10. Charcot-Marie-Tooth disease
31 clinical trials, 38 drugs (DrugBank: 9 drugs), 11 drug target genes, 14 drug target pathways
Searched query = "Charcot-Marie-Tooth disease", "CMT"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2015-002379-81-NL (EUCTR) | 08/01/2018 | 28/03/2017 | An International, multi-center, 9-month FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | International, multi-center, open-label 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | Charcot-Marie-Tooth Disease type 1A MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: PXT3003 Dose 1 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL Product Code: PXT3003 Dose 2 (equivalent to twice Dose 1) INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL | Pharnext SA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 290 | Phase 3 | France;United States;Canada;Belgium;Spain;Germany;Netherlands;United Kingdom | ||
| 2 | EUCTR2015-002379-81-DE (EUCTR) | 15/08/2017 | 19/01/2017 | An International, multi-center, 9-month FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | International, multi-center, open-label 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | Charcot-Marie-Tooth Disease type 1A MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: PXT3003 Dose 2 (equivalent to twice Dose 1) INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL Product Code: PXT3003 Dose 2 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL | Pharnext | NULL | Not Recruiting | Female: yes Male: yes | 290 | Phase 3 | France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom | ||
| 3 | EUCTR2015-002379-81-GB (EUCTR) | 28/07/2017 | 12/06/2017 | An International, multi-center, FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | International, multi-center, open-label FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | Charcot-Marie-Tooth Disease type 1A MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: PXT3003 Dose 1 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL Product Code: PXT3003 Dose 2 (equivalent to twice Dose 1) INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL | Pharnext SA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 187 | Phase 3 | United States;France;Canada;Spain;Belgium;Germany;Netherlands;United Kingdom | ||
| 4 | EUCTR2015-002379-81-ES (EUCTR) | 25/05/2017 | 07/04/2017 | An International, multi-center, 9-month FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | International, multi-center, double blind 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | Charcot-Marie-Tooth Disease type 1A MedDRA version: 19.1;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: PXT3003 Dose 1 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL Product Code: PXT3003 Dose 2 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL | Pharnext | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 300 | Phase 3 | France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom | ||
| 5 | EUCTR2015-002379-81-BE (EUCTR) | 04/05/2017 | 21/02/2017 | An International, multi-center, 9-month FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | International, multi-center, double blind 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | Charcot-Marie-Tooth Disease type 1A MedDRA version: 19.1;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: PXT3003 Dose 1 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL Product Code: PXT3003 Dose 2 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL | Pharnext | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 300 | Phase 3 | France;United States;Canada;Spain;Belgium;Netherlands;Germany;United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03023540 (ClinicalTrials.gov) | March 7, 2017 | 26/12/2016 | Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A | International, Multi-center, Open Label, 9-month Follow-up Extension Study Assessing the Long-term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A | Charcot-Marie-Tooth Disease, Type IA | Drug: PXT3003 | Pharnext SA | SynteractHCR;Syneos Health;Premier Research Group plc | Active, not recruiting | 16 Years | 67 Years | All | 187 | Phase 3 | United States;Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom |
| 7 | EUCTR2015-002379-81-FR (EUCTR) | 26/01/2017 | 12/01/2017 | An International, multi-center, 9-month FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | International, multi-center, double blind 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A. | Charcot-Marie-Tooth Disease type 1A MedDRA version: 19.1;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: PXT3003 Dose 1 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL Product Code: PXT3003 Dose 2 INN or Proposed INN: BACLOFEN Other descriptive name: NA INN or Proposed INN: NALTREXONE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL | Pharnext | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 300 | Phase 3 | United States;France;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom | ||
| 8 | EUCTR2015-002378-19-NL (EUCTR) | 21/04/2016 | 17/12/2015 | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months - PLEO-CMT | Charcot-Marie-Tooth Disease - Type 1A MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: PXT3003 dose 1 INN or Proposed INN: RS-BACLOFEN INN or Proposed INN: NALTREXONE HYDROCHLORIDE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: D-SORBITOL Other descriptive name: D-SORBITOL Product Code: PXT3003 dose 2 INN or Proposed INN: RS-BACLOFEN INN or Proposed INN: NALTREXONE HYDROCHLORIDE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: D-SORBITOL Other descriptive name: D-SORBITOL | PHARNEXT SA | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 3 | United States;Belgium;Spain;Germany;Netherlands;United Kingdom | ||
| 9 | EUCTR2015-002378-19-DE (EUCTR) | 11/02/2016 | 28/09/2015 | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months - PLEO-CMT | Charcot-Marie-Tooth Disease - Type 1A MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: PXT3003 dose 1 INN or Proposed INN: RS-BACLOFEN INN or Proposed INN: NALTREXONE HYDROCHLORIDE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: D-SORBITOL Other descriptive name: D-SORBITOL Product Code: PXT3003 dose 2 INN or Proposed INN: RS-BACLOFEN INN or Proposed INN: NALTREXONE HYDROCHLORIDE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: D-SORBITOL Other descriptive name: D-SORBITOL | PHARNEXT | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 3 | United States;Belgium;Spain;Netherlands;Germany;United Kingdom | ||
| 10 | EUCTR2015-002378-19-BE (EUCTR) | 18/01/2016 | 19/11/2015 | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months - PLEO-CMT | Charcot-Marie-Tooth Disease - Type 1A MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: PXT3003 dose 1 INN or Proposed INN: RS-BACLOFEN INN or Proposed INN: NALTREXONE HYDROCHLORIDE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: D-SORBITOL Other descriptive name: D-SORBITOL Product Code: PXT3003 dose 2 INN or Proposed INN: RS-BACLOFEN INN or Proposed INN: NALTREXONE HYDROCHLORIDE Other descriptive name: NALTREXONE HYDROCHLORIDE INN or Proposed INN: D-SORBITOL Other descriptive name: D-SORBITOL | PHARNEXT SA | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 3 | United States;Spain;Belgium;Netherlands;Germany;United Kingdom | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2015-002378-19-ES (EUCTR) | 28/12/2015 | 30/10/2015 | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months - PLEO-CMT | Charcot-Marie-Tooth Disease - Type 1A MedDRA version: 18.1;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | PHARNEXT | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 3 | France;United States;Belgium;Spain;Netherlands;Germany;United Kingdom | |||
| 12 | NCT02579759 (ClinicalTrials.gov) | December 2015 | 28/9/2015 | Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT) | International, Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study Assessing in Parallel Groups the Efficacy and Safety of 2 Doses of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A Treated 15 Months | Charcot-Marie-Tooth Disease Type 1A | Drug: PXT3003 dose 1;Drug: PXT3003 dose 2;Drug: placebo | Pharnext SA | NULL | Completed | 16 Years | 65 Years | All | 323 | Phase 3 | United States;Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom |
| 13 | EUCTR2015-002378-19-FR (EUCTR) | 27/08/2015 | 27/07/2015 | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months - PLEO-CMT | Charcot-Marie-Tooth Disease - Type 1A MedDRA version: 18.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | PHARNEXT | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 3 | United States;France;Belgium;Spain;Netherlands;Germany;United Kingdom | |||
| 14 | NCT01401257 (ClinicalTrials.gov) | December 2010 | 20/7/2011 | Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A | A Phase II, Randomized, Placebo-controlled Trial of the Safety, Efficacy, Pharmacodynamics and Pharmacokinetics of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A. | Charcot-Marie-Tooth Disease;Hereditary Neuropathy With Liability to Pressure Palsies;Genetic Disorders | Drug: PXT3003 Low dose;Drug: PXT3003 Intermediate Dose;Drug: PXT3003 High Dose;Other: Placebo | Pharnext SA | NULL | Completed | 18 Years | 65 Years | All | 80 | Phase 2 | France |
| 15 | EUCTR2010-023097-40-FR (EUCTR) | 29/11/2010 | 04/10/2010 | A Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX3003 in Patients with Charcot-Marie-Tooth Disease Type 1A - non applicable | A Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX3003 in Patients with Charcot-Marie-Tooth Disease Type 1A - non applicable | Charcot-Marie-Tooth disease (type 1A). MedDRA version: 12.1;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease | Product Name: non applicable Product Code: PXT3003 INN or Proposed INN: Baclofen INN or Proposed INN: Naltrexone INN or Proposed INN: Sorbitol Product Name: non applicable Product Code: PXT3003 INN or Proposed INN: Baclofen INN or Proposed INN: Naltrexone INN or Proposed INN: Sorbitol Product Name: non applicable Product Code: PXT3003 INN or Proposed INN: Baclofen INN or Proposed INN: Naltrexone INN or Proposed INN: Sorbitol | Pharnext | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 80 | Phase 2 | France | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | EUCTR2015-002378-19-GB (EUCTR) | 16/12/2015 | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months | International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months - PLEO-CMT | Charcot-Marie-Tooth Disease - Type 1A MedDRA version: 20.0;Level: LLT;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | PHARNEXT | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 3 | France;United States;Belgium;Netherlands;Germany;United Kingdom |