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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
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
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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
Recruiting
(Click here for study contact information)
Enrollment Count
150
Results Posted
Not Available
Therapy Information
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
3.25E13 vg
Dose 2
6.5E13 vg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase2
Submit Date
2022-10-25
Completion Date
2030-12-31
Last Update
2025-04-09
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
43
Locations
Austria,Netherlands,United States,United Kingdom,Germany,Spain
Regulatory Information
Has US IND
True
FDA Designations
Fast Track
Recent Updates
Received Fast Track designation 4/18/24
Resources/Links
Clinical Publications
(Presentation) Cardiovascular Gene Therapy: Efficient gene delivery to the heart? What are the best targets - ESC Meeting, February 2024
News and Press Releases
AskBio receives FDA Fast Track Designation for AB-1002 investigational gene therapy program in congestive heart failure
Preclinical Publications
Cardiac I-1c overexpression with reengineered AAV improves cardiac function in swine ischemic heart failure
Cardiac AAV9-S100A1 gene therapy rescues post-ischemic heart failure in a preclinical large animal model
AAV9.I-1c delivered via direct coronary infusion in a porcine model of heart failure improves contractility and mitigates adverse remodeling
Protein Phosphatase Inhibitor-1 Gene Therapy in a Swine Model of Nonischemic Heart Failure
AAV-9 mediated phosphatase-1 inhibitor-1 overexpression improves cardiac contractility in unchallenged mice but is deleterious in pressure-overload
Protocol
GenePHIT phase 2 study design: a double blind, placebo-controlled trial to assess efficacy, safety, and tolerability of AB-1002 gene therapy in adults with heart failure
Related NCTID
Phase 1: NCT04179643