201. アンジェルマン症候群 Disease details / Clinical trials / Drug dev / DR info


臨床試験数 : 33 / 薬物数 : 32 - (DrugBank : 13) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 21

薬物、開発者(Primary Sponsor)、臨床試験情報(症例登録開始年(Enrollment year)、フェーズ、ID、実施国)
Acylated and unacylated ghrelin dosages
   University Hospital, Toulouse
      2021   -   NCT04768803   France
Aknemin 50
   Dra. Cristina Avendaño Solá
      2013   -   EUCTR2013-002154-67-ES   Spain
Alogabat
   Hoffmann-La Roche
      2023   Phase 2   NCT05630066   Australia;France;Germany;Italy;Spain;United States
Betaine
   University of California, San Diego
      2006   -   NCT00348933   United States
Carbidopa
   Boston Children’s Hospital
      2009   Phase 1   NCT00829439   United States
Creatine
   University of California, San Diego
      2006   -   NCT00348933   United States
Dextrose
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 1;Phase 2   EUCTR2021-001793-36-FR   Australia;Canada;France;Germany;Israel;Spain;United Kingdom;United States
Elliotts B® solution
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 1;Phase 2   EUCTR2021-001793-36-FR   Australia;Canada;France;Germany;Israel;Spain;United Kingdom;United States
Gaboxadol
   Ovid Therapeutics Inc.
      2019   Phase 3   NCT04106557   Australia;Germany;Israel;Netherlands;United States
Gaboxadol monohydrate 0.5 MG
   Ovid Therapeutics Inc.
      2020   Phase 3   EUCTR2019-004478-24-DE   Australia;Germany;Israel;Netherlands;United States
      -   Phase 3   EUCTR2019-004478-24-NL   Australia;Germany;Israel;Netherlands;United States
Gaboxadol monohydrate 0.5 MG capsule
   Ovid Therapeutics Inc.
      2020   Phase 3   EUCTR2019-002907-17-NL   Australia;Germany;Israel;Italy;Netherlands;United States
Gaboxadol monohydrate 2 MG
   Ovid Therapeutics Inc.
      2020   Phase 3   EUCTR2019-004478-24-DE   Australia;Germany;Israel;Netherlands;United States
      -   Phase 3   EUCTR2019-004478-24-NL   Australia;Germany;Israel;Netherlands;United States
Gaboxadol monohydrate 2 MG capsule
   Ovid Therapeutics Inc.
      2020   Phase 3   EUCTR2019-002907-17-NL   Australia;Germany;Israel;Italy;Netherlands;United States
Gaboxadol monohydrate 5 MG
   Ovid Therapeutics Inc.
      2020   Phase 3   EUCTR2019-004478-24-DE   Australia;Germany;Israel;Netherlands;United States
      -   Phase 3   EUCTR2019-004478-24-NL   Australia;Germany;Israel;Netherlands;United States
GTX-102
   Ultragenyx Pharmaceutical Inc
      2024   Phase 3   NCT06617429   Australia;Canada;Germany;Japan;Netherlands;Poland;Spain;United States
      2024   Phase 3   NCT06415344   Australia;Canada;France;Germany;Israel;Spain;United Kingdom;United States
      2020   Phase 1/Phase 2   NCT04259281   Australia;Canada;France;Germany;Israel;Spain;United Kingdom;United States
   Ultragenyx Pharmaceutical Inc.
      2023   Phase 1;Phase 2   EUCTR2021-001793-36-FR   Australia;Canada;France;Germany;Israel;Spain;United Kingdom;United States
      2023   Phase 1;Phase 2   EUCTR2021-001793-36-DE   Australia;Canada;France;Germany;Israel;Spain;United Kingdom;United States
ION582
   IONIS PHARMACEUTICALS, INC.
      2022   Phase 1;Phase 2   EUCTR2021-003009-23-IT   Argentina;Australia;Canada;France;Germany;Israel;Italy;Netherlands;Spain;United Kingdom;United States
   Ionis Pharmaceuticals, Inc.
      2025   Phase 3   NCT06914609   United States
      2022   Phase 1;Phase 2   EUCTR2021-003009-23-FR   Argentina;Australia;Canada;France;Germany;Israel;Italy;Netherlands;Spain;United Kingdom;United States
      2021   Phase 1/Phase 2   NCT05127226   Australia;France;Israel;Italy;United Kingdom;United States
Levodopa
   Bennett Lavenstein
      2013   -   NCT03235037   -
   Boston Children’s Hospital
      2009   Phase 1   NCT00829439   United States
   Wen-Hann Tan
      2011   Phase 2/Phase 3   NCT01281475   United States
Metafolin
   University of California, San Diego
      2006   -   NCT00348933   United States
Minocycline
   Puerta de Hierro University Hospital
      2014   Phase 2   NCT02056665   Spain
   University of South Florida
      2012   -   NCT01531582   United States
Minocycline hydrochloride
   Dra. Cristina Avendaño Solá
      2013   -   EUCTR2013-002154-67-ES   Spain
NNZ-2591
   Neuren Pharmaceuticals Limited
      2022   Phase 2   NCT05011851   Australia
NON disponibile
   F. HOFFMANN - LA ROCHE LTD.
      2020   Phase 1   EUCTR2019-003787-48-IT   Canada;Italy;Netherlands;Spain;United Kingdom;United States
Nutritional formulation containing exogenous ketones
   University of Colorado, Denver
      2018   -   NCT03644693   United States
OV101
   Ovid Therapeutics Inc.
      2020   Phase 3   EUCTR2019-004478-24-DE   Australia;Germany;Israel;Netherlands;United States
      2020   Phase 3   EUCTR2019-002907-17-NL   Australia;Germany;Israel;Italy;Netherlands;United States
      2019   Phase 2   NCT03882918   Israel;United States
      2017   Phase 1   NCT03109756   United States
      -   Phase 3   EUCTR2019-004478-24-NL   Australia;Germany;Israel;Netherlands;United States
OV101 regimen 1
   Ovid Therapeutics Inc.
      2016   Phase 2   NCT02996305   Israel;United States
OV101 regimen 2
   Ovid Therapeutics Inc.
      2016   Phase 2   NCT02996305   Israel;United States
RO7248824
   Hoffmann-La Roche
      2021   Phase 1   NCT04863794   Netherlands
      2020   Phase 1   NCT04428281   Canada;Italy;Netherlands;Spain;United Kingdom;United States
RO7248824/F02-01
   F. HOFFMANN - LA ROCHE LTD.
      2020   Phase 1   EUCTR2019-003787-48-IT   Canada;Italy;Netherlands;Spain;United Kingdom;United States
RO7248824/F03-01 (diluent FOR RO7248824/F02-01
   F. Hoffmann-La Roche
      2020   Phase 1   EUCTR2019-003787-48-NL   Canada;Italy;Netherlands;Spain;United Kingdom;United States
      2020   Phase 1   EUCTR2019-003787-48-GB   Canada;Italy;Netherlands;Spain;United Kingdom;United States
SHAM-LP
   Ultragenyx Pharmaceutical Inc
      2024   Phase 3   NCT06617429   Australia;Canada;Germany;Japan;Netherlands;Poland;Spain;United States
Taurine
   Hospital of Fudan University at Xiamen
      2024   -   ChiCTR2400082614   China
Vitamin B12
   University of California, San Diego
      2006   -   NCT00348933   United States