The FDA has accepted an application to begin a phase 3 clinical trial of bone marrow-derived cell therapy for patients with HF, according to a press release from BioCardia Inc.
The randomized, controlled trial will study CardiAMP Therapy in 250 patients in as many as 40 clinical sites.
The CardiAMP therapy integrates a biomarker panel designed to identify patients with potent bone marrow cells and those who are likely to respond to treatment. The therapy also includes a cell-processing system consisting of a high-dose formulation of autologous bone marrow-derived cells and a transendocardial delivery system, according to the release.
Researchers will evaluate efficacy endpoints including improvement in functional capacity, as indicated by a 6-minute walk test; quality of life, assessed by responses to the Minnesota Living with Heart Failure Questionnaire; and patient survival. Efficacy endpoints will include noninferiority in survival and freedom from major adverse cardiac events.
The trial is the first to simultaneously assess a stem cell therapy simultaneously with a diagnostic, according to the release.
The phase 3 trial follows previously published results from phase 1 and 2 studies of CardiAMP, which indicated significant improvement in functional capacity and quality of life, and favorable safety data.
Carl J. Pepine
“CardiAMP builds on — and benefits from — what has been done in previous [Cardiovascular Cell Therapy Research Network] 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,” co-principal investigator Carl J. Pepine, MD, professor of medicine in the division of cardiovascular medicine at the University of Florida, Gainesville, and Chief Medical Editor of Cardiology Today, stated in the release. “This trial pulls together everything we have learned in the field of autologous bone marrow cell therapy to treat HF. There are very promising signals in the phase 2 data that we hope to see confirmed in the phase 3 trial.”