A Phase 2 interventional study of Human Heterologous Liver Cells in Urea Cycle Disorders, Carbamoylphosphate Synthetase I Deficiency and Ornithine Transcarbamylase Deficiency, sponsored by Cytonet GmbH & Co. KG. Completed at 2 sites in Germany. Open to participants aged 1 Day to 5 Years. Per ClinicalTrials.gov, last updated 2016-02-08.
Sponsored by Cytonet GmbH & Co. KG · Phase 2, Interventional, and Treatment
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.
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.
58 studies on the registry are indexed under Urea Cycle Disorders, Inborn; 12 are open to participants now.
This study's enrollment of 12 is below the median of 17 across 35 interventional studies indexed under Urea Cycle Disorders, Inborn.
Browse Urea Cycle Disorders, Inborn studies →Cytonet GmbH & Co. KG is the lead sponsor of 4 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Children aged > 3 months up to ≤ 5 years of age with unstable metabolism and confirmed urea cycle disorder of either:
A DNA analysis will further confirm diagnosis prior to or after inclusion according to the protocol.
Exclusion Criteria
Biological: Human Heterologous Liver Cells
Multiple applications of liver cell suspension for infusion
Safety of the application of liver cells, safety of the placement of an application catheter to the portal vein.
Time frame: 7 - 15 weeks
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
This study is completed, as verified in Feb 2016. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Cytonet GmbH & Co. KG