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

Clinical trials : 59 /  Drugs : 76 - (DrugBank : 12) / Drug target genes : 13 - Drug target pathways : 24

Drugs, Primary sponsors and Trial info (Enrollment year, Phase, Trial ID, Countries)
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
Bioimpedenziometry, handgrip strenght evaluation
   University of Bari
      2019   -   NCT04786522   Italy
Blood sample
   Hospices Civils de Lyon
      2026   -   NCT07038239   France
   University Hospital, Angers
      2022   -   NCT04881201   France
Blood samples analysis
   Assistance Publique Hopitaux De Marseille
      2016   -   NCT02596191   France
Blood test
   Assistance Publique Hopitaux De Marseille
      2025   -   NCT07049588   France
Blood test FOR PRO-inflammatory cytokines and ultrasound OF THE median nerves
   Centre Hospitalier Universitaire de Nice
      2022   -   NCT06808581   France
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
Clinical evaluation
   Assistance Publique Hopitaux De Marseille
      2016   -   NCT02596191   France
Clinical scores
   Assistance Publique Hopitaux De Marseille
      2025   -   NCT07049588   France
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
EDK060, dose A
   Novartis Pharmaceuticals
      2025   Phase 1   NCT07140614   Canada
EDK060, dose B
   Novartis Pharmaceuticals
      2025   Phase 1   NCT07140614   Canada
EDK060, dose C
   Novartis Pharmaceuticals
      2025   Phase 1   NCT07140614   Canada
EDK060, dose D
   Novartis Pharmaceuticals
      2025   Phase 1   NCT07140614   Canada
Electrophysiological record
   Assistance Publique Hopitaux De Marseille
      2016   -   NCT02596191   France
Elleone
   HÔPITAUX UNIVERSITAIRES DE STRASBOURG
      2015   Phase 2   EUCTR2015-001716-36-FR   France
ELP-02
   Elpida Therapeutics SPC
      2026   Phase 1/Phase 2   NCT07447557   -
EN001
   ENCell
      2024   Phase 1/Phase 2   NCT06328712   South Korea
      2022   Phase 1   NCT05333406   Korea, Republic of
Epalrestat
   The Third Xiangya Hospital of Central South University
      2023   Phase 2   NCT05777226   -
Evaluation ON blood specimens
   University of Bari
      2019   -   NCT04786522   Italy
Govorestat
   Applied Therapeutics, Inc.
      2026   Phase 3   NCT07191912   Australia;France;Germany;Italy;Spain;Turkey (Türkiye);United States
Human wharton'S jelly-derived mesenchymal stem cells
   Samsung Medical Center
      2024   -   NCT06218134   South Korea
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
Muscle MRI
   Assistance Publique Hopitaux De Marseille
      2016   -   NCT02596191   France
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
NL-GARS1-001
   n-Lorem Foundation
      2025   Phase 1/Phase 2   NCT07226297   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
NT3 blood draw
   Zarife Sahenk
      2027   -   NCT05011006   United States
Patient report outcomes measures
   Assistance Publique Hopitaux De Marseille
      2025   -   NCT07049588   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
Quantitative neuromuscular MRI
   Assistance Publique Hopitaux De Marseille
      2025   -   NCT07049588   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
Skin biopsy
   Assistance Publique Hopitaux De Marseille
      2025   -   NCT07049588   France
   Hospices Civils de Lyon
      2026   -   NCT07038239   France
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
VCA-894A
   Vanda Pharmaceuticals
      2025   Phase 1/Phase 2   NCT07223632   United States