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
113	筋ジストロフィー	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	NCT01826474 (ClinicalTrials.gov)	January 2013	20/3/2013	Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy	A Phase IIb, Open-label Study to Assess the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of Multiple Subcutaneous Doses of PRO045 in Subjects With Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Drug: PRO045, 0.15 mg/kg/week;Drug: PRO045, 1.0 mg/kg/week;Drug: PRO045, 3.0 mg/kg/week;Drug: PRO045, 6.0 mg/kg/week;Drug: PRO045, 9.0 mg/kg/week;Drug: PRO045, selected dose	BioMarin Pharmaceutical	NULL	Terminated	5 Years	18 Years	Male	15	Phase 1;Phase 2	Belgium;France;Italy;Netherlands;United Kingdom

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

NCT01826474
(ClinicalTrials.gov)

January 2013

20/3/2013

Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy

A Phase IIb, Open-label Study to Assess the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of Multiple Subcutaneous Doses of PRO045 in Subjects With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Drug: PRO045, 0.15 mg/kg/week;Drug: PRO045, 1.0 mg/kg/week;Drug: PRO045, 3.0 mg/kg/week;Drug: PRO045, 6.0 mg/kg/week;Drug: PRO045, 9.0 mg/kg/week;Drug: PRO045, selected dose

BioMarin Pharmaceutical

NULL

Terminated

5 Years

18 Years

Male

Phase 1;Phase 2

Belgium;France;Italy;Netherlands;United Kingdom