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	2

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	EUCTR2011-005042-35-BE (EUCTR)	26/06/2013	21/03/2013	A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy	A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy	Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN053 Product Code: BMN053 INN or Proposed INN: PS524	BioMarin Nederland B.V.	NULL	Not Recruiting			Female: no Male: yes	42	Phase 1;Phase 2	United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan
2	NCT01957059 (ClinicalTrials.gov)	June 2013	2/7/2013	A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)	A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.	Duchenne Muscular Dystrophy	Drug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: Dosing Extension	BioMarin Pharmaceutical	NULL	Terminated	5 Years	18 Years	Male	9	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

EUCTR2011-005042-35-BE
(EUCTR)

26/06/2013

21/03/2013

A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy

A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy

Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BMN053
Product Code: BMN053
INN or Proposed INN: PS524

BioMarin Nederland B.V.

NULL

Not Recruiting

Female: no
Male: yes

Phase 1;Phase 2

United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan

NCT01957059
(ClinicalTrials.gov)

June 2013

2/7/2013

A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy

Drug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: Dosing Extension

BioMarin Pharmaceutical

NULL

Terminated

5 Years

18 Years

Male

Phase 1;Phase 2

Belgium;France;Italy;Netherlands;United Kingdom