NCT05598333

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

A Phase 2, Adaptive, Double-blinded, Placebo Controlled, Randomized, Multi-center Trial to Evaluate the Efficacy, Safety and Tolerability of Intracoronary Infusion of AB-1002 in Adult Subjects With New York Heart Association (NYHA) Class III Heart Failure and Non-ischemic Cardiomyopathy

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 150 (estimated)
• Sponsor: AskBio Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

Subjects will be randomized into one of three treatment groups in a 1:1:1 fashion to either 7.15E13vg (n=30-50), 1.43E14 (n=30-50) or placebo group (n=30-50). Approximately 90 to 150 subjects will be randomly assigned to study intervention Placebo Study duration until the primary analysis at 52 week will be approximately 37 months including 25 months of recruitment and 52-week Observation Period after dosing. Once all the subjects complete the 52 weeks Observation Period, the treatment groups will be unblinded and primary analysis performed. Study participation duration: The study will last 52 weeks from dosing, with another 4 years of long-term follow-up for a total of 5 years. During the 4 year long-term follow up sites will contact subjects twice a year for two years, then once a year for the remaining two years for safety, efficacy assessments, and survival

Conditions

• Congestive Heart Failure

Interventions

Type	Name	Description
DRUG	AB-1002	Intracoronary Infusion of AB-1002 or placebo

Timeline

• Start date: 2023-10-20
• Primary completion: 2026-10-31
• Completion: 2030-12-31
• First posted: 2022-10-28
• Last updated: 2025-12-03

Locations

96 sites across 12 countries: United States, Austria, Belgium, Bulgaria, Canada, Germany, Hungary, Netherlands, Poland, Romania, Spain, United Kingdom

Regulatory

• FDA-regulated drug study