Status:
COMPLETED
Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
Lead Sponsor:
Cytonet GmbH & Co. KG
Conditions:
Urea Cycle Disorders
Carbamoylphosphate Synthetase I Deficiency
Eligibility:
All Genders
1-5 years
Phase:
PHASE2
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...
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
- Inclusion Criteria
- Neonates and infants up to the age of ≤ 3 months with prenatally or postnatally confirmed urea cycle disorder and
- Children aged \> 3 months up to ≤ 5 years of age with unstable metabolism and confirmed urea cycle disorder of either:
- Carbamylphosphate synthetase I \[CPSD\] or
- Ornithine transcarbamylase \[OTCD\] or
- Argininosuccinate synthetase \[Citrullinaemia\]
- A DNA analysis will further confirm diagnosis prior to or after inclusion according to the protocol.
- Accessibility of the portal vein
- Plasma ammonia level ≤ 250 μmol/l
- Written informed consent
- Exclusion Criteria
- Structural liver disease (cirrhosis, portal hypertension), or venoocclusive diseases
- Portal vein thrombosis
- Body Weight ≤3.5 kg
- Carrier of the human immuno-deficiency virus (HIV)
- Any other contraindication for immunosuppression
- Presence of acute infection at the time of inclusion
- Participation in other clinical trials or received experimental medication within the last 30 days
- Live vaccination planned during the course of the study
- Live vaccination within 4 weeks prior to beginning of study
- Allergic disposition against contrast medium used in study and/or antibiotics used in the manufacturing process
- Required valproate therapy
- Severe coagulopathy or thrombocytopenia
- Known diagnosis of hereditary thrombophilia (e.g. Factor V Leiden, Prothrombin 20210A variant) or parental history of hereditary thrombophilia and absense of thrombophilia testing in subject
- Cancer, severe systemic or chronic disease other than study indication (urea cycle deficiency)
Exclusion
Key Trial Info
Start Date :
July 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2015
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT00718627
Start Date
July 1 2008
End Date
November 1 2015
Last Update
February 8 2016
Active Locations (2)
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1
University Children's Hospital, Heinrich-Heine University
Düsseldorf, Germany, 40225
2
University Children's Hospital
Heidelberg, Germany, D-69120