DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
10	シャルコー・マリー・トゥース病	1

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

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

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