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Clinical Trial for Rare Urea Disorders at Children’s Hospital of Wisconsin and Medical College of Wisconsin

By Maureen Mack

Dec. 5, 2012

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The Children’s Hospital of Wisconsin Research Institute and the Medical College of Wisconsin are recruiting patients for a clinical trial investigating a novel treatment for urea cycle disorders (UCD) in children.

David Dimmock, M.D., clinical geneticist at Children’s Hospital of Wisconsin and assistant professor of pediatrics (genetics) at the Medical College, is the lead investigator for the study in Wisconsin and Michigan’s Upper Peninsula.

Urea cycle disorders are life-threatening disorders of the liver, often mistaken for infections in newborns and young children. Build-up of ammonia may lead to massive damage of the nerves and the brain; in some cases, it can be fatal. The definitive treatment is liver transplantation, which is a difficult procedure for very young patients. Additionally, there is a shortage of suitable organs available for transplantation.

Liver cell therapy is being evaluated to act as a therapeutic bridge, with the aim of normalizing ammonia and glutamine levels in the patient in order to avoid neurological damage. This therapy may also be an option for families not seeking a liver transplant.

Cytonet is recruiting patients at 11 United States institutions for the SELICA III trial. More information can be found at http://clinicaltrials.gov/ct2/show/NCT01195753.

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