AAV1-CMV-Serca2a GENe Therapy Trial in Heart Failure — AGENT-HF  

Ischemic Cardiomyopathy Clinical Trial

Official title: Phase 2 Study of SERCA2a Gene Transfer in Patients With Severe Heart Failure

Status Terminated

Clinical Trial Summary

The purpose of this study is to determine the effect of intracoronary SERCA2a Gene transfer on cardiac volumes and function using multimodality cardiac imaging.

Clinical Trial Description

The purpose of gene transfer of SERCA2a is to improve systolic and diastolic function of the failing ventricle. Studies show that reduction of SERCA2a in failing ventricle is a key factor in depression of contraction, and that restoration of SERCA2a levels can improve left ventricular function and remodeling. The aim of the study is to investigate the effect of an adeno-associated viral vector expressing the sarcoplasmic reticulum calcium ATPase (SERCA2a), driven by the CMV promoter (AAV1-CMV-SERCA2a), on the ventricular remodeling of patients with severe heart failure using multimodality cardiac imaging. This is a Phase 2 monocenter double blind randomized placebo-controled, parallel study. The study will enroll 44 symptomatic heart failure patients with NYHA IIIb/IV, with left-ventricular ejection fraction of 35% or less receiving an optimal standard medical therapy. The absence of neutralizing antibodies against AAV1 will be primarily checked. Seronegative patients will be randomized to receive either 1x10e13 AAV1-CMV-Serca2a or a placebo as a single intracoronary infusion. Evolution during the next 6 months of the left ventricular end-systolic volume (measured with a 256-slices CT-scan before injection and 6 months later) will be the primary endpoint. Secondary endpoints will include changes in the LVEF, diastolic volumes, VO2max, Echocardiographic remodeling, BNP, cardiac hemodynamics and biological safety profile. ;

Related Conditions & MeSH terms

• Cardiomyopathies
• Heart Failure
• Heart Failure Congestive
• Ischemic Cardiomyopathy
• Non-ischemic Cardiomyopathy

• NCT number: NCT01966887
• Study type: Interventional
• Source: Assistance Publique - Hôpitaux de Paris
• Status: Terminated
• Phase: Phase 2
• Start date: December 2013
• Completion date: February 2016