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
299	嚢胞性線維症	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	EUCTR2009-010261-23-Outside-EU/EEA (EUCTR)		06/06/2011	Study of VX-770 in Cystic Fibrosis Subjects Age 12 and Older Homozygous for the F508del-CFTR Mutation	A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of VX-770 in Subjects Aged 12 Years and Older With Cystic Fibrosis who are Homozygous for the F508del-CFTR Mutation - DISCOVER	Cystic Fibrosis MedDRA version: 13.1;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: VX-770 Product Code: VX-770; VRT-813077 INN or Proposed INN: ivacaftor (proposed INN) Other descriptive name: VRT-813077	Vertex Pharmaceuticals Incorporated	NULL	NA			Female: yes Male: yes	140	Phase 2	United States

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

EUCTR2009-010261-23-Outside-EU/EEA
(EUCTR)

06/06/2011

Study of VX-770 in Cystic Fibrosis Subjects Age 12 and Older Homozygous for the F508del-CFTR Mutation

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of VX-770 in Subjects Aged 12 Years and Older With Cystic Fibrosis who are Homozygous for the F508del-CFTR Mutation - DISCOVER

Cystic Fibrosis
MedDRA version: 13.1;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: VX-770
Product Code: VX-770; VRT-813077
INN or Proposed INN: ivacaftor (proposed INN)
Other descriptive name: VRT-813077

Vertex Pharmaceuticals Incorporated

NULL

Female: yes
Male: yes

140

Phase 2

United States