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Clinical trials for Argininosuccinic aciduria

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   44364   clinical trials with a EudraCT protocol, of which   7388   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    4 result(s) found for: Argininosuccinic aciduria. Displaying page 1 of 1.
    EudraCT Number: 2012-002120-33 Sponsor Protocol Number: HLSCS01-11 Start Date*: 2013-12-06
    Sponsor Name:AZIENDA SANITARIA OSPEDALIERA O.I.R.M. - S. ANNA
    Full Title: Human Liver Stem Cells (HLSCs) in patients suffering from liver-based inborn metabolic diseases causing life-threatening neonatal onset of hyperammonemic encephalopathy
    Medical condition: neonatal hyperammonemic encephalopathy associated with inborn liver metabolic diseases
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10059521 Methylmalonic aciduria PT
    14.1 10010331 - Congenital, familial and genetic disorders 10058299 Argininosuccinate lyase deficiency PT
    14.1 10010331 - Congenital, familial and genetic disorders 10052450 Ornithine transcarbamoylase deficiency PT
    14.1 10010331 - Congenital, familial and genetic disorders 10058297 Carbamoyl phosphate synthetase deficiency PT
    Population Age: Newborns, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2014-000650-11 Sponsor Protocol Number: HEP002 Start Date*: 2014-09-15
    Sponsor Name:Promethera Biosciences
    Full Title: A prospective, open label, multicountry, efficacy and safety study of several infusions of HepaStem in Urea Cycle Disorders pediatric patients.
    Medical condition: The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are seven disorders to be investigated: carbamoylphosphate synthetase I deficiency [CP...
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004861 10013373 Disorders of urea cycle metabolism LLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: BE (Completed) ES (Completed) FR (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2013-001045-14 Sponsor Protocol Number: SAF001 Start Date*: 2013-04-22
    Sponsor Name:Promethera Biosciences
    Full Title: SAF 001: A long-term safety follow-up study of patients suffering from Urea Cycle disorders (UCD) or Crigler-Najjar Syndrome (CN) having received infusions of HepaStem.
    Medical condition: 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 ur...
    Disease: Version SOC Term Classification Code Term Level
    15.1 100000004850 10021601 Inborn error of metabolism NOS LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed) PT (Completed) IT (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2011-004074-28 Sponsor Protocol Number: UKHEP001 Start Date*: 2012-07-20
    Sponsor Name:Promethera Biosciences
    Full Title: A prospective, open label, multicenter, randomized, safety and preliminary efficacy study of one cycle of Promethera HepaStem® in Urea Cycle Disorders and Crigler-Najjar Syndrome patients
    Medical condition: 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 ur...
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10021601 Inborn error of metabolism NOS LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) BE (Completed) IT (Completed)
    Trial results: View results
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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