201. Angelman syndrome Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 25 / Drugs : 40 - (DrugBank : 11) / Drug target genes : 22 - Drug target pathways : 20
Drugs and their primary sponsors and trial info
Acylated and unacylated ghrelin dosages
University Hospital, Toulouse
2021 - NCT04768803 -
Aknemin 50
Dra. Cristina Avendaño Solá
2013 - EUCTR2013-002154-67-ES Spain;
Betaine
University of California, San Diego
2006 - NCT00348933 United States;
Creatine
University of California, San Diego
2006 - NCT00348933 United States;
GTX-102
GeneTX Biotherapeutics, LLC
2020 Phase 1/Phase 2 NCT04259281 Canada;United Kingdom;United States;
Gaboxadol
Ovid Therapeutics Inc.
2019 Phase 3 NCT04106557 Australia;Germany;Israel;Netherlands;United States;
Gaboxadol monohydrate 0.5 mg (intended commercial formulation)
Ovid Therapeutics Inc.
2020 Phase 3 EUCTR2019-004478-24-DE 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 (intended commercial formulation)
Ovid Therapeutics Inc.
2020 Phase 3 EUCTR2019-004478-24-DE 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 (intended commercial formulation)
Ovid Therapeutics Inc.
2020 Phase 3 EUCTR2019-004478-24-DE Australia;Germany;Israel;Netherlands;United States;
ION582
Ionis Pharmaceuticals, Inc.
2021 Phase 1/Phase 2 NCT05127226 United States;
Levodopa
Bennett Lavenstein
2013 - NCT03235037 -
Wen-Hann Tan
2011 Phase 2/Phase 3 NCT01281475 United States;
Levodopa/Carbidopa (4:1)
Boston Children’s Hospital
2009 Phase 1 NCT00829439 United States;
MINOCYCLINE HYDROCHLORIDE
Dra. Cristina Avendaño Solá
2013 - EUCTR2013-002154-67-ES Spain;
Metafolin
University of California, San Diego
2006 - NCT00348933 United States;
Minocycline
University of South Florida
2012 - NCT01531582 United States;
NNZ-2591
Neuren Pharmaceuticals Limited
2021 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;
Vitamin B12
University of California, San Diego
2006 - NCT00348933 United States;
University Hospital, Toulouse
2021 - NCT04768803 -
Aknemin 50
Dra. Cristina Avendaño Solá
2013 - EUCTR2013-002154-67-ES Spain;
Betaine
University of California, San Diego
2006 - NCT00348933 United States;
Creatine
University of California, San Diego
2006 - NCT00348933 United States;
GTX-102
GeneTX Biotherapeutics, LLC
2020 Phase 1/Phase 2 NCT04259281 Canada;United Kingdom;United States;
Gaboxadol
Ovid Therapeutics Inc.
2019 Phase 3 NCT04106557 Australia;Germany;Israel;Netherlands;United States;
Gaboxadol monohydrate 0.5 mg (intended commercial formulation)
Ovid Therapeutics Inc.
2020 Phase 3 EUCTR2019-004478-24-DE 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 (intended commercial formulation)
Ovid Therapeutics Inc.
2020 Phase 3 EUCTR2019-004478-24-DE 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 (intended commercial formulation)
Ovid Therapeutics Inc.
2020 Phase 3 EUCTR2019-004478-24-DE Australia;Germany;Israel;Netherlands;United States;
ION582
Ionis Pharmaceuticals, Inc.
2021 Phase 1/Phase 2 NCT05127226 United States;
Levodopa
Bennett Lavenstein
2013 - NCT03235037 -
Wen-Hann Tan
2011 Phase 2/Phase 3 NCT01281475 United States;
Levodopa/Carbidopa (4:1)
Boston Children’s Hospital
2009 Phase 1 NCT00829439 United States;
MINOCYCLINE HYDROCHLORIDE
Dra. Cristina Avendaño Solá
2013 - EUCTR2013-002154-67-ES Spain;
Metafolin
University of California, San Diego
2006 - NCT00348933 United States;
Minocycline
University of South Florida
2012 - NCT01531582 United States;
NNZ-2591
Neuren Pharmaceuticals Limited
2021 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;
Vitamin B12
University of California, San Diego
2006 - NCT00348933 United States;