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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-jRCTs031190109
03/10/201903/10/2019Ascorbic acid treatment for childhood Charcot-Marie-Tooth disease 1AAscorbic acid treatment for childhood Charcot-Marie-Tooth disease 1A - Ascorbic acid for CMT1A Charcot-Marie-Tooth disease type 1A
CMT1A;G600
ascorbic acid dayly intakeFUJII KATSUNORINULLRecruitingNot applicable< 18age oldBoth12Phase 2Japan
2JPRN-UMIN000036332
2019/06/1528/03/2019Ascorbic acid treatment for Charcot-Marie-Tooth disease type 1AAscorbic acid treatment for Charcot-Marie-Tooth disease type 1A - Ascorbic acid treatment for CMT1A Charcot-Marie-Tooth diease type 1AAscorbic acid treatment: 20mg/kg/day, daylyChiba UniversityNULLPending1years-oldNot applicableMale and Female10Phase 1Japan
3NCT03943290
(ClinicalTrials.gov)
May 10, 201917/4/2019Extension 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.NULLTerminated18 YearsN/AAll62Phase 2United States;Canada;Spain
4EUCTR2015-002379-81-NL
(EUCTR)
08/01/201828/03/2017An 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 SANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
290Phase 3France;United States;Canada;Belgium;Spain;Germany;Netherlands;United Kingdom
5NCT03254199
(ClinicalTrials.gov)
October 16, 201716/8/2017A 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.NULLTerminated18 YearsN/AAll27Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2015-002379-81-DE
(EUCTR)
15/08/201719/01/2017An 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
PharnextNULLNot RecruitingFemale: yes
Male: yes
290Phase 3France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom
7NCT03124459
(ClinicalTrials.gov)
July 31, 201712/4/2017Study 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.NULLTerminated18 YearsN/AAll62Phase 2United States
8EUCTR2015-002379-81-GB
(EUCTR)
28/07/201712/06/2017An 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 SANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
187Phase 3United States;France;Canada;Spain;Belgium;Germany;Netherlands;United Kingdom
9EUCTR2015-002379-81-ES
(EUCTR)
25/05/201707/04/2017An 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
PharnextNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
300Phase 3France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom
10EUCTR2015-002379-81-BE
(EUCTR)
04/05/201721/02/2017An 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
PharnextNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
300Phase 3France;United States;Canada;Spain;Belgium;Netherlands;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03023540
(ClinicalTrials.gov)
March 7, 201726/12/2016Assessing 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 plcActive, not recruiting16 Years67 YearsAll187Phase 3United States;Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom
12EUCTR2015-002379-81-FR
(EUCTR)
26/01/201712/01/2017An 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
PharnextNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
300Phase 3United States;France;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom
13NCT02967679
(ClinicalTrials.gov)
December 5, 20168/11/2016SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot StudySERENDEM Study: 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 1B;Anti-MAG NeuropathyDrug: MD1003MedDay Pharmaceuticals SANULLCompleted20 Years85 YearsAll15Phase 2France
14EUCTR2015-002378-19-NL
(EUCTR)
21/04/201617/12/2015International, 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-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 SANULLNot RecruitingFemale: yes
Male: yes
300Phase 3United States;Belgium;Spain;Germany;Netherlands;United Kingdom
15EUCTR2015-001150-15-FR
(EUCTR)
14/03/201602/03/2016SERENDEM study: high dose biotin in patients suffering from disability related to peripheral nerve diseasesSERENDEM 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 PHARMACEUTICALSNULLNot RecruitingFemale: yes
Male: yes
15Phase 2France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2015-002378-19-DE
(EUCTR)
11/02/201628/09/2015International, 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-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
PHARNEXTNULLNot RecruitingFemale: yes
Male: yes
300Phase 3United States;Belgium;Spain;Netherlands;Germany;United Kingdom
17EUCTR2015-002378-19-BE
(EUCTR)
18/01/201619/11/2015International, 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-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 SANULLNot RecruitingFemale: yes
Male: yes
300Phase 3United States;Spain;Belgium;Netherlands;Germany;United Kingdom
18EUCTR2015-002378-19-ES
(EUCTR)
28/12/201530/10/2015International, 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-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]
PHARNEXTNULLNot Recruiting Female: yes
Male: yes
300Phase 3France;United States;Belgium;Spain;Netherlands;Germany;United Kingdom
19NCT02579759
(ClinicalTrials.gov)
December 201528/9/2015Phase 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 SANULLCompleted16 Years65 YearsAll323Phase 3United States;Belgium;Canada;France;Germany;Netherlands;Spain;United Kingdom
20NCT02561702
(ClinicalTrials.gov)
September 201524/9/2015Mexiletine for Muscle Cramps in Charcot Marie Tooth DiseaseMexiletine for Muscle Cramps in Charcot Marie Tooth DiseaseCharcot Marie Tooth DiseaseOther: Placebo;Drug: MexiletineUniversity of RochesterNULLCompleted18 YearsN/AAll4Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2015-002378-19-FR
(EUCTR)
27/08/201527/07/2015 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]
PHARNEXTNULLNot Recruiting Female: yes
Male: yes
300Phase 3United States;France;Belgium;Spain;Netherlands;Germany;United Kingdom
22EUCTR2015-001716-36-FR
(EUCTR)
03/08/201525/06/2015LONG 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) - 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]
HÔPITAUX UNIVERSITAIRES DE STRASBOURGNULLNot Recruiting Female: no
Male: yes
45Phase 2France
23NCT01401257
(ClinicalTrials.gov)
December 201020/7/2011Phase 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 SANULLCompleted18 Years65 YearsAll80Phase 2France
24EUCTR2010-023097-40-FR
(EUCTR)
29/11/201004/10/2010A 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 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
PharnextNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
80Phase 2France
25NCT00541164
(ClinicalTrials.gov)
September 20079/10/2007Effects 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 DiseaseDrug: Coenzyme Q10;Dietary Supplement: Coenzyme Q10Memorial Medical CenterDepartment of DefenseCompleted18 Years75 YearsBoth23Phase 1;Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT00484510
(ClinicalTrials.gov)
April 20078/6/2007High 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 AssociationCompleted13 Years70 YearsBoth110Phase 2;Phase 3United States
27NCT00271635
(ClinicalTrials.gov)
January 20063/1/2006Ascorbic 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)NULLCompleted12 Years25 YearsBoth13Phase 2Netherlands
28EUCTR2006-000032-27-IT
(EUCTR)
19/10/200514/03/2006MULTICENTRE 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-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 BESTANULLNot RecruitingFemale: yes
Male: yes
202Italy
29JPRN-UMIN000001535
2005/04/0101/01/2009A multicenter, randomized open trial of ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A Charcot-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.NULLComplete: follow-up complete15years-old75years-oldMale and Female60Phase 1;Phase 2Japan
30EUCTR2004-004501-24-IT
(EUCTR)
22/12/200413/02/2007Multicentre, 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 BESTANULLNot RecruitingFemale: yes
Male: yes
200Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31EUCTR2015-002378-19-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]
PHARNEXTNULLNot Recruiting Female: yes
Male: yes
300Phase 3France;United States;Belgium;Netherlands;Germany;United Kingdom