201. Angelman syndrome Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 40 / Drugs : 48 - (DrugBank : 11) / Drug target genes : 22 - Drug target pathways : 22
Drugs, Primary sponsors and Trial info (Enrollment year, Phase, Trial ID, Countries)
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
Blood collection
Biogen
2019 Early Phase 1 NCT04103333 United States
Carbidopa
Boston Children’s Hospital
2009 Phase 1 NCT00829439 United States
Creatine
University of California, San Diego
2006 - NCT00348933 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
Elliotts B® solution (buffered intrathecal electrolyte/dextrose injection)product name: elliotts B® solutionproduct code: elliotts B® solutionpharmaceutical form: solution FOR injection
Ultragenyx Pharmaceutical Inc.
2023 Phase 1;Phase 2 EUCTR2021-001793-36-ES 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
2025 Phase 2 NCT07157254 Argentina;Brazil;France;Israel;Italy;Portugal;United Kingdom;United States
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
GTX-102product code: GTX-102pharmaceutical form: solution FOR injectioninn or proposed INN: NOT availablecurrent sponsor code: GTX-102concentration unit: MG/ML milligram(S)/millilitreconcentration type: equalconcentration number: 11-
Ultragenyx Pharmaceutical Inc.
2023 Phase 1;Phase 2 EUCTR2021-001793-36-ES 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 Australia;Canada;France;Germany;Israel;Italy;Japan;Poland;Singapore;South Korea;Spain;United Kingdom;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
Junge Candice
2025 Phase 3 JPRN-jRCT2051250098 Argentina;Australia;Belgium;Canada;Colombia;Ireland;Israel;Italy;Japan;Poland;Singapore;South Korea;Spain;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
Lumbar puncture
Biogen
2019 Early Phase 1 NCT04103333 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
MVX-220
MavriX Bio, LLC
2025 Phase 1/Phase 2 NCT07181837 United States
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
Healx AI
2019 Phase 3 NCT03882918 Israel;United States
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
2017 Phase 1 NCT03109756 United States
- Phase 3 EUCTR2019-004478-24-NL Australia;Germany;Israel;Netherlands;United States
OV101 regimen 1
Healx AI
2016 Phase 2 NCT02996305 Israel;United States
OV101 regimen 2
Healx AI
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
Rugonersen
OHB Pediatrics Ltd.
2026 Phase 3 NCT07605429 -
Oak Hill Bio Ltd
2025 - NCT07136454 -
Sham procedure
OHB Pediatrics Ltd.
2026 Phase 3 NCT07605429 -
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
Blood collection
Biogen
2019 Early Phase 1 NCT04103333 United States
Carbidopa
Boston Children’s Hospital
2009 Phase 1 NCT00829439 United States
Creatine
University of California, San Diego
2006 - NCT00348933 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
Elliotts B® solution (buffered intrathecal electrolyte/dextrose injection)product name: elliotts B® solutionproduct code: elliotts B® solutionpharmaceutical form: solution FOR injection
Ultragenyx Pharmaceutical Inc.
2023 Phase 1;Phase 2 EUCTR2021-001793-36-ES 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
2025 Phase 2 NCT07157254 Argentina;Brazil;France;Israel;Italy;Portugal;United Kingdom;United States
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
GTX-102product code: GTX-102pharmaceutical form: solution FOR injectioninn or proposed INN: NOT availablecurrent sponsor code: GTX-102concentration unit: MG/ML milligram(S)/millilitreconcentration type: equalconcentration number: 11-
Ultragenyx Pharmaceutical Inc.
2023 Phase 1;Phase 2 EUCTR2021-001793-36-ES 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 Australia;Canada;France;Germany;Israel;Italy;Japan;Poland;Singapore;South Korea;Spain;United Kingdom;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
Junge Candice
2025 Phase 3 JPRN-jRCT2051250098 Argentina;Australia;Belgium;Canada;Colombia;Ireland;Israel;Italy;Japan;Poland;Singapore;South Korea;Spain;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
Lumbar puncture
Biogen
2019 Early Phase 1 NCT04103333 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
MVX-220
MavriX Bio, LLC
2025 Phase 1/Phase 2 NCT07181837 United States
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
Healx AI
2019 Phase 3 NCT03882918 Israel;United States
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
2017 Phase 1 NCT03109756 United States
- Phase 3 EUCTR2019-004478-24-NL Australia;Germany;Israel;Netherlands;United States
OV101 regimen 1
Healx AI
2016 Phase 2 NCT02996305 Israel;United States
OV101 regimen 2
Healx AI
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
Rugonersen
OHB Pediatrics Ltd.
2026 Phase 3 NCT07605429 -
Oak Hill Bio Ltd
2025 - NCT07136454 -
Sham procedure
OHB Pediatrics Ltd.
2026 Phase 3 NCT07605429 -
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
