193. プラダー・ウィリ症候群 Disease details / Clinical trials / Drug dev / DR info


臨床試験数 : 128 / 薬物数 : 94 - (DrugBank : 25) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 102

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)
1.2 MG ZGN-440 FOR injectable suspension
   Zafgen, Inc.
      2013   Phase 2   NCT01818921   United States
1.8 MG ZGN-440 FOR injectable suspension
   Zafgen, Inc.
      2013   Phase 2   NCT01818921   United States
Acetylcysteine
   Dutch Growth Research Foundation
      2021   Phase 2;Phase 3   EUCTR2019-000735-61-NL   Netherlands
Acomplia 20 MG FILM-coated tablets
   Karolinska University Hospital
      2008   -   EUCTR2007-006305-25-SE   Sweden
Acylated and unacylated ghrelin dosages
   University Hospital, Toulouse
      2021   -   NCT04768803   France
Anastrozole
   Assistance Publique - Hôpitaux de Paris
      2012   -   NCT01520467   France
ARD-101
   Aardvark Therapeutics, Inc.
      2024   Phase 3   NCT06828861   Australia;Canada;France;Italy;Korea, Republic of;Romania;Spain;United Kingdom;United States
      2022   Phase 2   NCT05153434   United States
AZP-531
   Alize Pharma
      2015   -   EUCTR2014-001670-34-IT   France;Italy;Spain
      2015   Phase 2   EUCTR2014-001670-34-FR   France;Italy;Spain
      2015   Phase 2   EUCTR2014-001670-34-ES   France;Italy;Spain
   Millendo Therapeutics SAS
      2020   Phase 2;Phase 3   EUCTR2018-003062-13-FR   Belgium;Canada;France;Italy;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-003062-13-NL   Australia;Belgium;France;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-003062-13-IT   Belgium;Canada;France;Italy;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-003062-13-GB   Australia;Belgium;France;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-003062-13-ES   Belgium;Canada;France;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-003062-13-BE   Australia;Belgium;France;Netherlands;Spain;United Kingdom;United States
B. lactis B94
   University of Florida
      2017   -   NCT03277157   United States
Biopsy
   University Hospital, Toulouse
      2009   Phase 4   NCT01298180   France
Byetta
   Aintree University Hospital NHS Foundation Trust
      2011   -   EUCTR2010-023179-25-GB   United Kingdom
Cannabidiol
   Benuvia Therapeutics Inc.
      2018   Phase 2   NCT02844933   United States
   Radius Pharmaceuticals, Inc.
      2022   Phase 2;Phase 3   EUCTR2021-005331-23-ES   Australia;Canada;France;Italy;Spain;Sweden;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-005331-23-SE   Australia;Belgium;Canada;France;Italy;Spain;Sweden;United Kingdom;United States
Cannabidiol oral solution
   Benuvia Therapeutics Inc.
      2018   Phase 2   NCT03458416   United States
Carbetocin
   ACADIA Pharmaceuticals Inc.
      2024   Phase 3   NCT06420297   Canada;Germany;United Kingdom;United States
      2023   Phase 3   NCT06173531   Canada;France;Germany;Spain;United Kingdom;United States
Cbdv compound
   Montefiore Medical Center
      2020   Phase 2   NCT03848481   United States
Coenzyme Q10
   The Hospital for Sick Children
      2023   Phase 2   NCT03831425   Canada
CSTI-500
   ConSynance Therapeutics
      2022   Phase 1   NCT05504395   United States
Dccr
   Essentialis, Inc.
      2014   Phase 1/Phase 2   NCT02034071   United States
   Soleno Therapeutics UK Ltd.
      2019   Phase 3   EUCTR2018-004216-22-GB   United Kingdom;United States
      2019   Phase 3   EUCTR2018-004215-50-GB   United Kingdom;United States
   Soleno Therapeutics, Inc.
      2023   Phase 3   NCT05701774   United Kingdom;United States
      2019   Phase 3   NCT04086810   -
      2018   Phase 3   NCT03714373   United Kingdom;United States
      2018   Phase 3   NCT03440814   United Kingdom;United States
Diazoxide choline
   Soleno Therapeutics UK Ltd.
      2019   Phase 3   EUCTR2018-004216-22-GB   United Kingdom;United States
      2019   Phase 3   EUCTR2018-004215-50-GB   United Kingdom;United States
Diazoxide choline controlled-release tablet
   Essentialis, Inc.
      2017   Phase 2   NCT02893618   -
Eating
   Corporacion Parc Tauli
      2025   -   NCT06900335   Spain
Eutropin
   LG Life Sciences
      2014   Phase 3   NCT02204163   Korea, Republic of
Exenatide
   Aintree University Hospital NHS Foundation Trust
      2011   -   EUCTR2010-023179-25-GB   United Kingdom
   Children's Hospital Los Angeles
      2012   -   NCT01444898   United States
   Garvan Institute of Medical Research
      2007   -   NCT00551343   Australia
FE 992097
   Ferring Pharmaceuticals
      2014   Phase 2   NCT01968187   United States
Fluimucil
   Dutch Growth Research Foundation
      2021   Phase 2;Phase 3   EUCTR2019-000735-61-NL   Netherlands
Follow-UP study OF THE treated cohort
   University Hospital, Toulouse
      2021   Phase 3   NCT05032326   France
Follow-UP study OF THE untreated cohort
   University Hospital, Toulouse
      2021   Phase 3   NCT05032326   France
Genotonorm
   CHU TOULOUSE
      2008   -   EUCTR2008-004612-12-FR   France
   University Hospital, Toulouse
      2009   Phase 4   NCT01298180   France
Genotonorm miniquick 0,2 MG
   Fundació Parc Taulí
      2017   Phase 4   EUCTR2017-002164-41-ES   Spain
Genotropin
   Dutch Growth Foundation
      2008   Phase 4   EUCTR2007-004716-31-NL   Netherlands
   Dutch growth research foundation
      2012   -   EUCTR2011-001313-14-NL   Netherlands
   LG Life Sciences
      2014   Phase 3   NCT02204163   Korea, Republic of
Genotropin goquick
   Pfizer, Inc.
      -   Phase 3   EUCTR2024-000101-32-Outside-EU/EEA   Japan
GLWL-01
   GLWL Research Inc.
      2018   Phase 2   NCT03274856   Canada;United States
Growth hormone
   Children's Hospital of Zhejiang University School of Medicine
      2019   Phase 4   ChiCTR1900027464   China
      2019   -   ChiCTR1900022809   China
   Corporacion Parc Tauli
      2017   Phase 4   NCT03616509   Spain
   Dutch growth research foundation
      2012   -   EUCTR2011-001313-14-NL   Netherlands
   University Hospital, Toulouse
      2009   Phase 4   NCT01298180   France
Guanfacine extended release
   Maimonides Medical Center
      2020   Phase 4   NCT05657860   United States
   NYU Langone Health
      2019   Phase 4   NCT04066088   United States
H01bb02
   Toulouse University Hospital
      2022   Phase 3   EUCTR2021-000094-91-FR   France
   University Hospital Toulouse
      -   Phase 3   EUCTR2019-002385-12-IT   Belgium;France;Germany;Italy;Netherlands
INN NOT YET proposed
   Alize Pharma
      2015   -   EUCTR2014-001670-34-IT   France;Italy;Spain
      2015   Phase 2   EUCTR2014-001670-34-FR   France;Italy;Spain
      2015   Phase 2   EUCTR2014-001670-34-ES   France;Italy;Spain
Intranasal oxytocin
   Eric Hollander
      2018   Phase 2   NCT03197662   United States
   Montefiore Medical Center
      2015   Phase 2   NCT02629991   United States
   University of Florida
      2015   Phase 1   NCT02013258   United States
Liraglutide
   Novo Nordisk A/S
      2016   Phase 3   EUCTR2014-004415-37-NL   Australia;Canada;European Union;France;Italy;Netherlands;New Zealand;Turkey;United States
      2015   Phase 3   NCT02527200   Australia;Canada;France;Italy;Netherlands;New Zealand;Turkey;United States
      2015   Phase 3   EUCTR2014-004415-37-IT   Australia;Canada;European Union;France;Italy;Netherlands;New Zealand;Turkey;United States
      -   Phase 3   EUCTR2014-004415-37-FR   Australia;Canada;European Union;France;Italy;Netherlands;New Zealand;Turkey;United States
   Vancouver General Hospital
      2012   Phase 4   NCT01542242   Canada
Livoletide
   Millendo Therapeutics SAS
      2020   Phase 2;Phase 3   EUCTR2018-003062-13-FR   Belgium;Canada;France;Italy;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2/Phase 3   NCT03790865   Australia;Belgium;France;Italy;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-003062-13-NL   Australia;Belgium;France;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-003062-13-GB   Australia;Belgium;France;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-003062-13-ES   Belgium;Canada;France;Netherlands;Spain;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-003062-13-BE   Australia;Belgium;France;Netherlands;Spain;United Kingdom;United States
LOW dose (1 MCG) acth stimulation test
   Nationwide Children's Hospital
      2014   -   NCT02368379   United States
Medical AIR VS oxygen
   The Hospital for Sick Children
      2016   Phase 4   NCT03031626   Canada
Metoprolol succinate
   Saniona A/S
      2022   Phase 2   EUCTR2021-000127-12-SE   Australia;Belgium;France;Ireland;Italy;New Zealand;Spain;Sweden;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003694-18-CZ   Czech Republic;Hungary
      2016   Phase 2   EUCTR2016-003694-18-HU   Czech Republic;Hungary
Metoprololsuccinat orion 25MG
   Saniona A/S
      2016   Phase 2   EUCTR2016-003694-18-HU   Czech Republic;Hungary
Metoprololsuccinat ”orion” 25MG
   Saniona A/S
      2017   Phase 2   EUCTR2016-003694-18-CZ   Czech Republic;Hungary
NBT-NM108
   Rutgers, The State University of New Jersey
      2023   Phase 2   NCT05541003   United States
NNZ-2591
   Neuren Pharmaceuticals Limited
      2023   Phase 2   NCT05879614   United States
Norditropin simplexx
   Karolinska University Hospital
      2005   -   NCT00372125   Denmark;Norway;Sweden
Nutropin AQ
   Children's Mercy Hospital Kansas City
      2005   Phase 3   NCT00444964   United States
Octreotide
   Duke University
      2006   -   NCT00399893   United States
   Oregon Health and Science University
      2005   -   NCT01613495   -
Omnitrope
   CHU TOULOUSE
      2008   -   EUCTR2008-004612-12-FR   France
   University Hospital, Toulouse
      2009   Phase 4   NCT01298180   France
Ossitocina
   University Hospital Toulouse
      -   Phase 3   EUCTR2019-002385-12-IT   Belgium;France;Germany;Italy;Netherlands
Overnight metyrapone test
   Nationwide Children's Hospital
      2014   -   NCT02368379   United States
Oxytocin
   Dutch Growth Research Foundation
      2017   Phase 2;Phase 3   EUCTR2017-003423-30-NL   Netherlands
      2016   Phase 3   EUCTR2016-003820-22-NL   Netherlands
      2014   Phase 3   EUCTR2013-004134-15-NL   Netherlands
   Toulouse University Hospital
      2022   Phase 3   EUCTR2021-000094-91-FR   France
      2021   Phase 3   EUCTR2020-006161-11-FR   France
   University Hospital Toulouse
      -   Phase 3   EUCTR2019-002385-12-IT   Belgium;France;Germany;Italy;Netherlands
   University Hospital of Toulouse
      2021   Phase 3   EUCTR2019-002385-12-NL   Belgium;France;Germany;Netherlands
      2021   Phase 3   EUCTR2019-002385-12-DE   Belgium;France;Germany;Italy;Netherlands
      2019   Phase 3   EUCTR2019-002385-12-FR   Belgium;France;Germany;Italy;Netherlands
      -   Phase 3   EUCTR2019-002385-12-BE   Belgium;France;Germany;Italy;Netherlands
   University Hospital, Toulouse
      2017   -   NCT03081832   France
      2016   -   NCT03114371   France
      2013   Phase 1/Phase 2   NCT02205034   France
      2011   Phase 1/Phase 2   NCT01548521   France
   University of Florida
      2017   Phase 1/Phase 2   NCT03245762   United States
Oxytocin (OXT) continuous
   University Hospital, Toulouse
      2014   Phase 2/Phase 3   NCT02804373   France
Oxytocin nasal spray
   University Hospital, Toulouse
      2022   Phase 2/Phase 3   NCT05298085   France
Pitolisant oral tablets
   Harmony Biosciences, LLC
      2020   Phase 2   NCT04257929   United States
Pitolisant tablet
   Harmony Biosciences Management, Inc.
      2024   Phase 3   NCT06366464   Australia;United States
Prebiotics with galactomannan and oligofructose
   Children's Hospital of Fudan University
      2023   -   NCT05791604   China
Probiotic
   Fundació Sant Joan de Déu
      2018   -   NCT03548480   Spain
Probiotics including B. lactis B420, B. lactis HN019, B. animalis BB-12, L. rhamnosus GG
   Children's Hospital of Fudan University
      2023   -   NCT05791604   China
R05cb01
   Dutch Growth Research Foundation
      2021   Phase 2;Phase 3   EUCTR2019-000735-61-NL   Netherlands
RAD011
   Radius Pharmaceuticals, Inc.
      2023   Phase 3   NCT05387798   -
      2022   Phase 2/Phase 3   NCT05098509   United States
      2022   Phase 2;Phase 3   EUCTR2021-005331-23-ES   Australia;Canada;France;Italy;Spain;Sweden;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-005331-23-SE   Australia;Belgium;Canada;France;Italy;Spain;Sweden;United Kingdom;United States
Rapamycin
   Peking Union Medical College Hospital
      2021   Phase 0   ChiCTR2000040947   China
Recombinant human growth hormon
   Fundació Parc Taulí
      2017   Phase 4   EUCTR2017-002164-41-ES   Spain
Recombinant human growth hormone
   Fundació Parc Taulí
      2017   Phase 4   EUCTR2017-002164-41-ES   Spain
Recombinant human growth hormone (rhgh) injection
   GeneScience Pharmaceuticals Co., Ltd.
      2018   Phase 3   NCT03554031   China
Recombinant somatropin
   Corporacion Parc Tauli
      2014   -   NCT02205450   Spain
RGH-706
   Chemical Works of Gedeon Richter Plc.
      2022   Phase 2   EUCTR2021-004262-35-IT   Czech Republic;Czechia;France;Italy;Spain;United States
   Gedeon Richter Plc.
      2022   Phase 2   NCT05322096   Czechia;France;Italy;Spain;United States
RGH-706 hydrochloride
   Gedeon Richter Plc.
      2022   Phase 2   EUCTR2021-004262-35-CZ   Czech Republic;Czechia;France;Italy;Spain;United States
Rhgh
   Cai Jinhua
      2021   -   ChiCTR2100046551   China
   Children's Hospital of Zhejiang University School of Medicine
      2019   Phase 4   ChiCTR1900027464   China
Rimonabant
   Weill Medical College of Cornell University
      2007   Phase 3   NCT00603109   United States
RM-493
   Rhythm Pharmaceuticals, Inc.
      2015   Phase 2   NCT02311673   United States
Sandostatin LAR
   University of British Columbia
      2004   Phase 3   NCT00175305   Canada
Saxenda
   Novo Nordisk A/S
      2016   Phase 3   EUCTR2014-004415-37-NL   Australia;Canada;European Union;France;Italy;Netherlands;New Zealand;Turkey;United States
      2015   Phase 3   EUCTR2014-004415-37-IT   Australia;Canada;European Union;France;Italy;Netherlands;New Zealand;Turkey;United States
      -   Phase 3   EUCTR2014-004415-37-FR   Australia;Canada;European Union;France;Italy;Netherlands;New Zealand;Turkey;United States
Setmelanotide
   Rhythm Pharmaceuticals, Inc.
      2025   Phase 2   NCT06772597   United States
Sodium chloride
   University Hospital, Toulouse
      2009   Phase 2   NCT01038570   France
Somatropin
   Corporacion Parc Tauli
      2025   -   NCT06900335   Spain
   Dutch growth research foundation
      2012   -   EUCTR2011-001313-14-NL   Netherlands
   Novo Nordisk A/S
      2011   Phase 1   NCT01401244   United States
      2008   -   NCT00705172   Denmark;Germany;Switzerland
   Oy Eli Lilly Finland Ab
      2007   -   EUCTR2007-000469-39-FI   Finland
Somatropin - adult cohort
   Kawai Norisuke
      2021   Phase 3   JPRN-jRCT2031200351   -
   Pfizer
      2021   Phase 3   NCT04697381   Japan
Somatropin - GH NAïVE pediatric cohort
   Pfizer
      2021   Phase 3   NCT04697381   Japan
Somatropin - GH treated cohort
   Kawai Norisuke
      2021   Phase 3   JPRN-jRCT2031200351   -
   Pfizer
      2021   Phase 3   NCT04697381   Japan
Somatropin FOR injection
   Pfizer, Inc.
      -   Phase 3   EUCTR2024-000101-32-Outside-EU/EEA   Japan
Subcutaneous beloranib IN suspension
   Zafgen Inc.
      2016   Phase 3   EUCTR2015-000660-33-BE   Belgium;Denmark;France;Germany;Italy;Spain;Sweden;United Kingdom
      2015   -   EUCTR2015-000660-33-SE   Belgium;Canada;Denmark;France;Germany;Italy;Spain;Sweden;United Kingdom;United States
      2015   Phase 3   EUCTR2015-000660-33-FR   Belgium;Denmark;France;Germany;Italy;Spain;Sweden;United Kingdom
      2015   Phase 3   EUCTR2015-000660-33-ES   Belgium;Denmark;France;Germany;Italy;Spain;Sweden;United Kingdom
Syntocinon
   Centre Hospitalier de Toulouse
      2014   Phase 3   EUCTR2013-004437-33-FR   France
      -   Phase 2   EUCTR2012-005325-67-FR   France
      -   Phase 2   EUCTR2010-022370-14-FR   France
   University Hospital of Toulouse
      2016   Phase 3   EUCTR2016-003273-18-FR   France
Syntocinon nasal spray
   Dutch Growth Research Foundation
      2017   Phase 2;Phase 3   EUCTR2017-003423-30-NL   Netherlands
      2016   Phase 3   EUCTR2016-003820-22-NL   Netherlands
      2014   Phase 3   EUCTR2013-004134-15-NL   Netherlands
Syntocinon®/- spray
   University Hospital, Toulouse
      2009   Phase 2   NCT01038570   France
Tesofensine
   Saniona A/S
      2017   Phase 2   EUCTR2016-003694-18-CZ   Czech Republic;Hungary
      2016   Phase 2   EUCTR2016-003694-18-HU   Czech Republic;Hungary
Tesofensine citrate
   Saniona A/S
      2022   Phase 2   EUCTR2021-000127-12-SE   Australia;Belgium;France;Ireland;Italy;New Zealand;Spain;Sweden;United Kingdom;United States
Tesofensine/metoprolol
   Saniona
      2017   Phase 2   NCT03149445   Czechia;Hungary
Tesomet
   Saniona
      2021   Phase 2   NCT05198362   United States
   Saniona A/S
      2022   Phase 2   EUCTR2021-000127-12-SE   Australia;Belgium;France;Ireland;Italy;New Zealand;Spain;Sweden;United Kingdom;United States
Tirzepatide
   Grace Kim
      2025   Phase 4   NCT06901245   United States
Topiramate
   ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
      -   Phase 3   EUCTR2011-003432-32-FR   France
   Assistance Publique - Hôpitaux de Paris
      2012   Phase 3   NCT02810483   France
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      2002   -   NCT00065923   United States
ZGN-440
   Zafgen Inc.
      2016   Phase 3   EUCTR2015-000660-33-BE   Belgium;Denmark;France;Germany;Italy;Spain;Sweden;United Kingdom
      2015   -   EUCTR2015-000660-33-SE   Belgium;Canada;Denmark;France;Germany;Italy;Spain;Sweden;United Kingdom;United States
      2015   Phase 3   EUCTR2015-000660-33-FR   Belgium;Denmark;France;Germany;Italy;Spain;Sweden;United Kingdom
      2015   Phase 3   EUCTR2015-000660-33-ES   Belgium;Denmark;France;Germany;Italy;Spain;Sweden;United Kingdom
ZGN-440 FOR injectable suspension
   Zafgen, Inc.
      2014   Phase 3   NCT02179151   United States
ZGN-440 sterile diluent
   Zafgen, Inc.
      2013   Phase 2   NCT01818921   United States