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 -
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 -