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	NCT03943290 (ClinicalTrials.gov)	May 10, 2019	17/4/2019	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.	NULL	Terminated	18 Years	N/A	All	62	Phase 2	United States;Canada;Spain
2	NCT03124459 (ClinicalTrials.gov)	July 31, 2017	12/4/2017	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.	NULL	Terminated	18 Years	N/A	All	62	Phase 2	United States

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

NCT03943290
(ClinicalTrials.gov)

May 10, 2019

17/4/2019

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.

NULL

Terminated

18 Years

N/A

All

Phase 2

United States;Canada;Spain

NCT03124459
(ClinicalTrials.gov)

July 31, 2017

12/4/2017

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.

NULL

Terminated

18 Years

N/A

All

Phase 2

United States