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

Full Name

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

Description

Although current management has improved survival in CHF patients, most therapies do not treat the underlying causes; consequently, the current standard of care does not reverse the trajectory of the disease to ultimate end stage heart failure and death. In the US, CHF has a 10% annual mortality rate and approximately 50% mortality at 5 years. In addition, CHF causes significant health economic burdens to patients, families, and healthcare systems due to the frequent recurrent hospitalization and intensive care that these patients require. Thus, there is an urgent need for therapies with the potential to reverse the course of ventricular dysfunction in CHF.

AB-1002 is a gene therapy product being developed as a one-time intracoronary infusion for the treatment of subjects with Congestive Heart Failure (CHF), a leading cause of morbidity and mortality in Westernized countries.

Purpose

The primary objective of this study is to evaluate the efficacy and safety of a single antegrade intracoronary artery infusion of AB-1002 compared to placebo infusion in subjects with non-ischemic cardiomyopathy and NYHA Class III symptoms of heart failure. Subjects will be randomly assigned by a computer to receive either a higher dose of AB-1002 or a lower dose of AB-1002 or placebo.

Eligibility

Inclusion Criteria

Inclusion Criteria-including but may not be limited to:

Subject must be age ≥18 years of age, at the time of signing the informed consent.
Chronic non-ischemic cardiomyopathy
15% ≤ LVEF ≤ 35% by transthoracic echocardiography (TTE) at screening

Exclusion Criteria

Chronic ischemic cardiomyopathy secondary to obstructive coronary artery disease
Restrictive cardiomyopathy, obstructive cardiomyopathy, pericardial disease, amyloidosis, infiltrative cardiomyopathy, uncorrected thyroid disease, or dyskinetic LV aneurysm
Any documented history of non-compliance with medications, illicit drug use or laboratory evidence of illicit drug use during screen period
Known hypersensitivity to contrast dyes (not easily controlled by antihistamines) used for angiography