251. 尿素サイクル異常症 Disease details / Clinical trials / Drug dev / DR info
臨床試験数 : 54 / 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28
薬物ごとの開発者（Primary Sponsor）、臨床試験情報（抜粋）です。
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;
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;