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Edison Pharmaceuticals & Bambino Gesu Children's Hospital Announce Initiation of EPI-743 Phase 2 Cobalamin C Deficiency Syndrome Clinical Trial
Date:2/18/2013

MOUNTAIN VIEW, Calif., Feb. 19, 2013 /PRNewswire/ -- Edison Pharmaceuticals today announced the initiation of a phase 2 clinical trial entitled, "Double-Blind, Placebo-Controlled Clinical Trial of EPI-743 in Patients with Cobalamin C Defect."

The trial is a placebo-controlled study lasting 12 months, preceded by a six-month run-in phase for all patients to establish a defined clinical and metabolic baseline. The primary endpoint is improvement in visual function with secondary outcome measurements assessing neurologic and neuromuscular function, glutathione biomarkers, quality of life, as well as safety parameters. More information on study specifics is available at www.ClinicalTrials.gov.

The rationale underlying this investigation is centered on understanding whether EPI-743 – a therapeutic targeting primary defects in electron transport– might hold therapeutic benefit in disorders of intermediary metabolism that also result in redox disturbances.

"Cobalamin C deficiency represents an inherited disorder that induces perturbations in the metabolism of glutathione. Given the central role of glutathione in cellular redox balance and antioxidant defense systems, we are eager to explore whether a therapeutic that increases glutathione such as EPI-743 will provide clinical benefit," stated Professor Carlo Dionisi-Vici, Division of Metabolism, Bambino Gesu Children's Hospital– the trial's principal investigator. 

Cobalamin C Deficiency Syndrome

Cobalamin C (Cbl-C) defect belongs to the family of diseases known as methylmalonic acidurias and is the most common inborn error of vitamin B12 metabolism. Cbl-C defect is a syndrome that results in multiorgan system disease most significantly impacting the central nervous system. While the clinical presentation can be heterogeneous, the onset of symptoms typically arises within the
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SOURCE Edison Pharmaceuticals, Inc.
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