156. レット症候群 Disease details / Clinical trials / Drug dev / DR info


臨床試験数 : 55 / 薬物数 : 51 - (DrugBank : 22) / 標的遺伝子数 : 77 - 標的パスウェイ数 : 116

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)
Administration OF A high dose OF desipramine
   Assistance Publique Hopitaux De Marseille
      2009   Phase 2   NCT00990691   France
Administration OF A LOW dose OF desipramine
   Assistance Publique Hopitaux De Marseille
      2009   Phase 2   NCT00990691   France
Alac, inulin, FOS, and sodium butyrate
   Azienda Ospedaliera Universitaria Senese
      2022   -   NCT05420805   Italy
Alpha-tocotrienol quinone
   EDISON PHARMACEUTICALS INC.
      2013   -   EUCTR2012-005021-76-IT   Italy
Anavex2-73
   Anavex Germany GmbH
      2020   Phase 2   EUCTR2019-004345-32-GB   Australia;United Kingdom
   Anavex Life Sciences Corp.
      2019   Phase 3   NCT03941444   Australia;United Kingdom
      2019   Phase 2   NCT03758924   United States
Anavex2-73 oral liquid
   Anavex Life Sciences Corp.
      2020   Phase 2/Phase 3   NCT04304482   Australia;Canada;United Kingdom
Bionetide
   Biomed Industries, Inc.
      2025   Phase 3   NCT06840496   Australia;United States
      2024   Phase 2/Phase 3   NCT06849973   Australia;United States
Blarcamesine
   Anavex Germany GmbH
      2020   Phase 2   EUCTR2019-004345-32-GB   Australia;United Kingdom
Calcium
   Baylor College of Medicine
      2008   -   NCT05352373   -
Cannabidiol
   GW RESEARCH LTD.
      2020   Phase 3   EUCTR2019-001605-24-IT   Australia;Canada;Italy;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003370-27-IT   Canada;Germany;Italy;Spain;United Kingdom;United States
   GW Research Ltd
      2020   Phase 3   EUCTR2019-001605-24-GB   Australia;Canada;France;Italy;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2019-001605-24-ES   Australia;Canada;Italy;Spain;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-003370-27-GB   Australia;Canada;France;Italy;Spain;United Kingdom;United States
Cannabidiolo
   GW RESEARCH LTD.
      2020   Phase 3   EUCTR2019-001605-24-IT   Australia;Canada;Italy;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003370-27-IT   Canada;Germany;Italy;Spain;United Kingdom;United States
CBD
   GW Research Ltd
      2020   Phase 3   EUCTR2019-001605-24-ES   Australia;Canada;Italy;Spain;United Kingdom;United States
CBD - oral solution, IS known AS epidyolex, and IS THE approved name
   GW Research Ltd
      2019   Phase 2;Phase 3   EUCTR2018-003370-27-GB   Australia;Canada;France;Italy;Spain;United Kingdom;United States
CBD - solution orale, conosciuto come epidiolex, questo è IL nome approvato negli stati uniti
   GW RESEARCH LTD.
      2019   Phase 3   EUCTR2018-003370-27-IT   Canada;Germany;Italy;Spain;United Kingdom;United States
CBD oral solution, IS known AS epidyolex and IS THE approved name IN THE EU
   GW Research Ltd
      2020   Phase 3   EUCTR2019-001605-24-GB   Australia;Canada;France;Italy;Spain;United Kingdom;United States
CBD soluzione orale nota come epidyolex E CON tale nome è stato approvato IN UE
   GW RESEARCH LTD.
      2020   Phase 3   EUCTR2019-001605-24-IT   Australia;Canada;Italy;Spain;United Kingdom;United States
Creatine monohydrate
   Medical University of Vienna
      2005   -   NCT01147575   Austria
Dextromethorphan
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      2004   Phase 3   NCT00069550   United States
      1995   -   NCT00004807   -
   Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
      2012   Phase 2   NCT01520363   United States
      2004   Phase 2   NCT00593957   United States
   Kennedy Krieger Inst. Johns Hopkins Medicine
      -   Phase 2   EUCTR2008-005571-10-NO   Norway
Donepezil
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      1995   -   NCT00004807   -
Donepezil hydrochloride
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      2004   Phase 3   NCT00069550   United States
EMD 128130
   NEWRON PHARMACEUTICALS SPA
      -   Phase 2   EUCTR2015-004448-20-IT   Australia;India;Italy;United Kingdom;United States
   Newron Pharmaceuticals S.p.A.
      2016   Phase 2   EUCTR2015-004448-20-GB   Australia;India;Italy;United Kingdom;United States
EPI-743
   EDISON PHARMACEUTICALS INC.
      2013   -   EUCTR2012-005021-76-IT   Italy
   Edison Pharmaceuticals Inc
      2013   Phase 2   NCT01822249   Italy
Epidyolex and IS THE approved name IN THE EU
   GW Research Ltd
      2020   Phase 3   EUCTR2019-001605-24-ES   Australia;Canada;Italy;Spain;United Kingdom;United States
Esketamine hydrochloride
   Children's Hospital of Fudan University
      2023   Early Phase 1   NCT06199700   China
Fingolimod
   University Hospital, Basel, Switzerland
      2013   Phase 1/Phase 2   NCT02061137   Switzerland
GCB-002
   Genecombio Ltd.
      2024   -   NCT06739434   China
Glatiramer acetate
   Montefiore Medical Center
      2013   Phase 2   NCT02153723   United States
   Sheba Medical Center
      2014   Phase 1   NCT02023424   Israel
Growth hormone
   The first hospital of JiLin University
      2024   -   ChiCTR2400089886   China
GWP42003-P
   GW RESEARCH LTD.
      2020   Phase 3   EUCTR2019-001605-24-IT   Australia;Canada;Italy;Spain;United Kingdom;United States
      2019   Phase 3   EUCTR2018-003370-27-IT   Canada;Germany;Italy;Spain;United Kingdom;United States
   GW Research Ltd
      2020   Phase 3   NCT04252586   Australia;Canada;Italy;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2019-001605-24-GB   Australia;Canada;France;Italy;Spain;United Kingdom;United States
      2020   Phase 3   EUCTR2019-001605-24-ES   Australia;Canada;Italy;Spain;United Kingdom;United States
      2019   Phase 2;Phase 3   EUCTR2018-003370-27-GB   Australia;Canada;France;Italy;Spain;United Kingdom;United States
   Jazz Pharmaceuticals
      2019   Phase 3   NCT03848832   Italy;Spain;United Kingdom;United States
Human insulin
   Boston Children's Hospital
      2013   Phase 2   NCT01777542   United States
Intrathecal injection OF AAV-MECP2 FOR THE treatment OF rett syndrome
   Guangzhou Women and Children's Medical Center
      2025   Phase 1/Phase 2   NCT06856759   China
Ketamine
   Rett Syndrome Research Trust
      2019   Phase 2   NCT03633058   United States
Lovastatin
   Montefiore Medical Center
      2015   Phase 2   NCT02563860   United States
Male rett
   Children's Hospital Colorado
      2023   -   NCT06346106   United States
NGN-401
   Neurogene Inc.
      2023   Phase 1/Phase 2   NCT05898620   Australia;United Kingdom;United States
NNZ-2566
   Neuren Pharmaceuticals Limited
      2016   Phase 2   NCT02715115   United States
      2013   Phase 2   NCT01703533   United States
NTI164
   Fenix Innovation Group
      2023   Phase 1/Phase 2   NCT06621043   Australia
Probiotic-lactobacillus plantarum PS128
   National Taiwan University Hospital
      2017   -   NCT03259971   Taiwan
RETT-T
   Anagnostou, Evdokia, M.D.
      2025   Phase 2   NCT04041713   Canada
Rhigf-1
   Boston Children's Hospital
      2013   Phase 2   NCT01777542   United States
   Boston Children’s Hospital
      2010   Phase 1   NCT01253317   United States
Risperidone
   Janssen-Ortho Inc., Canada
      1999   Phase 3   NCT00261508   -
Sarizotan
   Newron Pharmaceuticals SPA
      2016   Phase 2/Phase 3   NCT02790034   Australia;India;Italy;United Kingdom;United States
Sarizotan cloridrato
   NEWRON PHARMACEUTICALS SPA
      -   Phase 2   EUCTR2015-004448-20-IT   Australia;India;Italy;United Kingdom;United States
Sarizotan hydrochloride
   NEWRON PHARMACEUTICALS SPA
      -   Phase 2   EUCTR2015-004448-20-IT   Australia;India;Italy;United Kingdom;United States
   Newron Pharmaceuticals S.p.A.
      2016   Phase 2   EUCTR2015-004448-20-GB   Australia;India;Italy;United Kingdom;United States
Sodium butyrate and zinc oxide
   Azienda Ospedaliera Universitaria Senese
      2022   -   NCT05420805   Italy
Topiramate
   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
      1995   -   NCT00004807   -
Tridecanoic acid
   Sheba Medical Center
      2017   Phase 2   NCT03059160   -
Triheptanoin
   Center for Rare Neurological Diseases, Norcross, GA
      2016   Phase 2   NCT02696044   United States
Trofinetide
   ACADIA Pharmaceuticals Inc.
      2021   Phase 2/Phase 3   NCT04988867   United States
      2020   Phase 3   NCT04776746   United States
      2020   Phase 3   NCT04279314   United States
      2019   Phase 3   NCT04181723   United States
TSHA-102
   Taysha Gene Therapies, Inc.
      2023   Phase 1/Phase 2   NCT06152237   Canada;United Kingdom;United States
      2023   Phase 1/Phase 2   NCT05606614   Canada;United States
VYNT-0126
   Vyant Bio
      2023   Phase 2   NCT05625568   -