251. 尿素サイクル異常症 Disease details / Clinical trials / Drug dev / DR info

Aavlk03hotc
   University College, London
      2023   Phase 1/Phase 2   NCT05092685   United Kingdom
Acetohydroxamic acid
   Nicholas Ah Mew
      2017   Phase 1/Phase 2   NCT03181828   United States
      2016   Phase 1/Phase 2   NCT02670889   -
Adenoviral vector-mediated gene transfer
   University of Pennsylvania
      1998   Phase 1   NCT00004498   -
Allogenic human heterologous liver cells
   PROMETHERA Biosciences S.A./N.V.
      -   Phase 2   EUCTR2015-000988-14-Outside-EU/EEA   Canada;United States
Aminoacid, alanine-infusion
   University of Aarhus
      2021   -   NCT05076318   Denmark
Ammonia
   Hiroiku Furukawa
      2023   Phase 3   JPRN-jRCT2071220110   -
Ammonium (15N) chloride
   Unicyte AG
      2022   Phase 1   EUCTR2021-000824-36-IT   Austria;Belgium;Czechia;France;Germany;Israel;Italy;Netherlands;Poland;Portugal;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom
      2022   Phase 1   EUCTR2021-000824-36-DE   Austria;Belgium;France;Germany;Israel;Italy;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom
      2021   Phase 1   EUCTR2021-000824-36-ES   Austria;Belgium;Czechia;France;Germany;Israel;Italy;Netherlands;Poland;Portugal;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom
      2021   Phase 1   EUCTR2021-000824-36-AT   Austria;Belgium;France;Germany;Israel;Italy;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
Ammonium chloride
   Unicyte AG
      2022   Phase 1   EUCTR2021-000824-36-IT   Austria;Belgium;Czechia;France;Germany;Israel;Italy;Netherlands;Poland;Portugal;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom
      2022   Phase 1   EUCTR2021-000824-36-DE   Austria;Belgium;France;Germany;Israel;Italy;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom
      2021   Phase 1   EUCTR2021-000824-36-ES   Austria;Belgium;Czechia;France;Germany;Israel;Italy;Netherlands;Poland;Portugal;Saudi Arabia;Spain;Switzerland;Turkey;United Kingdom
      2021   Phase 1   EUCTR2021-000824-36-AT   Austria;Belgium;France;Germany;Israel;Italy;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
ARCT-810
   Arcturus Therapeutics, Inc.
      2024   Phase 2   NCT06488313   United States
      2023   Phase 2   EUCTR2021-001081-38-IT   Belgium;France;Italy;Spain;Sweden;United Kingdom
      2022   Phase 2   NCT05526066   Belgium;France;Italy;Spain;Sweden;United Kingdom
      2022   Phase 2   EUCTR2021-001081-38-FR   Belgium;France;Italy;Spain;Sweden;United Kingdom
      2022   Phase 2   EUCTR2021-001081-38-ES   Belgium;France;Italy;Spain;Sweden;United Kingdom
      2021   Phase 2   EUCTR2021-001081-38-BE   Belgium;Italy;Spain;Sweden;United Kingdom
      2020   Phase 1   NCT04442347   United States
      2020   Phase 1   NCT04416126   New Zealand
      -   Phase 2   EUCTR2021-001081-38-SE   Belgium;France;Italy;Spain;Sweden;United Kingdom
Arginine
   Brendan Lee
      2008   Phase 2   NCT00345605   United States
   University of Milan
      2023   -   NCT05412160   Italy
Avalotcagene ontaparvovec
   ULTRAGENYX PHARMACEUTICAL INC.
      2023   Phase 3   EUCTR2020-003384-25-IT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 3   EUCTR2020-003384-25-PT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-FR   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-ES   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-DE   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
Carglumic acid
   Ito Tetsuya
      2019   Phase 4   JPRN-jRCTs041190055   -
Celulas hepatocitos troncales adultas alogenicas DE higado expandidas
   Promethera Biosciences
      2015   Phase 2   EUCTR2014-000650-11-ES   Belgium;France;Poland;Spain
DTX301
   Dimension Therapeutics, Inc.
      2017   Phase 1;Phase 2   EUCTR2016-001057-40-ES   Canada;France;Spain;United Kingdom;United States
   Kajiwara Makoto
      2023   Phase 3   JPRN-jRCT2073220097   Argentina;Australia;Brazil;Canada;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
   ULTRAGENYX PHARMACEUTICAL INC.
      2023   Phase 3   EUCTR2020-003384-25-IT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc
      2022   Phase 3   NCT05345171   Argentina;Australia;Brazil;Canada;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 3   EUCTR2020-003384-25-PT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-FR   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-ES   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-DE   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;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;France;Spain;United Kingdom;United States
      2017   Phase 1;Phase 2   EUCTR2016-001057-40-GB   Canada;Spain;United Kingdom;United States
ECUR-506
   iECURE, Inc.
      2024   Phase 1/Phase 2   NCT06255782   Australia;Spain;United Kingdom;United States
Ethanoate
   ULTRAGENYX PHARMACEUTICAL INC.
      2023   Phase 3   EUCTR2020-003384-25-IT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 3   EUCTR2020-003384-25-PT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-FR   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-ES   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-DE   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
GT4P
   Hyperion Therapeutics Limited
      2008   -   EUCTR2008-003865-23-GB   United Kingdom
Heparesc
   PROMETHERA Biosciences S.A./N.V.
      -   Phase 2   EUCTR2015-000988-14-Outside-EU/EEA   Canada;United States
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   Phase 2   EUCTR2014-000650-11-ES   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
Hepastem infusion
   HLB Cell Co., Ltd.
      2018   Phase 2   NCT03884959   Korea, Republic of
Heterologous human adult liver-derived progenitor cells
   PROMETHERA BIOSCIENCES
      2012   -   EUCTR2011-004074-28-IT   Belgium;Italy;United Kingdom
   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
Hhalpc
   PROMETHERA BIOSCIENCES
      2012   -   EUCTR2011-004074-28-IT   Belgium;Italy;United Kingdom
   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   Phase 2   EUCTR2014-000650-11-ES   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
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
   Hiroiku Furukawa
      2023   Phase 3   JPRN-jRCT2071220110   -
   Hyperion Therapeutics Limited
      2008   -   EUCTR2008-003865-23-GB   United Kingdom
Human heterologous liver cells
   Cytonet GmbH & Co KG
      2008   -   EUCTR2006-000136-27-DE   Germany
   Cytonet GmbH & Co. KG
      2008   Phase 2   NCT00718627   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 1;Phase 2   EUCTR2018-004842-40-GB   Belgium;Canada;France;Germany;Mexico;Saudi Arabia;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
Mrna codificante PER ornitina transcarbamilasi modificata
   Arcturus Therapeutics, Inc.
      2023   Phase 2   EUCTR2021-001081-38-IT   Belgium;France;Italy;Spain;Sweden;United Kingdom
Mrna encoding modified ornithine transcarbamylase
   Arcturus Therapeutics, Inc.
      2022   Phase 2   EUCTR2021-001081-38-FR   Belgium;France;Italy;Spain;Sweden;United Kingdom
      2022   Phase 2   EUCTR2021-001081-38-ES   Belgium;France;Italy;Spain;Sweden;United Kingdom
      2021   Phase 2   EUCTR2021-001081-38-BE   Belgium;Italy;Spain;Sweden;United Kingdom
      -   Phase 2   EUCTR2021-001081-38-SE   Belgium;France;Italy;Spain;Sweden;United Kingdom
MRT5201
   Translate Bio, Inc.
      2019   Phase 1/Phase 2   NCT03767270   -
Napba
   Amgen
      2018   Phase 4   NCT03335488   Belgium;Italy;Spain;Switzerland;United States
   Hiroiku Furukawa
      2023   Phase 3   JPRN-jRCT2071220110   -
NEO-ASA
   Baylor College of Medicine
      2017   -   NCT03064048   United States
Nitric oxide supplement
   Baylor College of Medicine
      2014   -   NCT02252770   United States
Oral corticosteroids
   Ultragenyx Pharmaceutical Inc
      2022   Phase 3   NCT05345171   Argentina;Australia;Brazil;Canada;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
Ornithine transcarbamylase vector
   National Center for Research Resources (NCRR)
      1999   Phase 1   NCT00004307   United States
Phenylbutyrate
   Hyperion Therapeutics Limited
      2008   -   EUCTR2008-003865-23-GB   United Kingdom
Prednisolon
   ULTRAGENYX PHARMACEUTICAL INC.
      2023   Phase 3   EUCTR2020-003384-25-IT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
Prednisolone
   ULTRAGENYX PHARMACEUTICAL INC.
      2023   Phase 3   EUCTR2020-003384-25-IT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
Prednisone
   ULTRAGENYX PHARMACEUTICAL INC.
      2023   Phase 3   EUCTR2020-003384-25-IT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
Promethera hepastem
   PROMETHERA BIOSCIENCES
      2012   -   EUCTR2011-004074-28-IT   Belgium;Italy;United Kingdom
Prophylactic corticosteroid taper regimen
   Ultragenyx Pharmaceutical Inc
      2017   Phase 1/Phase 2   NCT02991144   Canada;Spain;United Kingdom;United States
Protein and calorie controlled diet
   National Center for Research Resources (NCRR)
      1999   Phase 1   NCT00004307   United States
Ravicti
   Amgen
      2018   Phase 4   NCT03335488   Belgium;Italy;Spain;Switzerland;United States
      2014   Phase 4   NCT02246218   Canada;United States
Reactive corticosteroid taper regimen
   Ultragenyx Pharmaceutical Inc
      2017   Phase 1/Phase 2   NCT02991144   Canada;Spain;United Kingdom;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
Sodium acetate
   ULTRAGENYX PHARMACEUTICAL INC.
      2023   Phase 3   EUCTR2020-003384-25-IT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc
      2022   Phase 3   NCT05345171   Argentina;Australia;Brazil;Canada;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 3   EUCTR2020-003384-25-PT   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-FR   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-ES   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2020-003384-25-DE   Argentina;Australia;Brazil;Canada;Croatia;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;United Kingdom;United States
Sodium phenylbutyrate
   Brendan Lee
      2008   Phase 2   NCT00345605   United States
   Hiroiku Furukawa
      2023   Phase 3   JPRN-jRCT2071220110   -
SYNB1020
   Synlogic
      2017   Phase 1   NCT03179878   United States
Urea cycle flux study
   University Children's Hospital, Zurich
      2019   -   NCT05671666   Switzerland