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NOVATO, Calif., Jan. 13, 2014 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. (Nasdaq:RPTP) today announced that its Phase 2b clinical trial, to evaluate the safety and potential efficacy of RP103 delayed-release cysteamine bitartrate as a prospective treatment of non-alcoholic fatty liver disease (NAFLD) in children, has met its enrollment target.
Raptor is conducting this trial in cooperation with the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the National Institutes of Health, under a Cooperative Research and Development Agreement (CRADA). The trial, called Cysteamine Bitartrate Delayed-Release for the Treatment of Non-alcoholic Fatty Liver Disease in Children (CyNCh), has enrolled 160 pediatric participants at ten U.S. centers in the NIDDK-sponsored NASH Clinical Research Network. NIDDK and Raptor are sharing development costs related to the CyNCh study.
The primary objective of this randomized, multicenter, double-blind, placebo-controlled Phase 2b clinical trial in children 8 to 17 years old is to evaluate whether 52 weeks of treatment with RP103 in pediatric patients with biopsy-confirmed moderate to severe NAFLD reverses damage caused by the condition as measured by changes in NAFLD Activity Score (NAS), a rating scale based on histological measurement of disease severity. Secondary endpoints will include, among others, blood markers for liver health including alanine transaminase (ALT) and aspartate transaminase (AST), and safety and tolerability will be assessed. Full results from the Phase 2b CyNCh trial are expected in the first half of 2015.
The CyNCh clinical trial follows the results of an open-label Phase 2a clinical trial which was conducted under a collaboration agreement between Raptor and the University of California, San Diego (UC San Diego) at UC San Diego's General Clinical Research Center. The Phase 2a clinical trial involved children with biopsy-confirmed diagnosis of moderate to severe NAFLD and baseline ALT and AST measurements at least twice that of normal levels. These patients received a prototype of RP103 twice daily for six months, followed by a six-month post-treatment monitoring period. Among all patients there was a mean 54% reduction in ALT (p=0.004), meeting the pre-defined primary endpoint of at least 50% ALT reduction from baseline. In addition, patients saw improvements in secondary endpoints including AST (41% avg reduction, p=0.02), cytokeratin 18 (45% avg reduction, p=0.026), and adiponectin (35% avg reduction, p=0.023). Serum transaminases were measured following drug withdrawal and the reductions in ALT and AST persisted during the 6 month post-treatment phase.