MedDataX Logo

Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion

NCT ID: NCT02070744

Last Updated: 2025-09-24

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-03-31

Study Completion Date

2016-05-27

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The objective of this study was to evaluate the safety and efficacy of VX-661in combination with ivacaftor in participants with cystic fibrosis (CF) who are homozygous for F508del cystic fibrosis transmembrane conductance regulator (CFTR) mutation

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

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

NCT02516410

Cystic Fibrosis
COMPLETED PHASE3

Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation

NCT01531673

Cystic Fibrosis
COMPLETED PHASE2

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

NCT02347657

Cystic Fibrosis
COMPLETED PHASE3

A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation

NCT02392234

Cystic Fibrosis
COMPLETED PHASE3

A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

NCT02508207

Cystic Fibrosis
COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h

Participants received VX-661 50 milligram (mg) tablet plus Ivacaftor (IVA) 150 mg tablet every 12 hours (q12h) for 12 weeks.

Group Type EXPERIMENTAL

VX-661

Intervention Type DRUG

Tablet, oral use

Ivacaftor

Intervention Type DRUG

Film coated tablet, oral use

PC Phase: VX 661 placebo q12h + IVA placebo q12h

Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.

Group Type PLACEBO_COMPARATOR

Placebo matched to VX-661

Intervention Type DRUG

Tablet, oral use

Placebo matched to Ivacaftor

Intervention Type DRUG

Film coated tablet, oral use

PC Phase: VX-661 100 mg qd + IVA 150 mg q12h

Participants received two VX-661 50 mg tablets once daily (qd) plus IVA 150 mg tablet q12h for 12 weeks.

Group Type EXPERIMENTAL

VX-661

Intervention Type DRUG

Tablet, oral use

Ivacaftor

Intervention Type DRUG

Film coated tablet, oral use

PC Phase: VX -661 placebo qd + IVA placebo q12h

Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.

Group Type PLACEBO_COMPARATOR

Placebo matched to VX-661

Intervention Type DRUG

Tablet, oral use

Placebo matched to Ivacaftor

Intervention Type DRUG

Film coated tablet, oral use

OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h

Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.

Group Type EXPERIMENTAL

VX-661

Intervention Type DRUG

Tablet, oral use

Ivacaftor

Intervention Type DRUG

Film coated tablet, oral use

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

VX-661

Tablet, oral use

Intervention Type DRUG

Ivacaftor

Film coated tablet, oral use

Intervention Type DRUG

Placebo matched to VX-661

Tablet, oral use

Intervention Type DRUG

Placebo matched to Ivacaftor

Film coated tablet, oral use

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Homozygous for the F508del CFTR mutation
* FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height
* Stable CF disease as judged by the investigator

Exclusion Criteria

* History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the participant
* Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at screening and Day 1 of the PC Phase and Day -7 or Day 1 of the OLE Phase (whichever was applicable)
* Sexually active participants of reproductive potential who are not willing to follow the contraception requirements
* The participant or a close relative of the participant is the investigator or sub investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Birmingham, Alabama, United States

Site Status

Palo Alto, California, United States

Site Status

Stanford, California, United States

Site Status

Altamonte Springs, Florida, United States

Site Status

Miami, Florida, United States

Site Status

Orlando, Florida, United States

Site Status

Tampa, Florida, United States

Site Status

Boise, Idaho, United States

Site Status

Chicago, Illinois, United States

Site Status

Boston, Massachusetts, United States

Site Status

New Brunswick, New Jersey, United States

Site Status

New York, New York, United States

Site Status

Durham, North Carolina, United States

Site Status

Cincinnati, Ohio, United States

Site Status

Oklahoma City, Oklahoma, United States

Site Status

Pittsburgh, Pennsylvania, United States

Site Status

Charleston, South Carolina, United States

Site Status

Memphis, Tennessee, United States

Site Status

Houston, Texas, United States

Site Status

Burlington, Vermont, United States

Site Status

Colchester, Vermont, United States

Site Status

Seattle, Washington, United States

Site Status

Milwaukee, Wisconsin, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

VX13-661-103

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation
NCT00953706 TERMINATED PHASE2
A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation
NCT02565914 COMPLETED PHASE3
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
NCT05668741 RECRUITING PHASE1/PHASE2
A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor
NCT02412111 COMPLETED PHASE3
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
NCT00909532 COMPLETED PHASE3
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
NCT00909727 COMPLETED PHASE3
Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
NCT01225211 COMPLETED PHASE2
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
NCT03525444 COMPLETED PHASE3
Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation
NCT00865904 COMPLETED PHASE2
Safety Study of Ivacaftor in Subjects With Cystic Fibrosis
NCT00457821 COMPLETED PHASE2
A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis
NCT03227471 COMPLETED PHASE1/PHASE2
A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis
NCT03224351 COMPLETED PHASE2
A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
NCT02730208 COMPLETED PHASE2
A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation
NCT02514473 COMPLETED PHASE3
A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis
NCT02951182 COMPLETED PHASE2
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
NCT02709109 COMPLETED PHASE2
A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del
NCT04105972 COMPLETED PHASE3
A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
NCT03525548 COMPLETED PHASE3
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF)
NCT02953314 COMPLETED PHASE3
Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation
NCT01262352 COMPLETED PHASE2
A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation
NCT05076149 COMPLETED PHASE3
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
NCT03447249 COMPLETED PHASE3
A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
NCT05033080 COMPLETED PHASE3
A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
NCT03460990 COMPLETED PHASE3
A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
NCT03447262 TERMINATED PHASE3