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)
Development Status	Active
Indication	Congestive Heart Failure
Disease Ontology Term	DOID:6000
Compound Name	AB-1002
Compound Description	BNP116.sc-CMV.I1c
Sponsor	AskBio Inc
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	150 (ESTIMATED)
Results Posted	Not Available

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	none
Dose 1	Phase 1: 3.25E13 vg
Dose 2	Phase 1: 1.08E14 vg
Dose 3	Phase 2: 7.15E13 vg
Dose 4	Phase 2: 1.43E14 vg
Dose 5

No.of Trial Sites	96
Locations	Canada,Austria,Netherlands,Romania,Belgium,Hungary,United States,Poland,United Kingdom,Bulgaria,Germany,Spain

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