Dose 1	Starting dose: 8 x 10^13 GC/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States

Has US IND	True
Recent Updates	5/2024: Received Orphan Medicial Product designation from EMA