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	EUCTR2019-002921-31-GB (EUCTR)	29/09/2020	02/07/2020	Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy.	A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY	DUCHENNE MUSCULAR DYSTROPHY (DMD) 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: PF-06939926 Product Code: PF-06939926 INN or Proposed INN: PF-06939926 Other descriptive name: Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene	Pfizer Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	99	Phase 3	United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Italy;Switzerland;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of

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

EUCTR2019-002921-31-GB
(EUCTR)

29/09/2020

02/07/2020

Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy.

A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

DUCHENNE MUSCULAR DYSTROPHY (DMD)
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: PF-06939926
Product Code: PF-06939926
INN or Proposed INN: PF-06939926
Other descriptive name: Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene

Pfizer Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

Phase 3

United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Italy;Switzerland;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of