BALTIMORE, Dec. 19, 2017 (GLOBE NEWSWIRE) -- Asklepion Pharmaceuticals, LLC (“Asklepion” or “the Company”) announced that it has reached an agreement with the U.S. Food and Drug Administration (FDA) regarding a Special Protocol Assessment (SPA) on the design of a pivotal Phase 3 trial for intravenous citrulline. This SPA provides agreement that the Phase 3 protocol design, notably clinical endpoints, trial population and statistical analyses adequately address objectives that, if met, would form the primary basis of an efficacy claim in support of a regulatory submission for approval of intravenous citrulline for the prevention of clinical sequelae of acute lung injury induced by cardiopulmonary bypass in pediatric subjects undergoing surgery for congenital heart defects.
Asklepion’s trial entitled, “A Phase III Double-Blind, Randomized, Placebo Controlled, Multi-Center Clinical Study to Evaluate the Efficacy and Safety of Intravenous L-citrulline for the Prevention of Clinical Sequelae of Acute Lung Injury induced by Cardiopulmonary Bypass in Pediatric Subjects Undergoing Surgery for Congenital Heart Defects” will enroll 190 subjects in the USA, Europe and Israel. The primary endpoint for the study is a composite endpoint, comprising the longer in duration of mechanical ventilation and inotrope therapy for each patient—a surrogate measure of postoperative time in the ICU. The trial is being conducted in pediatric patients, ages 0 – 18 years, with any of three representative specific types of congenital heart defects to assess effectiveness and safety.
Coordinating investigator Frederick E. Barr, M.D., chair of the Department of Pediatrics and associate dean for child health in the University of Arkansas for Medical Sciences College of Medicine, as well as pediatrician-in-chief for Arkansas Children’s, commented, “We are very encouraged by the results of the Phase 1b/2a study and believe in the importance of further evaluating whether intravenous citrulline could provide a major advancement in preventing postoperative complications after cardiopulmonary bypass and congenital cardiac surgery and overall improved patient outcomes.”
Gary R. Pasternack, MD, PhD, Chief Executive Officer of Asklepion Pharmaceuticals, stated “This SPA confirms the ongoing collaborative discussion Asklepion has had with the Agency and we would like to thank the FDA for its invaluable guidance throughout this process. Finalizing an agreement on the design of our Phase 3 trial has been a priority for us and we now look forward to the outcome of the trial.”
Special Protocol Assessments
A Special Protocol Assessment (SPA) from the FDA is a special procedure by which the FDA provides official evaluation and written agreement that the design and planned analysis of a study adequately address the objectives necessary to support a regulatory submission. However, FDA may rescind a SPA if the director of the FDA reviewing division determines that a substantial scientific issue essential to determining the safety or efficacy of the drug was identified after the trial began. For more information on the SPA process, please visit: http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm080571.pdf
Acute Lung Injury Induced by Cardiopulmonary Bypass
Children undergoing surgery for congenital heart problems are at risk for acute lung injury caused by cardiopulmonary bypass. Both the lung tissue and the blood vessels in the lungs can be damaged by CPB due to a primarily humoral inflammatory reaction, which can lead to significant heart and lung problems after surgery. In contrast to current methods of addressing the postoperative pulmonary dysfunction associated with cardiopulmonary bypass, all of which are remedial, citrulline is being evaluated as a preventive measure to avoid or reduce the risk of such sequelae of CPB.
Asklepion Pharmaceuticals
Asklepion Pharmaceuticals is a Baltimore-based, biopharmaceutical company, focused on the discovery, development, and commercialization of pharmaceutical products to help patients who have little to no therapeutic options due to the rarity of their conditions. Learn more at: www.asklepionpharm.com