Australia-based regenerative medicine company Mesoblast has signed an agreement with the US National Institutes of Health’s (NIH) National Heart, Lung and Blood Institute to carry out a clinical trial using the company’s adult stem cell therapy for the treatment of patients with advanced heart failure.
The treatment is indicated for patients who require an implantable left ventricular assist device (LVAD) to maintain circulatory support.
The trial is also supported by the National Institute of Neurological Disorders and Stroke, part of the NIH, and the Canadian Institutes for Health Research.
The company’s mesenchymal precursor cells (MPCs) are used in end-stage heart failure patients to improve heart muscle function in order to reduce the need for LVAD support, and to reduce the long-term complications of LVAD implantation.
“We are pleased that the NIH has chosen to evaluate Mesoblast’s cell-based therapy in patients with the most advanced stage of heart failure, a complementary area to our ongoing Phase III programme in patients with earlier stage disease.”
Mesoblast chief executive Silviu Itescu said: “We are pleased that the NIH has chosen to evaluate Mesoblast’s cell-based therapy in patients with the most advanced stage of heart failure, a complementary area to our ongoing Phase III programme in patients with earlier stage disease.”
The NIH-funded Cardiothoracic Surgical Trials Network will be responsible for carrying out the trial, which will enrol 120 patients in more than 20 sites across the US.
The double-blind, placebo-controlled, two-to-one randomised trial is designed to evaluate the effects of a single injection of 150 million allogeneic, or off-the-shelf, MPCs into the hearts of patients with advanced heart failure.
The primary efficacy endpoint of the trial is the number of temporary weans from LVAD tolerated over 12 months. In addition, the trial will evaluate patient survival and re-hospitalisation over 12 months.
The new study builds on data from a double-blind study in 30 patients which showed the potential benefits of a single intra-cardiac injection of 25 million MPCs in advanced heart failure and LVAD implantation.
The company said that the 150 million MPC dose selected for direct cardiac injection in this second trial is the same dose that is currently being evaluated in an ongoing Phase III trial of about 1,700 patients with NYHA class II-III heart failure.
Sponsored by Mesoblast’s development and commercial partner, Teva Pharmaceutical Industries, the Phase III trial is actively enrolling patients across multiple sites in the US.