Trial · NCT02347657
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor
NCT02347657, Phase 3, Completed, Vertex Pharmaceuticals Incorporated; results first posted 2018-06-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor. Trials, record NCT02347657. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02347657
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor
- NCT number
- NCT02347657
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 510 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Cystic Fibrosis
- Interventions
- VX-661 Plus Ivacaftor Combination, Ivacaftor, VX-661 Plus Ivacaftor Combination Placebo, Ivacaftor placebo
- First posted
- 2015-01-27
- Last updated
- 2018-06-12
- Results first posted
- 2018-06-12
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 | Day 1, Through Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. |
| Secondary | Relative Change From Baseline (Day 1) in ppFEV1 Through Week 24 | Day 1, Through Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. |
| Secondary | Number of Pulmonary Exacerbations Per Year | Day 1 through Week 24 | Pulmonary exacerbation was defined as a new event or change in antibiotic therapy for greater than or equal to 4 sinopulmonary signs/symptoms. Pulmonary exacerbation events per year (48 weeks) were reported. |
| Secondary | Absolute Change From Baseline (Day 1) Body Mass Index (BMI) at Week 24 | Day 1, Week 24 | BMI was defined as weight in kilograms (kg) divided by height in square meter (m\^2). |
| Secondary | Absolute Change From Baseline (Day 1) in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 | Day 1, Through Week 24 | 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, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. |
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 28 | 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. Any AE that increased in severity or newly developed at or after initial dosing of study drug to Week 28 was considered treatment-emergent. |
| Secondary | Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 | Day 1 through Week 24 | Pulmonary exacerbation was defined as a new event or change in antibiotic therapy for greater than or equal to 4 sinopulmonary signs/symptoms. Time to event data was not collected and instead, Number of Subjects with first event were collected and are reported. Time-to-first pulmonary exacerbation was planned to be estimated using Kaplan-Meier (KM) estimates. However, due to less than 50% of events, time-to-first event data was not estimated. Instead, number of participants with at least one pulmonary exacerbation event were collected and are reported. |
| Secondary | Absolute Change From Baseline (Day 1) in Sweat Chloride Through Week 24 | Day 1, Through Week 24 | Sweat samples were collected using an approved collection device. |
| Secondary | Absolute Change From Baseline (Day 1) in BMI Z-score at Week 24 in Participants Less Than (<) 20 Years Old at the Time of Screening) | Day 1, Week 24 | BMI was defined as weight in kg divided by height in m\^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score (BMI z-score). |
| Secondary | Absolute Change From Baseline (Day 1) in Body Weight at Week 24 | Day 1, Week 24 |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 | Day 1, Through Week 24 | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | Percentage of predicted FEV1 | LEAST_SQUARES_MEAN |
| SECONDARY | Relative Change From Baseline (Day 1) in ppFEV1 Through Week 24 | Day 1, Through Week 24 | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Pulmonary Exacerbations Per Year | Day 1 through Week 24 | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. | pulmonary exacerbation events per year | NUMBER |
| SECONDARY | Absolute Change From Baseline (Day 1) Body Mass Index (BMI) at Week 24 | Day 1, Week 24 | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | kilogram per square meter (kg/m^2) | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change From Baseline (Day 1) in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 | Day 1, Through Week 24 | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 28 | Safety Set included all participants who received at least 1 dose of the study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 | Day 1 through Week 24 | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Absolute Change From Baseline (Day 1) in Sweat Chloride Through Week 24 | Day 1, Through Week 24 | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | millimole per liter (mmol/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change From Baseline (Day 1) in BMI Z-score at Week 24 in Participants Less Than (<) 20 Years Old at the Time of Screening) | Day 1, Week 24 | Analysis population included all randomized participants who received at least 1 dose of study drug and were \<20 years of age at the time of screening. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | z-score | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change From Baseline (Day 1) in Body Weight at Week 24 | Day 1, Week 24 | FAS included all randomized participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here 'Number of participants analyzed' signifies those participants who were evaluable for this outcome measure. | kg | LEAST_SQUARES_MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group27
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 | Placebo | -0.6 | 95% Confidence Interval -1.3 to 0.0 | Percentage of predicted FEV1 | |
| Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 | VX-661/IVA | 3.4 | 95% Confidence Interval 2.7 to 4.0 | Percentage of predicted FEV1 | |
| Relative Change From Baseline (Day 1) in ppFEV1 Through Week 24 | Placebo | -0.5 | 95% Confidence Interval -1.7 to 0.6 | percent change | |
| Relative Change From Baseline (Day 1) in ppFEV1 Through Week 24 | VX-661/IVA | 6.3 | 95% Confidence Interval 5.1 to 7.4 | percent change | |
| Number of Pulmonary Exacerbations Per Year | Placebo | 0.99 | pulmonary exacerbation events per year | ||
| Number of Pulmonary Exacerbations Per Year | VX-661/IVA | 0.64 | pulmonary exacerbation events per year | ||
| Absolute Change From Baseline (Day 1) Body Mass Index (BMI) at Week 24 | Placebo | 0.12 | 95% Confidence Interval 0.03 to 0.22 | kilogram per square meter (kg/m^2) | |
| Absolute Change From Baseline (Day 1) Body Mass Index (BMI) at Week 24 | VX-661/IVA | 0.18 | 95% Confidence Interval 0.08 to 0.28 | kilogram per square meter (kg/m^2) | |
| Absolute Change From Baseline (Day 1) in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 | Placebo | -0.1 | 95% Confidence Interval -1.6 to 1.4 | units on a scale | |
| Absolute Change From Baseline (Day 1) in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 | VX-661/IVA | 5.0 | 95% Confidence Interval 3.5 to 6.5 | units on a scale |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 259 | |
| Overall Study | STARTED | VX-661/IVA | 251 | |
| Overall Study | Treated | Placebo | 258 | |
| Overall Study | Treated | VX-661/IVA | 251 | |
| Overall Study | COMPLETED | Placebo | 241 | |
| Overall Study | COMPLETED | VX-661/IVA | 236 | |
| Overall Study | NOT COMPLETED | Placebo | 18 | |
| Overall Study | NOT COMPLETED | VX-661/IVA | 15 |
Source ClinicalTrials.gov
Adverse events114
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | Placebo | 75 | 258 | |
| other | Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | VX-661/IVA | 57 | 251 | |
| other | Nasopharyngitis | Infections and infestations | Placebo | 39 | 258 | |
| other | Nasopharyngitis | Infections and infestations | VX-661/IVA | 42 | 251 | |
| other | Rhinitis | Infections and infestations | Placebo | 14 | 258 | |
| other | Rhinitis | Infections and infestations | VX-661/IVA | 10 | 251 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Placebo | 83 | 258 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | VX-661/IVA | 66 | 251 | |
| other | Sputum increased | Respiratory, thoracic and mediastinal disorders | Placebo | 42 | 258 | |
| other | Sputum increased | Respiratory, thoracic and mediastinal disorders | VX-661/IVA | 36 | 251 |
1 to 10 of 114Source ClinicalTrials.gov
See all 114 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 258 |
| Total, all-cause mortality | VX-661/IVA | 0 | 251 |
| Total, other adverse events | Placebo | 243 | 258 |
| Total, other adverse events | VX-661/IVA | 227 | 251 |
| Total, serious adverse events | Placebo | 47 | 258 |
| Total, serious adverse events | VX-661/IVA | 31 | 251 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| VX-661/IVA vs Placebo: deaths from any cause | Trial comparisons |
| VX-661/IVA vs Placebo: Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 | Trial comparisons |
| VX-661/IVA vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02347657
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