BALTIMORE, Dec. 08, 2017 (GLOBE NEWSWIRE) -- Asklepion Pharmaceuticals, LLC (“Asklepion” or “the Company”), a biotechnology company focused on the development of IV-citrulline for rare pediatric diseases, announced detailed results from a Phase 1b/2a double blind, randomized, placebo controlled, clinical trial to determine the pharmacokinetics and safety of a revised protocol of intravenous citrulline versus placebo in children undergoing cardiopulmonary bypass (CPB). The data was presented at the Pediatric Cardiac Intensive Care Society (PCICS) 13th Annual International Meeting on December 7th in Washington, D.C.
The results from this study show that the revised dosing protocol met the primary endpoint of achieving a sustained target plasma citrulline level of >100 μmol/L in patients receiving citrulline. Due to the small sample size, the study was not powered to evaluate statistical significance for the secondary endpoints; nonetheless, two of the secondary endpoints showed important improvements in shortening the duration of mechanical ventilation and inotrope therapy between the treated and placebo control groups. No unexpected AEs or SAEs occurred under administration of IV-citrulline.
“Reducing the need for mechanical ventilation and for inotropes to support cardiac output are both important outcomes. Cessation of mechanical ventilation and inotrope support are key determinants of a child’s readiness for discharge from the intensive care unit, which are important criteria in assessing hospital resource burden and overall costs,” stated Dr. Gary R. Pasternack, Chief Executive Officer of Asklepion. “We are encouraged by the findings of this study and look forward to completing our Phase 3 clinical trial program.”
This Phase 1b/2a double blind, randomized, placebo controlled clinical trial evaluated the prevention of clinical sequelae of acute lung injury induced by cardiopulmonary bypass in 22 pediatric patients undergoing surgical repair of an atrial septal defect, a ventricular septal defect, or a partial or complete atrioventricular septal defect. Eligible patients were randomized (1:1) to receive citrulline or placebo. A bolus of either citrulline or placebo was administered before, during, and immediately following surgery. The citrulline or placebo infusion was discontinued once invasive arterial blood pressure monitoring was discontinued or at 48 hours, whichever came first.
Asklepion Pharmaceuticals is currently conducting a Phase 3 trial to determine whether IV-citrulline is effective and safe in the prevention of clinical sequelae of acute lung injury in pediatric subjects undergoing surgery for congenital heart defects. More information can be found at www.asklepionpharm.com/clinical-trials
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. Current interventions focus on treating these serious complications once the patient displays symptoms post-surgery. Citrulline is not a treatment for lung or other organ damage if it occurs, but it is being studied to determine if it helps prevent or lessens the severity of problems resulting from acute lung injury.
About 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.