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Trial record 1 of 2 for:    HHlivc
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Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01195753
Recruitment Status : Terminated
First Posted : September 6, 2010
Last Update Posted : February 8, 2016
Information provided by (Responsible Party):
Cytonet GmbH & Co. KG

Brief Summary:
Treatment with liver cell infusion for children with urea cycle disorders (UCD).

Condition or disease Intervention/treatment Phase
Urea Cycle Disorders Biological: HHLivC Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (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.
Study Start Date : December 2010
Actual Primary Completion Date : December 2015
Actual Study Completion Date : December 2015

Arm Intervention/treatment
Experimental: Liver Cell Infusion Biological: HHLivC
multiple infusion of liver cells

Primary Outcome Measures :
  1. 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 :
  1. Frequency and severity of metabolic crises [ Time Frame: 6 months ]

Information from the National Library of Medicine

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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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01195753

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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

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Responsible Party: Cytonet GmbH & Co. KG Identifier: NCT01195753     History of Changes
Other Study ID Numbers: CCD05
First Posted: September 6, 2010    Key Record Dates
Last Update Posted: February 8, 2016
Last Verified: February 2016
Keywords provided by Cytonet GmbH & Co. KG:
Urea cycle Disorders
Additional relevant MeSH terms:
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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