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Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders

This study has been terminated.
Information provided by (Responsible Party):
Cytonet GmbH & Co. KG Identifier:
First received: March 2, 2010
Last updated: February 5, 2016
Last verified: February 2016
Treatment with liver cell infusion for children with urea cycle disorders (UCD).

Condition Intervention Phase
Urea Cycle Disorders
Biological: HHLivC
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open, Prospective, Historic-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Infusion of Liver Cell Suspension (HHLivC) in Children With Urea Cycle Disorders.

Resource links provided by NLM:

Further study details as provided by Cytonet GmbH & Co. KG:

Primary Outcome Measures:
  • Changes in 13C urea formation from baseline to 2 and 4 months after first HHLivC infusion [ Time Frame: Baseline to 2 and 4 months ]

Secondary Outcome Measures:
  • Frequency and severity of metabolic crises [ Time Frame: 6 months ]

Enrollment: 10
Study Start Date: December 2010
Study Completion Date: December 2015
Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Liver Cell Infusion Biological: HHLivC
multiple infusion of liver cells

Detailed Description:

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, young children with UCD 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.


Ages Eligible for Study:   up to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age: birth up to 5 years of age
  • Ornithine transcarbamylase deficiency [OTCD], Carbamyl phosphate synthetase I deficiency [CPSD], Argininosuccinate synthetase deficiency [ASSD, Citrullinaemia]
  • Written Informed Consent

Exclusion Criteria:

  • Weight ≤ 3.5 kg
  • Presence of acute infection at the time of inclusion
  • Severe chronic or systemic disease other than study indication
  • Structural liver disease (eg, cirrhosis, portal hypertension)
  • Required valproate therapy
  Contacts and Locations
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Please refer to this study by its identifier: NCT01195753

United States, California
Stanford University
Palo Alto, California, United States, 94304
University of California
San Diego, California, United States, 92103
United States, Connecticut
Yale University
New Haven, Connecticut, United States, 06250
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Sponsors and Collaborators
Cytonet GmbH & Co. KG
  More Information

Responsible Party: Cytonet GmbH & Co. KG Identifier: NCT01195753     History of Changes
Other Study ID Numbers: CCD05
Study First Received: March 2, 2010
Last Updated: February 5, 2016

Keywords provided by Cytonet GmbH & Co. KG:
Urea cycle Disorders

Additional relevant MeSH terms:
Urea Cycle Disorders, Inborn
Pathologic Processes
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases processed this record on April 24, 2017