Human Liver Stem Cells (HLSCs) in liver metabolic diseases

Phase 1

• Conditions: neonatal hyperammonemic encephalopathy associated with inborn liver metabolic diseases; MedDRA version: 14.1Level: PTClassification code 10059521Term: Methylmalonic aciduriaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 14.1Level: PTClassification code 10058299Term: Argininosuccinate lyase deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 14.1Level: PTClassification code 10052450Term: Ornithine transcarbamoylase deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 14.1Level: PTClassification code 10058297Term: Carbamoyl phosphate synthetase deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2012-002120-33-IT
• Lead Sponsor: AZIENDA SANITARIA OSPEDALIERA O.I.R.M. - S. ANNA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-06-12
• Last Update Posted: 19 November 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

neonatal-onset hyperammonemic encephalopathy; formal biochemical diagnosis of one of the five inborn metabolic diseases, as listed in the Table 1; formal evaluation for early liver transplantation; being referred to the Presidio Ospedaliero Infantile Regina Margherita, Centro delle Malattie Metaboliche, Torino, Italy;

Are the trial subjects under 18? yes
Number of subjects for this age range: 3
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

Refusal of signing the specific informed consent for entering the study; Evidence of uncontrolled coagulopathy;

Study & Design

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