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CompletedNCT02070744Updated Sep 24, 2025Results posted

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 interventional study of VX-661 and Ivacaftor in Cystic Fibrosis, sponsored by Vertex Pharmaceuticals Incorporated. Completed at 23 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-09-24.

Sponsored by Vertex Pharmaceuticals Incorporated · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
40
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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

02

Conditions studied

  • Cystic Fibrosis

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Keywords

  • CF
03

In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's enrollment of 40 is above the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

Vertex Pharmaceuticals Incorporated is the lead sponsor of 243 studies on the registry; 19 are open to participants now.

Of its 102 completed or terminated interventional studies of FDA-regulated products, 49 (48%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
40 participants (actual)

Study arms

  • Experimental
    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.

    Drug: VX-661 · Drug: Ivacaftor

  • Placebo comparator
    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.

    Drug: Placebo matched to VX-661 · Drug: Placebo matched to Ivacaftor

  • Experimental
    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.

    Drug: VX-661 · Drug: Ivacaftor

  • Placebo comparator
    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.

    Drug: Placebo matched to VX-661 · Drug: Placebo matched to Ivacaftor

  • Experimental
    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.

    Drug: VX-661 · Drug: Ivacaftor

Interventions

  • DrugVX-661

    Tablet, oral use

  • DrugIvacaftor

    Film coated tablet, oral use

  • DrugPlacebo matched to VX-661

    Tablet, oral use

  • DrugPlacebo matched to Ivacaftor

    Film coated tablet, oral use

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Baseline (PC Phase) up to 112 days

  2. 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.

    Time frame: Baseline (OLE Phase) up to 364 days

Secondary outcomes

  1. 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.

    Time frame: Baseline (PC Phase), Through Week 12

  2. 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.

    Time frame: Baseline (OLE Phase), Through Week 40

  3. 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.

    Time frame: Baseline (PC Phase), Through Week 12

  4. 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.

    Time frame: Baseline (OLE Phase), Through Week 40

  5. 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.

    Time frame: Baseline (PC Phase), Through Week 12

  6. 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.

    Time frame: Baseline (OLE Phase), Through Week 40

  7. PC Phase: Absolute Change From Baseline in Body Weight at Week 12

    Baseline was defined as Day 1 of PC Phase.

    Time frame: Baseline (PC Phase), Week 12

  8. OLE Phase: Absolute Change From Baseline in Body Weight at Week 40

    Baseline was defined as Day 1 of the OLE Phase.

    Time frame: Baseline (OLE Phase), Week 40

  9. 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.

    Time frame: Baseline (PC Phase), Week 12

  10. 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.

    Time frame: Baseline (OLE Phase), Week 40

  11. 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.

    Time frame: Baseline (PC Phase), Through Week 12

  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.

    Time frame: Baseline (OLE Phase), Through Week 40

  13. PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA

    Time frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85

  14. 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.

    Time frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85

  15. PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA

    Time frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85

  16. PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA

    Time frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85

07

Results

Posted Apr 13, 2018

Participant flow

PC Phase (12 Weeks)
Participant flow — PC Phase (12 Weeks)
MilestonePC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12hOLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Started6515140
Treated6515130
Completed6515130
Not completed00010
Withdrew: Randomized, but not treated00010
OLE Phase (48 Weeks)
Participant flow — OLE Phase (48 Weeks)
MilestonePC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12hOLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Started000027
Completed000024
Not completed00003
Withdrew: Withdrawal by subject00002
Withdrew: Other00001

Outcome measures

PrimaryPC 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.

Time frame:
Baseline (PC Phase) up to 112 days
Reported as:
Count of participants · Participants
PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
ParticipantsPC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12h
Participants with AEs651513
Participants with SAEs1245
PrimaryOLE 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.

Time frame:
Baseline (OLE Phase) up to 364 days
Reported as:
Count of participants · Participants
OLE Phase: Number of Participants With Treatment-Emergent AEs and SAEs
ParticipantsOLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Participants with AEs25
Participants with SAEs6
SecondaryPC 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.

Time frame:
Baseline (PC Phase), Through Week 12
Reported as:
Least squares mean · Percent predicted of FEV1
PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12
Percent predicted of FEV1PC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12h
PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 120.9 (-3.1 to 5.0)-0.1 (-4.4 to 4.3)3.0 (0.4 to 5.5)1.9 (-0.9 to 4.6)
SecondaryOLE 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.

Time frame:
Baseline (OLE Phase), Through Week 40
Reported as:
Least squares mean · Percent predicted of FEV1
OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 40
Percent predicted of FEV1OLE Phase:VX-661 100 mg qd + IVA 150 mg q12h
OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 402.7 (0.4 to 4.9)
SecondaryPC 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.

Time frame:
Baseline (PC Phase), Through Week 12
Reported as:
Least squares mean · Percent change
PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 12
Percent changePC Phase: VX-661 50 mg + IVA 150 mgPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12h
PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 122.6 (-6.0 to 11.1)1.0 (-8.2 to 10.3)6.0 (0.6 to 11.3)4.2 (-1.5 to 10.0)
SecondaryOLE 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.

Time frame:
Baseline (OLE Phase), Through Week 40
Reported as:
Least squares mean · Percent change
OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 40
Percent changeOLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 406.1 (1.7 to 10.5)
SecondaryPC 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.

Time frame:
Baseline (PC Phase), Through Week 12
Reported as:
Least squares mean · Millimole per liter (mmol/L)
PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12
Millimole per liter (mmol/L)PC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12h
PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12-10.6 (-16.6 to -4.6)2.9 (-3.0 to 8.9)-4.7 (-8.4 to -0.9)0.8 (-3.1 to 4.7)
SecondaryOLE 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.

Time frame:
Baseline (OLE Phase), Through Week 40
Reported as:
Least squares mean · mmol/L
OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40
mmol/LOLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40-6.6 (-9.7 to -3.5)
SecondaryPC Phase: Absolute Change From Baseline in Body Weight at Week 12

Baseline was defined as Day 1 of PC Phase.

Time frame:
Baseline (PC Phase), Week 12
Reported as:
Least squares mean · kilogram (kg)
PC Phase: Absolute Change From Baseline in Body Weight at Week 12
kilogram (kg)PC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12h
PC Phase: Absolute Change From Baseline in Body Weight at Week 121.0 (-0.4 to 2.3)1.6 (0.1 to 3.0)0.5 (-0.3 to 1.4)0.3 (-0.6 to 1.3)
SecondaryOLE Phase: Absolute Change From Baseline in Body Weight at Week 40

Baseline was defined as Day 1 of the OLE Phase.

Time frame:
Baseline (OLE Phase), Week 40
Reported as:
Least squares mean · kg
OLE Phase: Absolute Change From Baseline in Body Weight at Week 40
kgOLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
OLE Phase: Absolute Change From Baseline in Body Weight at Week 401.0 (-0.2 to 2.2)
SecondaryPC 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.

Time frame:
Baseline (PC Phase), Week 12
Reported as:
Least squares mean · Kilogram per square meter (kg/m^2)
PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 12
Kilogram per square meter (kg/m^2)PC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12h
PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 120.38 (-0.08 to 0.84)0.54 (0.03 to 1.04)0.18 (-0.11 to 0.47)0.11 (-0.21 to 0.42)
SecondaryOLE 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.

Time frame:
Baseline (OLE Phase), Week 40
Reported as:
Least squares mean · kg/m^2
OLE Phase: Absolute Change From Baseline BMI at Week 40
kg/m^2OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
OLE Phase: Absolute Change From Baseline BMI at Week 400.33 (-0.09 to 0.74)
SecondaryPC 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.

Time frame:
Baseline (PC Phase), Through Week 12
Reported as:
Least squares mean · units on a scale
PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12
units on a scalePC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12h
PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 125.6 (-3.9 to 15.1)-4.6 (-14.9 to 5.7)1.0 (-5.1 to 7.0)0.4 (-6.2 to 7.1)
SecondaryOLE 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.

Time frame:
Baseline (OLE Phase), Through Week 40
Reported as:
Least squares mean · units on a scale
OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40
units on a scaleOLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40-0.6 (-5.8 to 4.5)
SecondaryPC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA
Time frame:
Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
Reported as:
Mean · Nanogram per milliliter (ng/mL)
PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA
Nanogram per milliliter (ng/mL)PC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12h
VX-6614890 ± 31506460 ± 1240
IVA1490 ± 11501210 ± 585
SecondaryPC 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.

Time frame:
Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
Reported as:
Mean · Hour*nanogram per milliliter (hr*ng/mL)
PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661
Hour*nanogram per milliliter (hr*ng/mL)PC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12h
PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-66184900 ± 5590075500 ± 20300
SecondaryPC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA
Time frame:
Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
Reported as:
Mean · hr*ng/mL
PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA
hr*ng/mLPC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12h
PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA14700 ± 1160010100 ± 4890
SecondaryPC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA
Time frame:
Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
Reported as:
Median · hour
PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA
hourPC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12h
VX-6612.48 (1.75 to 4.08)3.23 (1.92 to 6.03)
IVA3.59 (1.98 to 6.00)4.00 (2.98 to 6.03)

Adverse events

Collected over Baseline (Day 1 of PC Phase) up to 112 days (Placebo control phase); Baseline (Day 1 of OLE Phase) up to 364 days (OLE phase). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h0/6 (0%)1/6 (16.7%)6/6 (100%)
PC Phase: VX 661 Placebo q12h + IVA Placebo q12h0/5 (0%)2/5 (40%)5/5 (100%)
PC Phase: VX-661 100 mg qd + IVA 150 mg q12h0/15 (0%)4/15 (26.7%)15/15 (100%)
PC Phase: VX -661 Placebo qd + IVA Placebo q12h0/13 (0%)5/13 (38.5%)13/13 (100%)
OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h0/27 (0%)6/27 (22.2%)25/27 (92.6%)
Most frequent serious events
Most frequent serious events
EventPC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX 661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12hOLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations0/62/54/154/135/27
Chronic sinusitisInfections and infestations1/60/50/150/130/27
PneumoniaInfections and infestations0/60/50/151/130/27
Intestinal obstructionGastrointestinal disorders0/60/50/151/130/27
PneumothoraxRespiratory, thoracic and mediastinal disorders0/60/50/151/130/27
Cystic fibrosis related diabetesCongenital, familial and genetic disorders0/60/50/150/131/27
DehydrationMetabolism and nutrition disorders0/60/50/150/131/27
NephrolithiasisRenal and urinary disorders0/60/50/150/131/27
Most frequent other events
Showing 10 of 99
Most frequent other events
EventPC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX 661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12hOLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations1/63/55/154/1313/27
CoughRespiratory, thoracic and mediastinal disorders3/62/54/155/1310/27
DyspnoeaRespiratory, thoracic and mediastinal disorders1/62/52/151/133/27
Sputum increasedRespiratory, thoracic and mediastinal disorders0/62/51/152/134/27
HaemoptysisRespiratory, thoracic and mediastinal disorders0/60/53/153/133/27
Nasal congestionRespiratory, thoracic and mediastinal disorders1/60/51/153/132/27
Respiration abnormalRespiratory, thoracic and mediastinal disorders1/61/52/150/133/27
Sputum discolouredRespiratory, thoracic and mediastinal disorders0/61/51/150/130/27
RalesRespiratory, thoracic and mediastinal disorders0/61/50/151/130/27
Pulmonary function test decreasedInvestigations1/61/52/151/130/27

Baseline characteristics

Full Analysis Set (FAS) was defined as all randomized participants who received at least 1 dose of study drug in PC Phase.

Age, Continuous
Age, Continuous(years)PC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12hTotal
Mean33.0 ± 10.624.2 ± 2.227.3 ± 5.230.3 ± 10.828.8 ± 8.3
Sex: Female, Male
Sex: Female, Male(Participants)PC Phase: VX-661 50 mg q12h + IVA 150 mg q12hPC Phase: VX-661 Placebo q12h + IVA Placebo q12hPC Phase: VX-661 100 mg qd + IVA 150 mg q12hPC Phase: VX -661 Placebo qd + IVA Placebo q12hTotal
Female227314
Male4381025
08

Study locations

23 sites
  • Birmingham, Alabama, United States
  • Palo Alto, California, United States
  • Stanford, California, United States
  • Altamonte Springs, Florida, United States
  • Miami, Florida, United States
  • Orlando, Florida, United States
  • Tampa, Florida, United States
  • Boise, Idaho, United States
  • Chicago, Illinois, United States
  • Boston, Massachusetts, United States
  • New Brunswick, New Jersey, United States
  • New York, New York, United States
  • Durham, North Carolina, United States
  • Cincinnati, Ohio, United States
  • Oklahoma City, Oklahoma, United States
  • Pittsburgh, Pennsylvania, United States
  • Charleston, South Carolina, United States
  • Memphis, Tennessee, United States
  • Houston, Texas, United States
  • Burlington, Vermont, United States
  • Colchester, Vermont, United States
  • Seattle, Washington, United States
  • Milwaukee, Wisconsin, United States
09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 24, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT02070744
Lead sponsor
Vertex Pharmaceuticals Incorporated
Responsible party
Sponsor
First posted
Feb 25, 2014
Start date
Mar 2014
Primary completion
May 27, 2016
Completion
May 27, 2016
Results posted
Apr 13, 2018
Last update
Sep 24, 2025

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.

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