A long-term safety follow-up study 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. 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: 19.0 Level: LLT Classification code 10021601 Term: Inborn error of metabolism NOS System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2013-001045-14-GB
• Lead Sponsor: Promethera Biosciences

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-04-28
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 19

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: parents or legal representative have provided a written informed consent before enrolment. The subject (if capable of signing) is required to sign an assent.
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: 20
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 0

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