Clinical Trial: Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Open, Prospective, Uncontrolled, Multicentre Study to Evaluate The Safety and Efficacy of Multiple Applications of Liver Cell Suspension Into The Portal Vein in Children With Urea Cycle Disorders (UCD
Brief Summary: Urea cycle disorders are rare inherited diseases that generally have a poor outcome. In this study, neonates and infants with UCD will be included within the first 3 months of life and will be treated by repetitive application of human liver cells to reduce the risk of neurological deterioration while awaiting OLT.
Detailed Summary:
Urea cycle disorders are rare inherited diseases that generally have a poor outcome, especially with onset of the disease in the neonatal period. UCDs are caused by a deficiency of one of six enzymes responsible for removing ammonia from the bloodstream. Instead of being converted into urea which is removed from the body with the urine, ammonia accumulates in UCD patients leading to brain damage or death. In the light of a mortality rate of > 50% at the age of 10 years the current pharmacological and dietary therapy is of modest success. Furthermore, mental retardation, cerebral palsy and other neurological sequelae are common among surviving patients.
In the last years, orthotopic liver transplantation (OLT) has become the best therapeutic option for UCD with long-term survival rates of about 90%. However, in the first weeks of life OLT still is technically demanding and prone to complications. With larger size of the recipient, the technical problems with OLT decrease considerably. The increased body weight usually achieved at the age of more than 8 weeks is related to a major reduction in transplantation related morbidity. Stabilization of metabolism until the patient can undergo OLT is essential.
In this study, neonates and infants with UCD will be included within the first 3 months of life and will be treated by repetitive application of human liver cells. In the last consequence, the aim of this new therapy option is to supply a sufficient amount of healthy liver cells to compensate for the metabolic defect and to reduce the risk of neurological deterioration while awaiting OLT.
Sponsor: Cytonet GmbH & Co. KG
Current Primary Outcome: Safety of the application of liver cells, safety of the placement of an application catheter to the portal vein. [ Time Frame: 7 - 15 weeks ]
Original Primary Outcome: Safety of the application of liver cells, increase in the respective enzyme activity in liver biopsies taken from the explanted organ compared to the enzyme activity in the liver biopsy taken prior to the first liver cell application. [ Time Frame: 7 - 15 weeks ]
Current Secondary Outcome: Changes in 13C urea formation. Changes in the respective enzyme activity in liver biopsies from the explanted organ compared to the enzyme activity in the liver before cell application. [ Time Frame: 7-15 weeks ]
Original Secondary Outcome: Safety, metabolic crises, laboratory parameters [ Time Frame: 7-15 weeks ]
Information By: Cytonet GmbH & Co. KG
Dates:
Date Received: July 16, 2008
Date Started: July 2008
Date Completion:
Last Updated: February 5, 2016
Last Verified: February 2016
