DDrare: Database of Drug Development for Rare Diseases

10. シャルコー・マリー・トゥース病
［臨床試験数：31，薬物数：38（DrugBank：9），標的遺伝子数：11，標的パスウェイ数：14］

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.

Drug = ACE-083; ACIDO ASCORBICO; ACIDO ASCORBICO DC.IT; Acetate; Ascorbic acid; Ascorbic acid (Vitamin C); Ascorbic acid vit C; BACLOFEN; Baclofen; Biotin; CEBION 500*20CPR MAST 500MG; CEBION 500MG 20CPR MAST.ARAN; Coenzyme Q10; D-BIOTIN; D-SORBITOL; ElleOne; FLX-787-ODT (orally disintegrating tablet); MD1003; Mexiletine; NALTREXONE; NALTREXONE HYDROCHLORIDE; Naltrexone; Non applicable; PXT3003; PXT3003 Dose 1; PXT3003 Dose 2; PXT3003 Dose 2 (equivalent to twice Dose 1); PXT3003 High Dose; PXT3003 Intermediate Dose; PXT3003 Low dose; PXT3003 dose 1; PXT3003 dose 2; RS-BACLOFEN; SODIO ASCORBATO DC.IT; Sorbitol; ULIPRISTAL ACETATE; Ulipristal; Vitamin C

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	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

No.

TrialID

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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

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

Phase 2

United States