10. Charcot-Marie-Tooth disease Disease details / Clinical trials / Drug dev / DR info

Clinical trials : 41 /  Drugs : 46 - (DrugBank : 9) / Drug target genes : 11 - Drug target pathways : 15

Drugs and their primary sponsors and trial info
(RS)-baclofen   
   Pharnext SA
      2022   Phase 3   EUCTR2020-004805-30-DK   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
      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;
      -   Phase 3   EUCTR2020-004805-30-DE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
      -   Phase 3   EUCTR2020-004805-30-BE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
(RS)-baclofen, naltrexone hydrochloride and D-sorbitol   
   Pharnext SA
      2021   Phase 3   NCT04762758   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
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
      2004   -   EUCTR2004-004501-24-IT   Italy;
ACIDO ASCORBICO DC.IT   
   ISTITUTO NEUROLOGICO CARLO BESTA
      2005   -   EUCTR2006-000032-27-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;
   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   Japan;
Ascorbic acid (Vitamin C)   
   Wayne State University
      2007   Phase 2/Phase 3   NCT00484510   United States;
Ascorbic acid vit C   
   ISTITUTO NEUROLOGICO CARLO BESTA
      2004   -   EUCTR2004-004501-24-IT   Italy;
BACLOFENE   
   Pharnext SA
      2021   Phase 3   EUCTR2020-004805-30-IT   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
Baclofen   
   Pharnext
      2010   -   EUCTR2010-023097-40-FR   France;
CEBION 500*20CPR MAST   
   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;
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;
      2017   Phase 3   EUCTR2015-002379-81-BE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States;
   Pharnext SA
      2022   Phase 3   EUCTR2020-004805-30-DK   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
      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;
      -   Phase 3   EUCTR2020-004805-30-DE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
      -   Phase 3   EUCTR2020-004805-30-BE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
ElleOne   
   HÔPITAUX UNIVERSITAIRES DE STRASBOURG
      2015   Phase 2   EUCTR2015-001716-36-FR   France;
FLX-787-ODT (orally disintegrating tablet)   
   Flex Pharma, Inc.
      2017   Phase 2   NCT03254199   United States;
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 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;
      2017   Phase 3   EUCTR2015-002379-81-BE   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;
Naltrexone   
   Pharnext
      2010   -   EUCTR2010-023097-40-FR   France;
Naltrexone hydrochloride   
   Pharnext SA
      2022   Phase 3   EUCTR2020-004805-30-DK   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
      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;
      -   Phase 3   EUCTR2020-004805-30-DE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
      -   Phase 3   EUCTR2020-004805-30-BE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
Non applicable   
   Pharnext
      2010   -   EUCTR2010-023097-40-FR   France;
PXT3003   
   Pharnext
      2010   -   EUCTR2010-023097-40-FR   France;
   Pharnext SA
      2022   Phase 3   EUCTR2020-004805-30-DK   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
      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;
      2017   Phase 3   NCT03023540   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States;
      -   Phase 3   EUCTR2020-004805-30-DE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
      -   Phase 3   EUCTR2020-004805-30-BE   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
PXT3003 Dose 1   
   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-BE   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;
PXT3003 Dose 2   
   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-BE   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States;
PXT3003 Dose 2 (equivalent to twice Dose 1)   
   Pharnext
      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;
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;
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 SA
      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 SA
      2015   Phase 3   NCT02579759   Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom;United States;
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;
SODIO ASCORBATO DC.IT   
   ISTITUTO NEUROLOGICO CARLO BESTA
      2005   -   EUCTR2006-000032-27-IT   Italy;
SORBITOLO   
   Pharnext SA
      2021   Phase 3   EUCTR2020-004805-30-IT   Belgium;Canada;Denmark;France;Germany;Israel;Italy;Netherlands;Spain;United States;
Sorbitol   
   Pharnext
      2010   -   EUCTR2010-023097-40-FR   France;
ULIPRISTAL ACETATE   
   HÔPITAUX UNIVERSITAIRES DE STRASBOURG
      2015   Phase 2   EUCTR2015-001716-36-FR   France;