A clinical study for evaluating long-term safety of patients suffering from Urea Cycle disorders (UCD) or Crigler-Najjar Syndrome (CN) having received infusions of HepaStem.

Phase 1

Conditions: Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The UCD are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are 8 disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia I&II, argininosuccinic aciduria, HHH syndrome & argininemia
MedDRA version: 16.0Level: LLTClassification code 10021601Term: Inborn error of metabolism NOSSystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Registration Number: EUCTR2013-001045-14-PT

Lead Sponsor: Promethera Biosciences

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 21

Inclusion Criteria

1. Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) patients having received infusions of HepaStem during a former interventional clinical study conducted by Promethera Biosciences and who have terminated their participation in that study.
2. For subject under 18 years of age (16 year in UK) and for adult subject legally incapable: subject (if capable of signing) and parents or legal representative have provided a written informed assent/consent before enrolment or
For adult subject capable of signing: subject has provided a written informed assent/consent before enrolment.

Are the trial subjects under 18? yes
Number of subjects for this age range: 21
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Subject has received mature liver cells, stem cells transplantation other than HepaStem, or organ liver transplant.