FDA Accepts Protocol for Phase III Clinical Trial of Biocardia’s CardiAMP Therapy
November 11, 2014—BioCardia, Inc. announced that the US Food and Drug Administration (FDA) has accepted the company’s application to begin a phase III clinical trial of its bone marrow-derived CardiAMP therapy for heart failure. The clinical trial is a randomized, controlled, multicenter study of 250 patients evaluating CardiAMP therapy at up to 40 clinical sites.
According to the company, CardiAMP therapy for heart failure integrates a biomarker panel to identify candidates likely to respond to therapy. The therapy is a cell processing system consisting of a high-dosage formulation of autologous bone marrow-derived cells and a transendocardial delivery system that ensures efficient and consistent targeted delivery. The FDA’s Center for Biologics Evaluation and Research division will review this therapy under the agency’s premarketing approval regulations.
The CardiAMP trial efficacy endpoints include improvements in functional capacity as measured by the six-minute walk test, quality of life as measured by the Minnesota Living with Heart Failure Questionnaire, and survival. Safety endpoints include noninferiority with respect to survival and freedom from major adverse cardiac events.
The trial’s coprincipal investigators are Carl Pepine, MD, and Amish Raval, MD, who were involved in the trial design. Dr. Pepine is Professor of Medicine, Division of Cardiovascular Medicine, at the University of Florida and principal investigator for the University of Florida Center for the Cardiovascular Cell Therapy Research Network (CCTRN). He is also past president of the American College of Cardiology. Dr. Raval is Associate Professor of Medicine, Division of Cardiovascular Medicine, at the University of Wisconsin, where he practices as an interventional cardiologist conducting cardiovascular clinical trials for cell and biologic therapy. He is also Director of Cardiovascular Clinical Research and Director of the Regional ST Elevation Myocardial Infarction Program.
In the company’s press release, Dr. Raval commented, “CardiAMP has the potential to bring an effective therapy forward that will provide meaningful clinical benefit to patients with ischemic heart failure. There is an enormous unmet need here, and CardiAMP is a worthy endeavor that has a high probability of meeting both the safety and the efficacy required to become a therapeutic option for heart failure patients.”
Dr. Pepine added, “CardiAMP builds on—and benefits from—what has been done in previous CCTRN trials in that it provides the highest effective dosage that has been studied in a rigorous trial to date, and the companion diagnostic selects patients that have potent autologous bone marrow. This trial pulls together everything we have learned in the field of autologous bone marrow cell therapy to treat heart failure. There are very promising signals in the phase II data that we hope to see confirmed in the phase III trial.”
The company stated that there are two studies supporting the CardiAMP therapy with statistically and clinically significant results demonstrating an excellent safety profile, as well as functional and quality-of-life improvements. One study is the phase I TAB MMI (Transendocardial AutologousBone Marrow in Myocardial Infarction Study), which was published by Luis de la Fuente, MD, et al in Eurointervention (2011;7:805–812). The other study is the phase I/II TAC-HFT (Transendocardial Autologous Cells in Heart Failure Trial), which was published by Alan W. Heldman, MD in JAMA: Journal of the American Medical Association (2014;311:62–73).