Status:
TERMINATED
Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
Lead Sponsor:
Cytonet GmbH & Co. KG
Conditions:
Urea Cycle Disorders
Eligibility:
All Genders
1-5 years
Phase:
PHASE2
Brief Summary
Treatment with liver cell infusion for children with urea cycle disorders (UCD).
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 ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
December 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2015
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT01195753
Start Date
December 1 2010
End Date
December 1 2015
Last Update
February 8 2016
Active Locations (5)
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1
Stanford University
Palo Alto, California, United States, 94304
2
University of California
San Diego, California, United States, 92103
3
Yale University
New Haven, Connecticut, United States, 06250
4
Children's Memorial Hospital
Chicago, Illinois, United States, 60614