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	NCT01401257 (ClinicalTrials.gov)	December 2010	20/7/2011	Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A	A 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 Disorders	Drug: PXT3003 Low dose;Drug: PXT3003 Intermediate Dose;Drug: PXT3003 High Dose;Other: Placebo	Pharnext SA	NULL	Completed	18 Years	65 Years	All	80	Phase 2	France

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agemin

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NCT01401257
(ClinicalTrials.gov)

December 2010

20/7/2011

Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A

A 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 Disorders

Drug: PXT3003 Low dose;Drug: PXT3003 Intermediate Dose;Drug: PXT3003 High Dose;Other: Placebo

Pharnext SA

NULL

Completed

18 Years

65 Years

All

Phase 2

France