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"
16 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2015-002379-81-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
2EUCTR2015-002379-81-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
3EUCTR2015-002379-81-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
4EUCTR2015-002379-81-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
5EUCTR2015-002379-81-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
6NCT03023540
(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
7EUCTR2015-002379-81-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
8EUCTR2015-002378-19-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
9EUCTR2015-002378-19-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
10EUCTR2015-002378-19-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2015-002378-19-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
12NCT02579759
(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
13EUCTR2015-002378-19-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
14NCT01401257
(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
15EUCTR2010-023097-40-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
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-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