Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects with Non-Ischemic NYHA Class III Heart Failure

NCTID	NCT05598333 (View at clinicaltrials.gov)
Description	This is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center trial study to evaluate the safety and efficacy of a single dose of AB-1002, administered via antegrade intracoronary artery infusion, in males and females age \>18 years with non-ischemic cardiomyopathy and NYHA Class III symptoms of HF. Subjects will be randomized into one of three treatment groups in a 1:1:1 (Show More)
Indication	Congestive Heart Failure
Compound Name	AB-1002 (BNP116.sc-CMV.I1c)
Sponsor	Asklepios Biopharmaceutical, Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	150

Target Gene/Variant	PPP1R1A
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intraarterial
Drug Product Type	Viral vector
Target Tissue/Cell	Cardiomyocyte
Delivery System	Viral transduction
Vector Type	AAV2i8
Editor Type
Dose 1	3.25 x 10^13 vg
Dose 2	6.5 x 10^13 vg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	28
Locations	United States

Has US IND	True
Recent Updates	Received Fast Track designation 4/18/24