10. シャルコー・マリー・トゥース病 Disease details / Clinical trials / Drug dev / DR info

臨床試験数 : 45 / 薬物数 : 34 - (DrugBank : 10) / 標的遺伝子数 : 12 - 標的パスウェイ数 : 22

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）
ACE-083
   Acceleron Pharma, Inc.
      2019   Phase 2   NCT03943290   Canada;Spain;United States
      2017   Phase 2   NCT03124459   United States
Acido ascorbico
   ISTITUTO NEUROLOGICO CARLO BESTA
      2005   -   EUCTR2006-000032-27-IT   Italy
      2004   -   EUCTR2004-004501-24-IT   Italy
Ascorbic acid
   Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
      2006   Phase 2   NCT00271635   Netherlands
   Chiba University
      2019   Phase 1   JPRN-UMIN000036332   Japan
   ISTITUTO NEUROLOGICO CARLO BESTA
      2004   -   EUCTR2004-004501-24-IT   Italy
   New treatment strategies for intractable neuropathies based on its pathomechanism. The Research Grant 19A-5 for Nervous and Mental Disorders from the Ministry of Health, Labour and Welfare.
      2005   Phase 1,2   JPRN-UMIN000001535   Japan
   SHIOHAMA TADASHI
      2019   Phase 2   JPRN-jRCTs031190109   -
   Wayne State University
      2007   Phase 2/Phase 3   NCT00484510   United States
Baclofen
   Pharnext
      2017   Phase 3   EUCTR2015-002379-81-FR   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-ES   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-DE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2010   -   EUCTR2010-023097-40-FR   France
   Pharnext S.C.A.
      2021   Phase 3   NCT04762758   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
   Pharnext SA
      2021   Phase 3   EUCTR2020-004805-30-NL   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-FR   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-ES   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2018   Phase 3   EUCTR2015-002379-81-NL   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-GB   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-BE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext SCA
      2022   Phase 3   EUCTR2020-004805-30-DK   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-BE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      -   Phase 3   EUCTR2020-004805-30-DE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
Baclofene
   Pharnext SA
      2021   Phase 3   EUCTR2020-004805-30-IT   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
Cebion 500*20CPR mast 500MG
   ISTITUTO NEUROLOGICO CARLO BESTA
      2004   -   EUCTR2004-004501-24-IT   Italy
Cebion 500MG 20CPR mast.aran
   ISTITUTO NEUROLOGICO CARLO BESTA
      2005   -   EUCTR2006-000032-27-IT   Italy
CLZ-2002
   Cellatoz Therapeutics, Inc
      2023   Phase 1   NCT05947578   Korea, Republic of
Coenzyme Q10
   Memorial Medical Center
      2007   Phase 1/Phase 2   NCT00541164   United States
D-biotin
   MEDDAY PHARMACEUTICALS
      2016   Phase 2   EUCTR2015-001150-15-FR   France
D-sorbitol
   PHARNEXT
      2016   Phase 3   EUCTR2015-002378-19-DE   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   EUCTR2015-002378-19-FR   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   EUCTR2015-002378-19-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2015-002378-19-GB   Belgium;France;Germany;Netherlands;United Kingdom;United States
   PHARNEXT SA
      2016   Phase 3   EUCTR2015-002378-19-NL   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
      2016   Phase 3   EUCTR2015-002378-19-BE   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext
      2017   Phase 3   EUCTR2015-002379-81-FR   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-ES   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-DE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext S.C.A.
      2021   Phase 3   NCT04762758   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
   Pharnext SA
      2021   Phase 3   EUCTR2020-004805-30-NL   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-IT   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-FR   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-ES   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2018   Phase 3   EUCTR2015-002379-81-NL   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-GB   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-BE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext SCA
      2022   Phase 3   EUCTR2020-004805-30-DK   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-BE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      -   Phase 3   EUCTR2020-004805-30-DE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
Elleone
   HÔPITAUX UNIVERSITAIRES DE STRASBOURG
      2015   Phase 2   EUCTR2015-001716-36-FR   France
EN001
   ENCell
      2024   Phase 1   NCT06328712   Korea, Republic of
      2022   Phase 1   NCT05333406   Korea, Republic of
Engensis
   Helixmith Co., Ltd.
      2020   Phase 1/Phase 2   NCT05361031   Korea, Republic of
Epalrestat
   The Third Xiangya Hospital of Central South University
      2023   Phase 2   NCT05777226   -
MD1003
   MEDDAY PHARMACEUTICALS
      2016   Phase 2   EUCTR2015-001150-15-FR   France
   MedDay Pharmaceuticals SA
      2016   Phase 2   NCT02967679   France
Mexiletine
   University of Rochester
      2015   Phase 2   NCT02561702   United States
Naltrexone
   Pharnext
      2010   -   EUCTR2010-023097-40-FR   France
Naltrexone cloridrato
   Pharnext SA
      2021   Phase 3   EUCTR2020-004805-30-IT   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
Naltrexone hydrochloride
   PHARNEXT
      2016   Phase 3   EUCTR2015-002378-19-DE   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   EUCTR2015-002378-19-FR   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   EUCTR2015-002378-19-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2015-002378-19-GB   Belgium;France;Germany;Netherlands;United Kingdom;United States
   PHARNEXT SA
      2016   Phase 3   EUCTR2015-002378-19-NL   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
      2016   Phase 3   EUCTR2015-002378-19-BE   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext
      2017   Phase 3   EUCTR2015-002379-81-FR   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-ES   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-DE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext S.C.A.
      2021   Phase 3   NCT04762758   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
   Pharnext SA
      2021   Phase 3   EUCTR2020-004805-30-NL   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-IT   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-FR   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-ES   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2018   Phase 3   EUCTR2015-002379-81-NL   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-GB   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-BE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext SCA
      2022   Phase 3   EUCTR2020-004805-30-DK   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-BE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      -   Phase 3   EUCTR2020-004805-30-DE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
NMD670
   NMD Pharma A/S
      2024   Phase 2   NCT06482437   Belgium;Denmark;France;Spain;United States
NON applicable
   Pharnext
      2010   -   EUCTR2010-023097-40-FR   France
Posturological examination ON kplates and amti platforms
   Assistance Publique - Hôpitaux de Paris
      2021   -   NCT04283175   France
PXT3003
   Pharnext
      2010   -   EUCTR2010-023097-40-FR   France
   Pharnext S.C.A.
      2017   Phase 3   NCT03023540   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext SA
      2021   Phase 3   EUCTR2020-004805-30-NL   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-IT   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-FR   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-ES   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
   Pharnext SCA
      2022   Phase 3   EUCTR2020-004805-30-DK   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-004805-30-BE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
      -   Phase 3   EUCTR2020-004805-30-DE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
PXT3003 dose 1
   PHARNEXT
      2016   Phase 3   EUCTR2015-002378-19-DE   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   EUCTR2015-002378-19-FR   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   EUCTR2015-002378-19-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2015-002378-19-GB   Belgium;France;Germany;Netherlands;United Kingdom;United States
   PHARNEXT SA
      2016   Phase 3   EUCTR2015-002378-19-NL   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
      2016   Phase 3   EUCTR2015-002378-19-BE   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext
      2017   Phase 3   EUCTR2015-002379-81-FR   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-ES   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext SA
      2018   Phase 3   EUCTR2015-002379-81-NL   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-GB   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-BE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   NCT02579759   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
PXT3003 dose 2
   PHARNEXT
      2016   Phase 3   EUCTR2015-002378-19-DE   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   EUCTR2015-002378-19-FR   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   EUCTR2015-002378-19-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2015-002378-19-GB   Belgium;France;Germany;Netherlands;United Kingdom;United States
   PHARNEXT SA
      2016   Phase 3   EUCTR2015-002378-19-NL   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
      2016   Phase 3   EUCTR2015-002378-19-BE   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext
      2017   Phase 3   EUCTR2015-002379-81-FR   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-ES   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-DE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
   Pharnext SA
      2018   Phase 3   EUCTR2015-002379-81-NL   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-GB   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2017   Phase 3   EUCTR2015-002379-81-BE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   NCT02579759   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States
PXT3003 high dose
   Pharnext SA
      2010   Phase 2   NCT01401257   France
PXT3003 intermediate dose
   Pharnext SA
      2010   Phase 2   NCT01401257   France
PXT3003 LOW dose
   Pharnext SA
      2010   Phase 2   NCT01401257   France
RS-baclofen
   PHARNEXT
      2016   Phase 3   EUCTR2015-002378-19-DE   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   EUCTR2015-002378-19-FR   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      2015   Phase 3   EUCTR2015-002378-19-ES   Belgium;France;Germany;Netherlands;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2015-002378-19-GB   Belgium;France;Germany;Netherlands;United Kingdom;United States
   PHARNEXT SA
      2016   Phase 3   EUCTR2015-002378-19-NL   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
      2016   Phase 3   EUCTR2015-002378-19-BE   Belgium;Germany;Netherlands;Spain;United Kingdom;United States
Sorbitol
   Pharnext
      2010   -   EUCTR2010-023097-40-FR   France
Sorbitolo
   Pharnext SA
      2021   Phase 3   EUCTR2020-004805-30-IT   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States
Ulipristal acetate
   HÔPITAUX UNIVERSITAIRES DE STRASBOURG
      2015   Phase 2   EUCTR2015-001716-36-FR   France
Vitamin C
   Wayne State University
      2007   Phase 2/Phase 3   NCT00484510   United States