Open, Prospective, Uncontrolled, Multicentre Study to Evaluate The Safety and Efficacy of Multiple Applications of Liver Cell Suspension Into The Portal Vein in Children with Urea Cycle Disorders (UCDs)

Cytonet GmbH & Co KG0 个研究点开始时间: 2007年9月4日最近更新: 2016年4月18日

研究概览

暂无简介。

•Neonates and Infants up to 3 months including with prenatally or postnatally confirmed urea cycle disorder with deficiency of either:
•- Carbamyl-phosphate Synthetase I Deficiency (CPS1D)
•- Ornithine Transcarbamylase Deficiency (OTCD)
•- Argininosuccinate Synthetase Deficiency (ASSD/Citrullinaemia)
•and children aged > 3 months up to 5 years including with confirmed urea cycle disorder and unstable metabolism with deficiency of either
•- Carbamyl-phosphate Synthetase I Deficiency (CPSD)
•- Ornithine Transcarbamylase Deficiency (OTCD)
•- Argininosuccinate Synthetase Deficiency (Citrullinaemia)
•can be included.
•- Accessibility of the portal vein.
•- Plasma ammonia level - Written informed consent from parents or legal guardian(s).
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•- Structural liver disease (cirrhosis, portal hypertension), or venoocclusive diseases,
•- Portal vein thrombosis
•- Body Weight less than/equal 3.5 kg
•- Carrier of the human immuno-deficieny 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 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 absence of thrombophilia testing in subject
•- Cancer, severe systemic or chronic disease other than study indication (urea cycle deficiency).

发起方

Cytonet GmbH & Co KG