NCT01966887

AAV1-CMV-Serca2a GENe Therapy Trial in Heart Failure

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Assistance Publique - Hôpitaux de Paris · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

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

Interventions

Type	Name	Description
GENETIC	MYDICAR-single intracoronary infusion	AAV1/Serca2a
GENETIC	Placebo; single intracoronary infusion	single intracoronary infusion

Timeline

• Start date: 2013-12-01
• Primary completion: 2015-09-01
• Completion: 2016-02-01
• First posted: 2013-10-22
• Last updated: 2017-08-29

Locations

1 site across 1 country: France