Cystic FibrosisPhase 2NCT02070744
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
A Phase 2 study testing VX-661 and Ivacaftor in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 23 sites in United States.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- VX-661 and Ivacaftor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 23 sitesUnited States
- Study dates
- Mar 2014to 27 May 2016
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
23 sites in 1 country.
23 sites in 1 country
United States23 sites
Study site
Altamonte Springs, Florida
Study site
Birmingham, Alabama
Study site
Boise, Idaho
Study site
Boston, Massachusetts
Study site
Burlington, Vermont
Study site
Charleston, South Carolina
Study site
Chicago, Illinois
Study site
Cincinnati, Ohio
Study site
Colchester, Vermont
Study site
Durham, North Carolina
Study site
Houston, Texas
Study site
Memphis, Tennessee
Study site
Miami, Florida
Study site
Milwaukee, Wisconsin
Study site
New Brunswick, New Jersey
Study site
New York, New York
Study site
Oklahoma City, Oklahoma
Study site
Orlando, Florida
Study site
Palo Alto, California
Study site
Pittsburgh, Pennsylvania
Study site
Seattle, Washington
Study site
Stanford, California
Study site
Tampa, Florida
About this study
Official title: A Phase 2, Randomized, Multicenter, Double Blind, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 in Combination With Ivacaftor for 12 Weeks in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation With an Open-Label Extension
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
- Conditions
- Cystic Fibrosis
- Being tested
- VX-661, Ivacaftor, Placebo matched to VX-661, Placebo matched to Ivacaftor
- Keywords
- CF
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VX-661Drug
Tablet, oral use
IvacaftorDrug
Film coated tablet, oral use
Placebo matched to VX-661Drug
Tablet, oral use
Placebo matched to IvacaftorDrug
Film coated tablet, oral use
| Study group | Role | What they receive |
|---|---|---|
| PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h | Experimental | Participants received VX-661 50 milligram (mg) tablet plus Ivacaftor (IVA) 150 mg tablet every 12 hours (q12h) for 12 weeks. Receives: Drug: VX-661; Drug: Ivacaftor |
| PC Phase: VX 661 placebo q12h + IVA placebo q12h | Placebo comparator | Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks. Receives: Drug: Placebo matched to VX-661; Drug: Placebo matched to Ivacaftor |
| PC Phase: VX-661 100 mg qd + IVA 150 mg q12h | Experimental | Participants received two VX-661 50 mg tablets once daily (qd) plus IVA 150 mg tablet q12h for 12 weeks. Receives: Drug: VX-661; Drug: Ivacaftor |
| PC Phase: VX -661 placebo qd + IVA placebo q12h | Placebo comparator | Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. Receives: Drug: Placebo matched to VX-661; Drug: Placebo matched to Ivacaftor |
| OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h | Experimental | 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. Receives: Drug: VX-661; Drug: Ivacaftor |
What is being measured
The primary outcome is the main question the study is designed to answer.
PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of PC Phase.
Measured: Baseline (PC Phase) up to 112 days
OLE Phase: Number of Participants With Treatment-Emergent AEs and SAEs
AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of the OLE Phase.
Measured: Baseline (OLE Phase) up to 364 days
Secondary outcomes (16)
PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of PC Phase.
Measured: Baseline (PC Phase), Through Week 12
OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 40
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of the OLE Phase.
Measured: Baseline (OLE Phase), Through Week 40
PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 12
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of PC Phase.
Measured: Baseline (PC Phase), Through Week 12
OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 40
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of the OLE Phase.
Measured: Baseline (OLE Phase), Through Week 40
PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12
Sweat samples were collected using an approved collection device. Baseline was defined as Day 1 of PC Phase.
Measured: Baseline (PC Phase), Through Week 12
OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40
Sweat samples were collected using an approved collection device. Baseline was defined as Day 1 of the OLE Phase.
Measured: Baseline (OLE Phase), Through Week 40
PC Phase: Absolute Change From Baseline in Body Weight at Week 12
Baseline was defined as Day 1 of PC Phase.
Measured: Baseline (PC Phase), Week 12
OLE Phase: Absolute Change From Baseline in Body Weight at Week 40
Baseline was defined as Day 1 of the OLE Phase.
Measured: Baseline (OLE Phase), Week 40
PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 12
BMI was calculated using following formula: BMI = Weight in kg/height in square meter (m^2). Baseline was defined as Day 1 of PC Phase.
Measured: Baseline (PC Phase), Week 12
OLE Phase: Absolute Change From Baseline BMI at Week 40
BMI was calculated using following formula: BMI = Weight in kg/height in m^2. Baseline was defined as Day 1 of the OLE Phase.
Measured: Baseline (OLE Phase), Week 40
PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Baseline was defined as Day 1 of PC Phase.
Measured: Baseline (PC Phase), Through Week 12
OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Baseline was defined as Day 1 of the OLE Phase.
Measured: Baseline (OLE Phase), Through Week 40
PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA
Measured: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661
Pharmacokinetic (PK) sampling was performed up to 12 hours post-dose on Day 85. For Arm VX-661 50 mg q12h + IVA 150 mg q12h (VX-661 q12h regimen), area under the concentration versus time curve from time 0 to 12 hours (AUC0-12h) was multiplied by 2 to obtain AUC0-24h.
Measured: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA
Measured: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA
Measured: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2014 (estimated)
- Study startMar 2014
- Primary completion27 May 2016
- Study completion27 May 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02070744
- Sponsor's protocol ID
- VX13-661-103
- Status last verified
- Sep 2025
- First submitted
- 21 Feb 2014
- Last updated
- 24 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes