251. Urea cycle disorder Disease details / Clinical trials / Drug dev / DR info


Clinical trials : 54 Drugs : 61 - (DrugBank : 15) / Drug target genes : 3 - Drug target pathways : 28

Drugs and their primary sponsors and trial info
15N ammonium chloride (15NH4Cl)   
   Unicyte AG
      2021   Phase 1   EUCTR2021-000824-36-ES   Austria;Belgium;Czechia;France;Germany;Israel;Italy;Netherlands;Poland;Portugal;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;
      -   Phase 1   EUCTR2021-000824-36-BE   Austria;Belgium;France;Germany;Israel;Italy;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;
      -   Phase 1   EUCTR2021-000824-36-AT   Austria;Belgium;France;Germany;Israel;Italy;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;
ALLOGENIC HUMAN HETEROLOGOUS LIVER CELLS   
   PROMETHERA Biosciences S.A./N.V.
      -   Phase 2   EUCTR2015-000988-14-Outside-EU/EEA   Canada;United States;
ARCT-810   
   Arcturus Therapeutics, Inc.
      2022   Phase 2   EUCTR2021-001081-38-ES   Belgium;Spain;United Kingdom;
      2020   Phase 1   NCT04442347   United States;
      2020   Phase 1   NCT04416126   New Zealand;
      -   Phase 2   EUCTR2021-001081-38-BE   Belgium;United Kingdom;
Acetohydroxamic Acid Oral Tablet [Lithostat]   
   Nicholas Ah Mew
      2017   Phase 1/Phase 2   NCT03181828   United States;
Alanine   
   University of Aarhus
      2021   -   NCT05076318   Denmark;
Ammonium (15N) chloride   
   Unicyte AG
      2021   Phase 1   EUCTR2021-000824-36-ES   Austria;Belgium;Czechia;France;Germany;Israel;Italy;Netherlands;Poland;Portugal;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;
      -   Phase 1   EUCTR2021-000824-36-BE   Austria;Belgium;France;Germany;Israel;Italy;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;
      -   Phase 1   EUCTR2021-000824-36-AT   Austria;Belgium;France;Germany;Israel;Italy;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;
Arginine   
   Brendan Lee
      2008   Phase 2   NCT00345605   United States;
Carglumic acid   
   Ito Tetsuya
      2019   Phase 4   JPRN-jRCTs041190055   Japan;
Celulas hepatocitos troncales adultas alogenicas de higado expandidas   
   Promethera Biosciences
      2015   -   EUCTR2014-000650-11-ES   Belgium;Spain;
DTX301   
   Dimension Therapeutics, Inc.
      2017   Phase 1;Phase 2   EUCTR2016-001057-40-ES   Canada;France;Spain;United Kingdom;United States;
   Ultragenyx Pharmaceutical, Inc.
      2020   Phase 1;Phase 2   EUCTR2018-000156-18-FR   Canada;France;Spain;United Kingdom;United States;
      2018   Phase 1;Phase 2   EUCTR2018-000156-18-GB   Canada;Spain;United Kingdom;United States;
      2018   Phase 1;Phase 2   EUCTR2018-000156-18-ES   Canada;Spain;United Kingdom;United States;
      2017   Phase 1;Phase 2   EUCTR2016-001057-40-GB   Canada;Spain;United Kingdom;United States;
Glyceryl tri-(4-phenylbutyrate)   
   Hyperion Therapeutics Limited
      2008   -   EUCTR2008-003865-23-GB   United Kingdom;
Glyceryl tri-(4-phenylbutyrate)(GT4P)   
   Hyperion Therapeutics Limited
      2008   -   EUCTR2008-003865-23-GB   United Kingdom;
HHLivC   
   Cytonet GmbH & Co KG
      2008   -   EUCTR2006-000136-27-DE   Germany;
   PROMETHERA Biosciences S.A./N.V.
      -   Phase 2   EUCTR2015-000988-14-Outside-EU/EEA   Canada;United States;
HPN-100   
   Hyperion Therapeutics Limited
      2008   -   EUCTR2008-003865-23-GB   United Kingdom;
HepaStem   
   Promethera Biosciences
      2015   Phase 2   EUCTR2014-000650-11-PL   Belgium;France;Poland;Spain;
      2015   Phase 2   EUCTR2014-000650-11-FR   Belgium;France;Poland;Spain;
      2015   -   EUCTR2014-000650-11-ES   Belgium;Spain;
      2014   Phase 2   EUCTR2014-000650-11-BE   Belgium;France;Poland;Spain;
      2014   Phase 1;Phase 2   EUCTR2013-001045-14-PT   Belgium;Israel;Italy;Portugal;United Kingdom;
      2014   Phase 1;Phase 2   EUCTR2013-001045-14-IT   Belgium;Israel;Italy;Portugal;United Kingdom;
      2014   Phase 1;Phase 2   EUCTR2013-001045-14-GB   Belgium;Italy;Portugal;United Kingdom;
      2013   Phase 1;Phase 2   EUCTR2013-001045-14-BE   Belgium;Italy;Portugal;United Kingdom;
      2012   -   EUCTR2011-004074-28-GB   Belgium;Italy;United Kingdom;
      2012   -   EUCTR2011-004074-28-BE   Belgium;Italy;United Kingdom;
HepaStem Infusion   
   HLB Cell Co., Ltd.
      2018   Phase 2   NCT03884959   Korea, Republic of;
Heparesc   
   PROMETHERA Biosciences S.A./N.V.
      -   Phase 2   EUCTR2015-000988-14-Outside-EU/EEA   Canada;United States;
Heterologous Human Adult Liver-derived Progenitor Cells   
   Promethera Biosciences
      2015   Phase 2   EUCTR2014-000650-11-PL   Belgium;France;Poland;Spain;
      2015   Phase 2   EUCTR2014-000650-11-FR   Belgium;France;Poland;Spain;
      2014   Phase 2   EUCTR2014-000650-11-BE   Belgium;France;Poland;Spain;
      2014   Phase 1;Phase 2   EUCTR2013-001045-14-PT   Belgium;Israel;Italy;Portugal;United Kingdom;
      2014   Phase 1;Phase 2   EUCTR2013-001045-14-IT   Belgium;Israel;Italy;Portugal;United Kingdom;
      2014   Phase 1;Phase 2   EUCTR2013-001045-14-GB   Belgium;Italy;Portugal;United Kingdom;
      2013   Phase 1;Phase 2   EUCTR2013-001045-14-BE   Belgium;Italy;Portugal;United Kingdom;
      2012   -   EUCTR2011-004074-28-GB   Belgium;Italy;United Kingdom;
      2012   -   EUCTR2011-004074-28-BE   Belgium;Italy;United Kingdom;
Heterologous Human Adult Liver-derived Progenitor Cells (HHALPC)   
   PROMETHERA BIOSCIENCES
      2012   -   EUCTR2011-004074-28-IT   Belgium;Italy;United Kingdom;
Human Heterologous Liver Cells   
   Cytonet GmbH & Co. KG
      2008   Phase 2   NCT00718627   Germany;
Human heterologous liver cells (for infusion)   
   Cytonet GmbH & Co KG
      2008   -   EUCTR2006-000136-27-DE   Germany;
Isotopic Intravenous [13C]-Urea   
   Nicholas Ah Mew
      2016   Phase 1/Phase 2   NCT02670889   -
KB195   
   Kaleido Biosciences
      2020   Phase 2   EUCTR2018-004842-40-FR   Belgium;Canada;France;Germany;Mexico;Netherlands;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;United States;
      2019   Phase 2   NCT03933410   Belgium;Germany;Mexico;Spain;Switzerland;Turkey;United Kingdom;United States;
      2019   Phase 2   EUCTR2018-004842-40-ES   Belgium;Canada;France;Germany;Mexico;Netherlands;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;United States;
      2019   Phase 2   EUCTR2018-004842-40-DE   Belgium;Canada;France;Germany;Mexico;Netherlands;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;United States;
      2019   Phase 2   EUCTR2018-004842-40-BE   Belgium;Canada;France;Germany;Mexico;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;United States;
      2019   Phase 1;Phase 2   EUCTR2018-004842-40-GB   Belgium;Canada;France;Germany;Mexico;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom;United States;
MRNA encoding modified Ornithine transcarbamylase   
   Arcturus Therapeutics, Inc.
      2022   Phase 2   EUCTR2021-001081-38-ES   Belgium;Spain;United Kingdom;
      -   Phase 2   EUCTR2021-001081-38-BE   Belgium;United Kingdom;
MRT5201   
   Translate Bio, Inc.
      2019   Phase 1/Phase 2   NCT03767270   -
NaPBA   
   Horizon Therapeutics, LLC
      2018   Phase 4   NCT03335488   Belgium;Italy;Spain;Switzerland;United States;
Neo-ASA   
   Baylor College of Medicine
      2017   -   NCT03064048   United States;
Nitric oxide supplement   
   Baylor College of Medicine
      2014   -   NCT02252770   United States;
Oral prednisone   
   Ultragenyx Pharmaceutical Inc
      2017   Phase 1/Phase 2   NCT02991144   Canada;Spain;United Kingdom;United States;
Ornithine transcarbamylase vector   
   National Center for Research Resources (NCRR)
      1999   Phase 1   NCT00004307   United States;
Promethera HepaStem   
   PROMETHERA BIOSCIENCES
      2012   -   EUCTR2011-004074-28-IT   Belgium;Italy;United Kingdom;
SYNB1020   
   Synlogic
      2017   Phase 1   NCT03179878   United States;
Sodium Phenylbutyrate   
   Brendan Lee
      2008   Phase 2   NCT00345605   United States;
recombinant adenovirus containing the ornithine transcarbamylase gene   
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      1995   Phase 1   NCT00004386   -
scAAV8OTC   
   Ultragenyx Pharmaceutical Inc
      2017   Phase 1/Phase 2   NCT02991144   Canada;Spain;United Kingdom;United States;