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	EUCTR2015-002069-52-IT (EUCTR)	19/01/2017	07/02/2018	A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients	A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A	Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;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: SRP-4045 Product Code: SRP-4045 Other descriptive name: Oligomero morfolino fosforodiamidato per skipping dell’esone 45 Product Name: SRP-4053 Product Code: SRP-4053 Other descriptive name: Oligomero morfolino fosforodiamidato per skipping dell’esone 53	SAREPTA THERAPEUTICS, INC.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	99	Phase 3	France;Czech Republic;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;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

EUCTR2015-002069-52-IT
(EUCTR)

19/01/2017

07/02/2018

A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A

Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
Other descriptive name: Oligomero morfolino fosforodiamidato per skipping dell’esone 45
Product Name: SRP-4053
Product Code: SRP-4053
Other descriptive name: Oligomero morfolino fosforodiamidato per skipping dell’esone 53

SAREPTA THERAPEUTICS, INC.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

Phase 3

France;Czech Republic;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden