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
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