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)
Development Status	Active
Indication	PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Disease Ontology Term	DOID:0050431
Compound Name	RP-A601
Compound Description	AAVrh.74-PKP2a
Sponsor	Rocket Pharmaceuticals Inc.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	9 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	PKP2
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	none
Dose 1	Starting dose: 8E13 GC/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
Recent Updates	Preliminary data from the Phase 1 study is expected in the first half of 2025