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Safety and Efficacy of Liver Cell Application in Children with Urea Cycle Disorders
NCT00718627Phase 2
Key facts
- Status
- Completed
- Start
- Jul 2008
- Primary completion
- Nov 2015
- Countries
- 1
- Sponsor
- Cytonet
- Modality
- Cell Therapies
- Drugs
- Heparesc
- Conditions
- urea cycle disorders (UCD)carbamoyl phosphate synthetase I deficiency (CPS1D)ornithine transcarbamylase deficiencycitrullinemia
01
Overview
Phase 2, open-label, single-arm trial of Heparesc in urea cycle disorders (UCD), carbamoyl phosphate synthetase I deficiency (CPS1D), ornithine transcarbamylase deficiency and 1 more, sponsored by Cytonet. Primary endpoint: Safety of the application of liver cells, safety of the placement of an application catheter to the portal vein. Started Jul 2008; status: completed.
02
Design
1 arm- Study design
- Non-randomizedOpen-label
HHLivC Therapy Group
Infusion of Human Heterologous Liver Cells (Heparesc)
03
Who it enrols
Neonates and infants (0-5 years) with confirmed urea cycle disorders (CPSD, OTCD, Citrullinemia), weight > 3.5 kg, and plasma ammonia <= 250 umol/l. Excludes structural liver disease, portal vein thrombosis, HIV, and severe coagulopathy.
04
Endpoints
Primary
Safety of the application of liver cells, safety of the placement of an application catheter to the portal vein
Secondary
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
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