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Clinical trials for Ornithine Transcarbamylase Deficiency

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   44334   clinical trials with a EudraCT protocol, of which   7366   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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    12 result(s) found for: Ornithine Transcarbamylase Deficiency. Displaying page 1 of 1.
    EudraCT Number: 2018-000156-18 Sponsor Protocol Number: 301OTC02 Start Date*: 2018-08-15
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Lat...
    Medical condition: Ornithine transcarbamylase deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10071107 Ornithine transcarbamylase deficiency LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-003384-25 Sponsor Protocol Number: DTX301-CL301 Start Date*: 2022-12-16
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-Onset...
    Medical condition: Late-onset Ornithine transcarbamylase (OTC) deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10071107 Ornithine transcarbamylase deficiency LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) ES (Trial now transitioned) DE (Trial now transitioned) PT (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2006-000136-27 Sponsor Protocol Number: CCD02 Start Date*: 2008-03-17
    Sponsor Name:Cytonet GmbH & Co KG
    Full Title: Open, Prospective, Uncontrolled, Multicentre Study to Evaluate The Safety and Efficacy of Multiple Applications of Liver Cell Suspension Into The Portal Vein in Children with Urea Cycle Disorders (...
    Medical condition: Neonates and Infants up to 3 months including with prenatally or postnatally confirmed urea cycle disorder with below listed deficiency and children aged > 3 months up to 5 years including with con...
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10052450 Ornithine transcarbamoylase deficiency PT
    18.1 10010331 - Congenital, familial and genetic disorders 10058298 Argininosuccinate synthetase deficiency PT
    18.1 10010331 - Congenital, familial and genetic disorders 10058297 Carbamoyl phosphate synthetase deficiency PT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2016-001057-40 Sponsor Protocol Number: 301OTC01 Start Date*: 2017-03-14
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OT...
    Medical condition: Ornithine transcarbamylase deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10071107 Ornithine transcarbamylase deficiency LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Completed)
    Trial results: View results
    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: 2021-001081-38 Sponsor Protocol Number: ARCT-810-03 Start Date*: 2021-12-23
    Sponsor Name:Arcturus Therapeutics, Inc.
    Full Title: Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult ...
    Medical condition: Ornithine transcarbamylase deficiency
    Disease: Version SOC Term Classification Code Term Level
    26.0 100000004850 10013373 Disorders of urea cycle metabolism LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) ES (Completed) FR (Completed) SE (Completed) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2011-002472-16 Sponsor Protocol Number: CCD09 Start Date*: 2011-12-19
    Sponsor Name:Cytonet GmbH & Co KG
    Full Title: Open, prospective, diagnostic, multicentre study in healthy subjects, patients with urea cycle disorders (UCD), and carriers of UCD mutations, to evaluate in vivo ureagenesis measured after a singl...
    Medical condition: 1. Healthy subjects 2. Symptomatic UCD patients with genetically confirmed CPSD, OTCD, ASSD, or ASLD 3. Asymptomatic carriers of UCD mutations (e.g. parents)
    Disease: Version SOC Term Classification Code Term Level
    14.0 10027433 - Metabolism and nutrition disorders 10013373 Disorders of urea cycle metabolism LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-000988-14 Sponsor Protocol Number: CCD05 Start Date*: 2016-10-12
    Sponsor Name:PROMETHERA Biosciences S.A./N.V.
    Full Title: Title: Open, Prospective, Historic-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Infusion of Liver Cell Suspension (HHLivC) in Children with Urea Cycle Disorders
    Medical condition: Neonatal-onset type of the following urea cycle disorders (UCD): ornithine transcarbamylase deficiency (OTCD), carbamoylphosphate synthetase I deficiency (CPS1D), or argininosuccinate synthetas...
    Disease:
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    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: 2022-000933-18 Sponsor Protocol Number: HLSC-UCD-02 Start Date*: Information not available in EudraCT
    Sponsor Name:Unicyte AG
    Full Title: An Open-label, Non-controlled, Multi-center, Phase 2 Study to Assess the Efficacy and Safety of Allogeneic Human Liver Stem Cells (HLSCs) in the Treatment of Pediatric Patients with Early-onset Ure...
    Medical condition: Urea cycle disorders
    Disease: Version SOC Term Classification Code Term Level
    21.1 200000003094 10013373 Disorders of urea cycle metabolism LLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    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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