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

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

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	NCT02958202 (ClinicalTrials.gov)	April 2016	27/10/2016	Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)	A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Drug: BMN 044 IV 6 mg/kg;Drug: BMN 044 IV 9 mg/kg;Drug: BMN 044 SC 6 mg/kg	BioMarin Pharmaceutical	NULL	Terminated	5 Years	N/A	Male	7	Phase 2	Belgium;Italy;Netherlands;Sweden

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

NCT02958202
(ClinicalTrials.gov)

April 2016

27/10/2016

Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Drug: BMN 044 IV 6 mg/kg;Drug: BMN 044 IV 9 mg/kg;Drug: BMN 044 SC 6 mg/kg

BioMarin Pharmaceutical

NULL

Terminated

5 Years

N/A

Male

Phase 2

Belgium;Italy;Netherlands;Sweden