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)

• Conditions: eonates and Infants up to 3 months including with prenatally or postnatally confirmed urea cycle disorder with below listed deficiency 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 (CPS1D) - Ornithine Transcarbamylase Deficiency (OTCD) - Argininosuccinate Synthetase Deficiency (ASSD/Citrullinaemia)can be included.; MedDRA version: 18.1Level: PTClassification code 10052450Term: Ornithine transcarbamoylase deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 18.1Level: PTClassification code 10058298Term: Argininosuccinate synthetase deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 18.1Level: PTClassification code 10058297Term: Carbamoyl phosphate synthetase deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2006-000136-27-DE
• Lead Sponsor: Cytonet GmbH & Co KG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2007-09-04
• Last Update Posted: 18 April 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

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

Exclusion Criteria

- 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).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified