Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure

Summary

The study is divided into 2 parts. In the first part, the safety of the gene transfer agent MYDICAR® will be evaluated. In the second part, the ability of MYDICAR® to improve heart function will be studied.

Conditions

• Heart Failure, Congestive
• Dilated Cardiomyopathy

Interventions

GENETIC

MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study)

MYDICAR administered by antegrade epicardial coronary artery infusion

PROCEDURE

Placebo Infusion

Saline; epicardial coronary artery infusion

GENETIC

MYDICAR Phase 2 (Placebo-controlled, Randomized Study)

MYDICAR administered by antegrade epicardial coronary artery infusion

Sponsors & Collaborators

• Celladon Corporation

  lead INDUSTRY

Principal Investigators

• Brian Jaski, MD · San Diego Cardiac Center

• Donna Mancini, MD · Columbia University Hospital

• Randall Starling, MD · The Cleveland Clinic

• Mariell Jessup, MD · University of Pennsylvania

• Thomas Cappola, MD, ScM · University of Pennsylvania

• Daniel Pauly, MD · Shands Hospital, University of Florida at Gainesville

• Barry London, MD · University of Pittsburgh Medical Center

• Barry Greenberg, MD · University of California at San Diego Medical Center

• A. G. Kfoury, MD · Intermountain Medical Center

• Stephen Archer, MD · University of Chicago

• Andrew Kao, MD · Mid America Heart Institute, Saint Luke's Hospital

• Paul J. Hauptman, MD · St. Louis University Hospital

• Jill Kalman, MD · Icahn School of Medicine at Mount Sinai

• Douglas W. Losordo, MD · Northwestern University

• Eric J. Eichhorn, MD, FACC · Cardiopulmonary Research Science and Technology Institutte, Medical City Dallas Hospital

• Stephanie H. Dunlap, DO · University of Cincinnati

• Vinay Thohan, MD · Wake Forest University

• Maryl R. Johnson, MD · University of Wisconsin, Madison

• Mark Dunlap, MD · MetroHealth Medical Center

• Joaquin E. Cigarroa, MD · Oregon Health and Science University

• Dinesh K. Gupta, MD · Tennessee Center for Clinical Trials, Harton Regional Medical Center

• Marc Klapholz, MD · University of Medicine and Dentistry of New Jersey

• Guillermo Torre, MD · The Methodist Hospital Research Institute

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Max Age

75 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2007-03-31

Primary Completion

2010-08-31

Completion

2012-08-31

Countries

• United States

Study Locations