DDrare: Database of Drug Development for Rare Diseases

ID	Disease name (Link within this page)	Number of trials
299	Cystic fibrosis	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	NCT02516410 (ClinicalTrials.gov)	August 2015	28/7/2015	A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation	A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and With a Second CFTR Mutation That Is Not Likely to Respond to VX-661 and/or Ivacaftor Therapy (F508del/NR)	Cystic Fibrosis	Drug: VX-661 plus ivacaftor combination;Drug: Ivacaftor;Drug: Placebo (matched to VX-661 plus ivacaftor combination);Drug: Placebo (matched to ivacaftor)	Vertex Pharmaceuticals Incorporated	NULL	Completed	12 Years	N/A	All	168	Phase 3	United States;Australia;Austria;Canada;France;Israel;Spain
2	NCT02347657 (ClinicalTrials.gov)	January 2015	12/1/2015	A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor	A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation	Cystic Fibrosis	Drug: VX-661 Plus Ivacaftor Combination;Drug: Ivacaftor;Drug: VX-661 Plus Ivacaftor Combination Placebo;Drug: Ivacaftor placebo	Vertex Pharmaceuticals Incorporated	NULL	Completed	12 Years	N/A	All	510	Phase 3	United States;Canada;Denmark;France;Germany;Ireland;Italy;Netherlands;Spain;Sweden;Switzerland;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

NCT02516410
(ClinicalTrials.gov)

August 2015

28/7/2015

A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and With a Second CFTR Mutation That Is Not Likely to Respond to VX-661 and/or Ivacaftor Therapy (F508del/NR)

Cystic Fibrosis

Drug: VX-661 plus ivacaftor combination;Drug: Ivacaftor;Drug: Placebo (matched to VX-661 plus ivacaftor combination);Drug: Placebo (matched to ivacaftor)

Vertex Pharmaceuticals Incorporated

NULL

Completed

12 Years

N/A

All

168

Phase 3

United States;Australia;Austria;Canada;France;Israel;Spain

NCT02347657
(ClinicalTrials.gov)

January 2015

12/1/2015

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor

A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation