A clinical study for evaluating long-term safety of patients 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. Thereare 8 disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia I&II, argininosuccinic aciduria, HHH syndrome & argininemia; MedDRA version: 16.1Level: 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-IT
• Lead Sponsor: Promethera Biosciences

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-12-31
• Last Update Posted: 12 February 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 21

Inclusion Criteria

1. All patients having received infusions of HepaStem during a former interventional clinical study conducted by PB and who have terminated their participation in that study.
2. For subjects under 18 years of age (16 year in UK) and for adult subjects legally incapable: the subject (if capable of signing) and parents or legal representative have provided a written informed assent/consent before enrolment or
For an adult subject legally capable: the subject has provided a written informed 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.

Study & Design

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