10. Charcot-Marie-Tooth disease Clinical trials / Disease details
Clinical trials : 41 / Drugs : 46 - (DrugBank : 9) / Drug target genes : 11 - Drug target pathways : 15
| 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 | NCT05333406 (ClinicalTrials.gov) | February 22, 2022 | 6/1/2022 | Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A | Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A | Charcot-Marie-Tooth Disease, Type IA | Drug: EN001 | ENCell | NULL | Completed | 19 Years | 75 Years | All | 9 | Phase 1 | Korea, Republic of |
| 2 | EUCTR2020-004805-30-DK (EUCTR) | 31/01/2022 | 08/06/2021 | Phase 3 trial of PXT3003 in Charcot-Marie-Tooth (CMT) Type 1A patients | A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A) | Charcot Marie Tooth 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 Name: NA Product Code: PXT3003 INN or Proposed INN: BACLOFEN Other descriptive name: (RS)-baclofen 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 | 350 | Phase 3 | France;United States;Canada;Spain;Belgium;Denmark;Israel;Netherlands;Germany;Italy | ||
| 3 | EUCTR2020-004805-30-NL (EUCTR) | 23/09/2021 | 27/05/2021 | Phase 3 trial of PXT3003 in Charcot-Marie-Tooth (CMT) Type 1A patients | A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A) | Charcot Marie Tooth 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 Name: NA Product Code: PXT3003 INN or Proposed INN: BACLOFEN Other descriptive name: (RS)-baclofen 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 | 350 | Phase 3 | France;United States;Canada;Spain;Belgium;Denmark;Israel;Germany;Netherlands;Italy | ||
| 4 | EUCTR2020-004805-30-IT (EUCTR) | 19/05/2021 | 02/08/2021 | Phase 3 trial of PXT3003 in Charcot-Marie-Tooth (CMT) Type 1A patients | A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A) - na | Charcot Marie Tooth 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 Name: PXT3003 Product Code: [PXT3003] INN or Proposed INN: BACLOFENE INN or Proposed INN: NALTREXONE CLORIDRATO Other descriptive name: naltrexone hydrochloride INN or Proposed INN: SORBITOLO Other descriptive name: D-SORBITOL | Pharnext SA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 350 | Phase 3 | France;United States;Canada;Spain;Belgium;Denmark;Israel;Netherlands;Germany;Italy | ||
| 5 | EUCTR2020-004805-30-ES (EUCTR) | 04/05/2021 | 30/07/2021 | Phase 3 trial of PXT3003 in Charcot-Marie-Tooth (CMT) Type 1A patients | A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A) | Charcot Marie Tooth 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 Name: NA Product Code: PXT3003 INN or Proposed INN: BACLOFEN Other descriptive name: (RS)-baclofen 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 | 350 | Phase 3 | France;United States;Canada;Belgium;Spain;Denmark;Israel;Netherlands;Germany;Italy | ||
| 6 | EUCTR2020-004805-30-FR (EUCTR) | 04/05/2021 | 19/01/2022 | Phase 3 trial of PXT3003 in Charcot-Marie-Tooth (CMT) Type 1A patients | A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A) | Charcot Marie Tooth 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 Name: NA Product Code: PXT3003 INN or Proposed INN: BACLOFEN Other descriptive name: (RS)-baclofen 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 | 350 | Phase 3 | United States;France;Canada;Spain;Belgium;Denmark;Israel;Netherlands;Germany;Italy | ||
| 7 | NCT04762758 (ClinicalTrials.gov) | March 30, 2021 | 17/2/2021 | Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients | A Multi-center, Randomized, Double-blind, Placebo Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth Type 1A (CMT1A) | Charcot-Marie-Tooth Disease | Drug: (RS)-baclofen, naltrexone hydrochloride and D-sorbitol;Drug: Placebo | Pharnext SA | Worldwide Clinical Trials | Active, not recruiting | 16 Years | 65 Years | All | 350 | Phase 3 | United States;Belgium;Canada;Denmark;France;Germany;Israel;Italy;Spain;Netherlands |
| 8 | NCT05361031 (ClinicalTrials.gov) | September 21, 2020 | 31/3/2022 | The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A) | Single Center, Open Label, Repeat Intramuscular Administration, 270 Days, Phase I/2a Clinical Trial to Evaluate Safety, Tolerability of Investigational Product (Engensis: VM202) With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A) | CMT | Biological: Engensis (VM202) | Helixmith Co., Ltd. | NULL | Completed | 19 Years | 65 Years | All | 12 | Phase 1/Phase 2 | Korea, Republic of |
| 9 | JPRN-jRCTs031190109 | 25/10/2019 | 03/10/2019 | Ascorbic acid treatment for childhood Charcot-Marie-Tooth disease 1A | Ascorbic acid treatment for childhood Charcot-Marie-Tooth disease 1A - Ascorbic acid for CMT1A | Charcot-Marie-Tooth disease type 1A CMT1A;G600 | ascorbic acid dayly intake | SHIOHAMA TADASHI | NULL | Recruiting | Not applicable | < 18age old | Both | 12 | Phase 2 | Japan |
| 10 | JPRN-UMIN000036332 | 2019/06/15 | 28/03/2019 | Ascorbic acid treatment for Charcot-Marie-Tooth disease type 1A | Ascorbic acid treatment for Charcot-Marie-Tooth disease type 1A - Ascorbic acid treatment for CMT1A | Charcot-Marie-Tooth diease type 1A | Ascorbic acid treatment: 20mg/kg/day, dayly | Chiba University | NULL | Pending | 1years-old | Not applicable | Male and Female | 10 | Phase 1 | Japan |
| 11 | NCT03943290 (ClinicalTrials.gov) | May 10, 2019 | 17/4/2019 | Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX) | An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients With Charcot-Marie Tooth (CMT) Disease Types 1 and X Previously Enrolled in Study A083-03 | Facioscapulohumeral Muscular Dystrophy;Charcot-Marie-Tooth Disease | Drug: ACE-083 | Acceleron Pharma, Inc. | NULL | Terminated | 18 Years | N/A | All | 62 | Phase 2 | United States;Canada;Spain |
| 12 | EUCTR2015-002379-81-NL (EUCTR) | 08/01/2018 | 28/03/2017 | 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 | Not Recruiting | Female: yes Male: yes | 186 | Phase 3 | France;United States;Canada;Spain;Belgium;Germany;Netherlands;United Kingdom | ||
| 13 | NCT03254199 (ClinicalTrials.gov) | October 16, 2017 | 16/8/2017 | A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps. | A Randomized, Double-Blind, Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of FLX-787-ODT for Treatment of Muscle Cramps in Adult Subjects With Charcot-Marie-Tooth Disease | Charcot-Marie-Tooth Disease | Drug: FLX-787-ODT (orally disintegrating tablet);Drug: Placebo ODT | Flex Pharma, Inc. | NULL | Terminated | 18 Years | N/A | All | 27 | Phase 2 | United States |
| 14 | 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 | ||
| 15 | NCT03124459 (ClinicalTrials.gov) | July 31, 2017 | 12/4/2017 | Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease | A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease Types 1 and X | Charcot-Marie-Tooth Disease | Drug: ACE-083;Drug: Placebo | Acceleron Pharma, Inc. | NULL | Terminated | 18 Years | N/A | All | 62 | Phase 2 | United States |
| 16 | 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 | ||
| 17 | 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 | ||
| 18 | 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 | ||
| 19 | 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, 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;Netherlands;Spain;United Kingdom;Germany |
| 20 | 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 | ||
| 21 | NCT02967679 (ClinicalTrials.gov) | December 5, 2016 | 8/11/2016 | SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study | SERENDEM Study: MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study | Chronic Inflammatory Demyelinating Polyneuropathy;Peripheral Neuropathy;Charcot-Marie-Tooth Disease;Charcot-Marie-Tooth Disease Type 1A;Charcot-Marie-Tooth Disease, Type 1B;Anti-MAG Neuropathy | Drug: MD1003 | MedDay Pharmaceuticals SA | NULL | Completed | 20 Years | 85 Years | All | 15 | Phase 2 | France |
| 22 | 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 | ||
| 23 | EUCTR2015-001150-15-FR (EUCTR) | 14/03/2016 | 02/03/2016 | SERENDEM study: high dose biotin in patients suffering from disability related to peripheral nerve diseases | SERENDEM study: MD1003 in patients suffering from demyelinating neuropathies, an open label pilot study | chronic inflammatory demyelinating polyradiculoneuropathy, demyelinating neuropathy associated with immunoglobulin M (IgM) monoclonal gammopathy and antibodies against myelin-associated glycoprotein (MAG), Charcot Marie Tooth Ia (CMT Ia) neuropathy MedDRA version: 18.1;Level: LLT;Classification code 10066137;Term: Anti-MAG neuropathy;System Organ Class: 10029205 - Nervous system disorders MedDRA version: 18.1;Level: PT;Classification code 10057645;Term: Chronic inflammatory demyelinating polyradiculoneuropathy;Classification code 10008414;Term: Charcot-Marie-Tooth disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: biotin Product Code: MD1003 INN or Proposed INN: D-BIOTIN | MEDDAY PHARMACEUTICALS | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | France | ||
| 24 | 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 | ||
| 25 | 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 | ||
| 26 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Belgium;Spain;Netherlands;Germany;United Kingdom | ||
| 27 | 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 |
| 28 | NCT02561702 (ClinicalTrials.gov) | September 2015 | 24/9/2015 | Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease | Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease | Charcot Marie Tooth Disease | Other: Placebo;Drug: Mexiletine | University of Rochester | NULL | Completed | 18 Years | N/A | All | 4 | Phase 2 | United States |
| 29 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;France;Belgium;Spain;Netherlands;Germany;United Kingdom | ||
| 30 | EUCTR2015-001716-36-FR (EUCTR) | 03/08/2015 | 25/06/2015 | LONG TERM EFFECT AND TOLERANCE OF ULIPRISTAL ACETATE IN Charcot-Marie-TOOTH DISEASE TYPE 1A (UPACOMT) | LONG TERM EFFECT AND TOLERANCE OF ULIPRISTAL ACETATE IN Charcot-Marie-TOOTH DISEASE TYPE 1A (UPACOMT) - UPACOMT | 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] | Trade Name: elleOne Product Name: ULIPRISTAL ACETATE | HÔPITAUX UNIVERSITAIRES DE STRASBOURG | NULL | Not Recruiting | Female: no Male: yes | 45 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France | ||
| 31 | 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 |
| 32 | 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 | ||
| 33 | NCT00541164 (ClinicalTrials.gov) | September 2007 | 9/10/2007 | Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease | Effects of Coenzyme Q10 (CoQ10) on Subjects With Charcot-Marie-Tooth Disease (CMT):A Double Blind, Randomized, Controlled Trial With an Open Label Follow-up Study | Charcot Marie Tooth Disease | Drug: Coenzyme Q10;Dietary Supplement: Coenzyme Q10 | Memorial Medical Center | Department of Defense | Completed | 18 Years | 75 Years | Both | 23 | Phase 1/Phase 2 | United States |
| 34 | NCT00484510 (ClinicalTrials.gov) | April 2007 | 8/6/2007 | High Dose Ascorbic Acid Treatment of CMT1A | A Randomized, Placebo-controlled, Double Masked 120 Subject Futility Design Clinical Trial of Ascorbic Acid Treatment of Charcot Marie Tooth Disease Type 1A. | Charcot-Marie-Tooth Disease, Type Ia | Drug: Ascorbic acid (Vitamin C);Drug: placebo | Wayne State University | Muscular Dystrophy Association;Charcot-Marie-Tooth Association | Completed | 13 Years | 70 Years | Both | 110 | Phase 2/Phase 3 | United States |
| 35 | NCT00271635 (ClinicalTrials.gov) | January 2006 | 3/1/2006 | Ascorbic Acid Treatment in CMT1A Trial (AATIC) | Phase 2 Study of Ascorbic Acid Treatment in Charcot-Marie-Tooth Type 1A | Charcot-Marie-Tooth Disease;Hereditary Motor and Sensory Neuropathies | Drug: Placebo;Drug: ascorbic acid | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | NULL | Completed | 12 Years | 25 Years | Both | 13 | Phase 2 | Netherlands |
| 36 | EUCTR2006-000032-27-IT (EUCTR) | 19/10/2005 | 14/03/2006 | MULTICENTRE RANDOMISED DOUBLE BLIND PLACEBO CONTROLLED TRIAL OF LONG-TERM ASCORBIC ACID TREATMENT IN CHARCOT-MARIE-TOOTH DISEASE TYPE 1A CMT-TRIAAL CMT-TRial Italian with Ascorbic Acid Long term - CMT-TRIAAL | MULTICENTRE RANDOMISED DOUBLE BLIND PLACEBO CONTROLLED TRIAL OF LONG-TERM ASCORBIC ACID TREATMENT IN CHARCOT-MARIE-TOOTH DISEASE TYPE 1A CMT-TRIAAL CMT-TRial Italian with Ascorbic Acid Long term - CMT-TRIAAL | Charcot-Marie-Tooth disease type 1A CMT1A MedDRA version: 6.1;Level: HLGT;Classification code 10034606 | Trade Name: CEBION 500MG 20CPR MAST.ARAN INN or Proposed INN: ACIDO ASCORBICO DC.IT INN or Proposed INN: SODIO ASCORBATO DC.IT | ISTITUTO NEUROLOGICO CARLO BESTA | NULL | Not Recruiting | Female: yes Male: yes | 202 | Italy | |||
| 37 | JPRN-UMIN000001535 | 2005/04/01 | 01/01/2009 | A multicenter, randomized open trial of ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A | A multicenter, randomized open trial of ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A - CMT-AA | Charcot-Marie-Tooth disease 1A | Ascorbic acid (AA) treatment group (12 weeks oral AA (20mg/kg/day) no AA treatment group | 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. | NULL | Complete: follow-up complete | 15years-old | 75years-old | Male and Female | 60 | Phase 1,2 | Japan |
| 38 | EUCTR2004-004501-24-IT (EUCTR) | 22/12/2004 | 13/02/2007 | Multicentre, randomised, double blind, placebo controlled trial of ascorbic acid treatment in Charcot-Marie-Tooth Disease Type 1A CMT1A . | Multicentre, randomised, double blind, placebo controlled trial of ascorbic acid treatment in Charcot-Marie-Tooth Disease Type 1A CMT1A . | Charcot-Marie-Tooth Disease Type 1A CMT1A . MedDRA version: 6.1;Level: HLT;Classification code 10022031 | Trade Name: CEBION 500*20CPR MAST 500MG Product Name: ACIDO ASCORBICO INN or Proposed INN: Ascorbic acid vit C | ISTITUTO NEUROLOGICO CARLO BESTA | NULL | Not Recruiting | Female: yes Male: yes | 200 | Italy | |||
| 39 | EUCTR2020-004805-30-BE (EUCTR) | 04/03/2021 | Phase 3 trial of PXT3003 in Charcot-Marie-Tooth (CMT) Type 1A patients | A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A) | Charcot Marie Tooth 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 Name: NA Product Code: PXT3003 INN or Proposed INN: BACLOFEN Other descriptive name: (RS)-baclofen INN or Proposed INN: NALTREXONE Other descriptive name: naltrexone hydrochloride INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL | Pharnext SA | NULL | NA | Female: yes Male: yes | 387 | Phase 3 | France;United States;Canada;Spain;Belgium;Denmark;Israel;Netherlands;Germany;Italy | |||
| 40 | EUCTR2020-004805-30-DE (EUCTR) | 05/02/2021 | Phase 3 trial of PXT3003 in Charcot-Marie-Tooth (CMT) Type 1A patients | A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A) | Charcot Marie Tooth 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 Name: NA Product Code: PXT3003 INN or Proposed INN: BACLOFEN Other descriptive name: (RS)-baclofen INN or Proposed INN: NALTREXONE Other descriptive name: naltrexone hydrochloride INN or Proposed INN: Sorbitol Other descriptive name: D-SORBITOL | Pharnext SA | NULL | NA | Female: yes Male: yes | 387 | Phase 3 | France;United States;Canada;Belgium;Spain;Denmark;Israel;Netherlands;Germany;Italy | |||
| 41 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Belgium;Netherlands;Germany;United Kingdom |