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 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:9,標的パスウェイ数:9] 

Searched query = "Charcot-Marie-Tooth disease"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-jRCTs03119010903/10/20197 October 2019Ascorbic acid treatment for childhood Charcot-Marie-Tooth disease 1AAscorbic acid treatment for childhood Charcot-Marie-Tooth disease 1A - Ascorbic acid for CMT1ACharcot-Marie-Tooth disease type 1A
CMT1A;G600
ascorbic acid dayly intakeKATSUNORI FUJIIRecruitingNot applicable< 18age oldBoth12Phase 2none
2JPRN-UMIN0000363322019/06/1523 April 2019Ascorbic acid treatment for Charcot-Marie-Tooth disease type 1AAscorbic acid treatment for Charcot-Marie-Tooth disease type 1A - Ascorbic acid treatment for CMT1ACharcot-Marie-Tooth diease type 1AAscorbic acid treatment: 20mg/kg/day, daylyChiba UniversityNot Recruiting1years-oldNot applicableMale and Female10Phase 1Japan
3NCT03943290May 10, 201914 October 2019Extension 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 A083-03Facioscapulohumeral Muscular Dystrophy;Charcot-Marie-Tooth DiseaseDrug: ACE-083Acceleron Pharma, Inc.Recruiting18 YearsN/AAll150Phase 2United States;Canada;Spain
4NCT03254199October 16, 201710 September 2018A 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 DiseaseCharcot-Marie-Tooth DiseaseDrug: FLX-787-ODT (orally disintegrating tablet);Drug: Placebo ODTFlex Pharma, Inc.Not recruiting18 YearsN/AAll27Phase 2United States
5NCT03124459July 31, 201711 November 2019Study of ACE-083 in Patients With Charcot-Marie-Tooth DiseaseA Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease Types 1 and XCharcot-Marie-Tooth DiseaseDrug: ACE-083;Drug: PlaceboAcceleron Pharma, Inc.Not recruiting18 YearsN/AAll63Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03023540March 7, 201730 September 2019Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1AInternational, 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 1ACharcot-Marie-Tooth Disease, Type IADrug: PXT3003Pharnext SASynteractHCR;Syneos Health;Premier Research Group plcNot recruiting16 Years67 YearsAll187Phase 3United States;Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom
7NCT02967679September 201628 November 2016SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot StudyChronic Inflammatory Demyelinating Polyneuropathy;Peripheral Neuropathy;Charcot-Marie-Tooth Disease;Charcot-Marie-Tooth Disease Type 1A;Charcot-Marie-Tooth Disease, Type IADrug: MD1003MedDay Pharmaceuticals SARecruiting20 Years85 YearsBoth15Phase 1/Phase 2France
8NCT02579759December 201522 October 2018Phase 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 MonthsCharcot-Marie-Tooth Disease Type 1ADrug: PXT3003 dose 1;Drug: PXT3003 dose 2;Drug: placeboPharnext SANot recruiting16 Years65 YearsAll323Phase 3United States;Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom
9NCT02561702September 201516 December 2017Mexiletine for Muscle Cramps in Charcot Marie Tooth DiseaseMexiletine for Muscle Cramps in Charcot Marie Tooth DiseaseCharcot Marie Tooth DiseaseOther: Placebo;Drug: MexiletineUniversity of RochesterNot recruiting18 YearsN/AAll4Phase 2United States
10EUCTR2015-001716-36-FR03/08/201526 October 2015LONG 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) - UPACOMTCharcot-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 STRASBOURGAuthorisedFemale: no
Male: yes
France
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2015-002378-19-GB28 February 2019International, 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 monthsInternational, 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-CMTCharcot-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
PHARNEXTNot Recruiting Female: yes
Male: yes
300Phase 3France;United States;Belgium;Netherlands;Germany;United Kingdom
12NCT01401257December 201016 December 2017Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1AA 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 DisordersDrug: PXT3003 Low dose;Drug: PXT3003 Intermediate Dose;Drug: PXT3003 High Dose;Other: PlaceboPharnext SANot recruiting18 Years65 YearsAll80Phase 2France
13EUCTR2010-023097-40-FR29/11/201019 March 2012A Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX3003 in Patients with Charcot-Marie-Tooth Disease Type 1A - non applicableA Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX3003 in Patients with Charcot-Marie-Tooth Disease Type 1A - non applicableCharcot-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
PharnextAuthorisedFemale: yes
Male: yes
80Phase 2France
14NCT00541164September 200719 February 2015Effects of Coenzyme Q10 on Charcot-Marie-Tooth DiseaseEffects of Coenzyme Q10 (CoQ10) on Subjects With Charcot-Marie-Tooth Disease (CMT):A Double Blind, Randomized, Controlled Trial With an Open Label Follow-up StudyCharcot Marie Tooth DiseaseDietary Supplement: Coenzyme Q10;Drug: Coenzyme Q10Memorial Medical CenterDepartment of DefenseNot recruiting18 Years75 YearsBoth23Phase 1/Phase 2United States
15NCT00484510April 200719 February 2015High Dose Ascorbic Acid Treatment of CMT1AA 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 IaDrug: Ascorbic acid (Vitamin C);Drug: placeboWayne State UniversityMuscular Dystrophy Association;Charcot-Marie-Tooth AssociationNot recruiting13 Years70 YearsBoth110Phase 2/Phase 3United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT00271635January 200619 February 2015Ascorbic Acid Treatment in CMT1A Trial (AATIC)Phase 2 Study of Ascorbic Acid Treatment in Charcot-Marie-Tooth Type 1ACharcot-Marie-Tooth Disease;Hereditary Motor and Sensory NeuropathiesDrug: Placebo;Drug: ascorbic acidAcademisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)Not recruiting12 Years25 YearsBoth13Phase 2Netherlands
17EUCTR2006-000032-27-IT19/10/200519 March 2012MULTICENTRE 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-TRIAALMULTICENTRE 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-TRIAALCharcot-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 BESTANot RecruitingFemale: yes
Male: yes
202Italy
18JPRN-UMIN0000015352005/04/012 April 2019A multicenter, randomized open trial of ascorbic acid treatment in Charcot-Marie-Tooth disease type 1ACharcot-Marie-Tooth disease 1AAscorbic 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 Recruiting15years-old75years-oldMale and Female60Phase 1,2Japan
19EUCTR2004-004501-24-IT22/12/200419 March 2012Multicentre, 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 BESTANot RecruitingFemale: yes
Male: yes
200Italy

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