Celulas hepatocitos troncales adultas alogenicas de higado expandidas    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
251Urea cycle disorder1

251. Urea cycle disorder    [ 42 clinical trials,   45 drugs,   (DrugBank: 10 drugs),   4 drug target genes,   29 drug target pathways]
Searched query = "Urea cycle disorder", "N-acetylglutamate synthase deficiency", "NAGS deficiency", "Carbamoyl phosphate synthetase I deficiency", "CPS1 deficiency", "Ornithine transcarbamylase deficiency", "OTC deficiency", "Classic citrullinemia", "Citrullinemia type I", "Argininosuccinic aciduria", "Argininemia", "Hiperornitinemia-hiperamonemia-homocitrulinuria syndrome", "HHH syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 42 trial found
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1EUCTR2014-000650-11-ES
(EUCTR)
13/04/201520/10/2014HEP002 is a clinical study designed for paediatric patients with urea cycle disorders (UCD). UCD patients will receive several infusions of HepaStem. The efficacy as well as the safety of the medicinal product will be assessed during the year following infusions.A prospective, open label, multi-country, multicenter, efficacy and safety study of several infusions of Promethera HepaStem in Urea Cycle Disorders pediatric patients The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are eight disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia type I, argininosuccinic aciduria, argininemia, HHH syndrme and citrullinemia type II.Mutations in each of the enzymes have been identified, resulting in failed production of urea, the end product of the urea cycle.
MedDRA version: 18.0;Level: LLT;Classification code 10013373;Term: Disorders of urea cycle metabolism;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: HepaStem
INN or Proposed INN: Celulas hepatocitos troncales adultas alogenicas de higado expandidas
Promethera BiosciencesNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Belgium;Spain