10. シャルコー・マリー・トゥース病 ［臨床試験数：19，薬物数：31（DrugBank：9），標的遺伝子数：12，標的パスウェイ数：14］
Searched query = "Charcot Marie Tooth disease"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-jRCTs031190109 | 03/10/2019 | 7 October 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 | KATSUNORI FUJII | Recruiting | Not applicable | < 18age old | Both | 12 | Phase 2 | none | |
| 2 | JPRN-UMIN000036332 | 2019/06/15 | 23 April 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 | Not Recruiting | 1years-old | Not applicable | Male and Female | 10 | Phase 1 | Japan | |
| 3 | NCT03943290 | May 10, 2019 | 14 October 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. | Recruiting | 18 Years | N/A | All | 150 | Phase 2 | United States;Canada;Spain | |
| 4 | NCT03254199 | October 16, 2017 | 10 September 2018 | 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. | Not recruiting | 18 Years | N/A | All | 27 | Phase 2 | United States | |
| 5 | NCT03124459 | July 31, 2017 | 11 November 2019 | 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. | Not recruiting | 18 Years | N/A | All | 63 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03023540 | March 7, 2017 | 30 September 2019 | 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 | Not recruiting | 16 Years | 67 Years | All | 187 | Phase 3 | United States;Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom |
| 7 | NCT02967679 | September 2016 | 28 November 2016 | SERENDEM : 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 IA | Drug: MD1003 | MedDay Pharmaceuticals SA | Recruiting | 20 Years | 85 Years | Both | 15 | Phase 1/Phase 2 | France | ||
| 8 | NCT02579759 | December 2015 | 22 October 2018 | 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 | Not recruiting | 16 Years | 65 Years | All | 323 | Phase 3 | United States;Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom | |
| 9 | NCT02561702 | September 2015 | 16 December 2017 | 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 | Not recruiting | 18 Years | N/A | All | 4 | Phase 2 | United States | |
| 10 | EUCTR2015-001716-36-FR | 03/08/2015 | 26 October 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 Pharmaceutical Form: Tablet Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | HÔPITAUX UNIVERSITAIRES DE STRASBOURG | Authorised | Female: no Male: yes | France | |||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | 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-GB | 28 February 2019 | 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 Pharmaceutical Form: Oral solution INN or Proposed INN: RS-BACLOFEN CAS Number: 1134-47-0 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 0.6- INN or Proposed INN: NALTREXONE HYDROCHLORIDE CAS Number: 16676-29-2 Other descriptive name: NALTREXONE HYDROCHLORIDE Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 0.07- INN or Proposed INN: D-SORBITOL CAS Number: 50-70-4 Other descriptive name: D-SORBITOL Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 21- Pharmaceutical form of the placebo: Oral solution Route of administration of the placebo: Oral use Product Code: PXT3003 dose 2 Pharmaceutical Form: Oral solution INN or Proposed INN: RS-BACLOFEN CAS Number: 1134-47-0 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 1.2- INN or Proposed INN: NALTREXONE HYDROCHLORIDE CAS Number: 16676-29-2 Other descriptive name: NALTREXONE HYDROCHLORIDE Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 0.14- INN or Proposed INN: D-SORBITOL CAS Number: 50-70-4 Other descriptive name: D-SORBITOL Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal | PHARNEXT | Not Recruiting | Female: yes Male: yes | 300 | Phase 3 | France;United States;Belgium;Netherlands;Germany;United Kingdom | ||||
| 12 | NCT01401257 | December 2010 | 16 December 2017 | 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 | Not recruiting | 18 Years | 65 Years | All | 80 | Phase 2 | France | |
| 13 | EUCTR2010-023097-40-FR | 29/11/2010 | 19 March 2012 | 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 Pharmaceutical Form: Oral solution INN or Proposed INN: Baclofen CAS Number: 1134-47-0 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 0.3- INN or Proposed INN: Naltrexone CAS Number: 16676-29-2 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 0.035- INN or Proposed INN: Sorbitol CAS Number: 50-70-4 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 10.5- Pharmaceutical form of the placebo: Oral solution Route of administration of the placebo: Oral use Product Name: non applicable Product Code: PXT3003 Pharmaceutical Form: Oral solution INN or Proposed INN: Baclofen CAS Number: 1134-47-0 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 0.6- INN or Proposed INN: Naltrexone CAS Number: 16676-29-2 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 0.07- INN or Proposed INN: Sorbitol CAS Number: 50-70-4 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 21- Pharmaceutical form of the placebo: Oral solution Route of administration of the placebo: Oral use Product Name: non applicable Product Code: PXT3003 Pharmaceutical Form: Oral solution INN or Proposed INN: Baclofen CAS Number: 1134-47-0 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 3- INN or Proposed INN: Naltrexone CAS Number: 16676-29-2 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 0.35- INN or Proposed INN: Sorbitol CAS Number: 50-70-4 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 105- Pharmaceutical form of the placebo: Oral solution Route of administration of the placebo: Oral use | Pharnext | Authorised | Female: yes Male: yes | 80 | Phase 2 | France | |||
| 14 | NCT00541164 | September 2007 | 19 February 2015 | 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 | Dietary Supplement: Coenzyme Q10;Drug: Coenzyme Q10 | Memorial Medical Center | Department of Defense | Not recruiting | 18 Years | 75 Years | Both | 23 | Phase 1/Phase 2 | United States |
| 15 | NCT00484510 | April 2007 | 19 February 2015 | 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 | Not recruiting | 13 Years | 70 Years | Both | 110 | Phase 2/Phase 3 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT00271635 | January 2006 | 19 February 2015 | 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) | Not recruiting | 12 Years | 25 Years | Both | 13 | Phase 2 | Netherlands | |
| 17 | EUCTR2006-000032-27-IT | 19/10/2005 | 19 March 2012 | 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 Pharmaceutical Form: Chewable tablet INN or Proposed INN: ACIDO ASCORBICO DC.IT CAS Number: 50-81-7 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- INN or Proposed INN: SODIO ASCORBATO DC.IT CAS Number: 134-03-2 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- Pharmaceutical form of the placebo: Chewable tablet Route of administration of the placebo: Oral use | ISTITUTO NEUROLOGICO CARLO BESTA | Not Recruiting | Female: yes Male: yes | 202 | Italy | ||||
| 18 | JPRN-UMIN000001535 | 2005/04/01 | 2 April 2019 | A multicenter, randomized open trial of ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A | 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. | Not Recruiting | 15years-old | 75years-old | Male and Female | 60 | Phase 1,2 | Japan | ||
| 19 | EUCTR2004-004501-24-IT | 22/12/2004 | 19 March 2012 | 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 Pharmaceutical Form: Chewable tablet INN or Proposed INN: Ascorbic acid vit C CAS Number: 50-81-7 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- | ISTITUTO NEUROLOGICO CARLO BESTA | Not Recruiting | Female: yes Male: yes | 200 | Italy |