Trial · NCT01807923
A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
NCT01807923, Phase 3, Completed, Vertex Pharmaceuticals Incorporated; results first posted 2015-08-31
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation. Trials, record NCT01807923. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01807923
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
- NCT number
- NCT01807923
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 559 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
- Interventions
- Lumacaftor Plus Ivacaftor Combination, Ivacaftor, Placebo
- First posted
- 2013-03-08
- Last updated
- 2015-08-31
- Results first posted
- 2015-08-31
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24 | Baseline, Week 16 and 24 | Absolute change from baseline at Week 24 was assessed as the average treatment effect at Week 16 and at Week 24. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. |
| Secondary | Relative Change From Baseline in Percent Predicted FEV1 at Week 24 | Baseline, Week 16 and 24 | Assessed as the average treatment effect at Week 16 and at Week 24. FEV1 and percent predicted FEV1 are defined in Outcome Measure (OM) 1. |
| Secondary | Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 | Baseline, Week 24 | BMI was defined as weight in kilogram (kg) divided by height\*height in square meter (m\^2). |
| Secondary | Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24 | Baseline, 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 (for example, coughing, congestion, wheezing), the scaled score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. |
| Secondary | Percentage of Participants With Response Based on Percent Predicted FEV1 | Week 16 and 24 | A participant was considered as a responder if the participant had \>=5% increase from baseline in average percent predicted FEV1 at Week 16 and at Week 24 (relative change). FEV1 and percent predicted FEV1 are defined in OM 1. A participant with a missing average relative change from baseline in percent predicted FEV1 at Week 16 and at Week 24 was considered as a non-responder. |
| Secondary | Number of Pulmonary Exacerbation Events | through Week 24 | The total number of days on study is equal to the Week 24 date or the last dose date (whichever occurred last) minus the first dose date plus 1. The total number of years (48 weeks) on study is equal to the number of days on study divided by 336. Pulmonary exacerbation events per year (48 weeks) are reported. |
| Secondary | Absolute Change From Baseline in Weight at Week 24 | Baseline, Week 24 | |
| Secondary | Absolute Change From Baseline in BMI-for-age Z-score at Week 24 | Baseline, Week 24 | Z-Score is a statistical measure to evaluate how a single data point compares to a standard. It describes whether a mean was above or below the standard and how unusual the measurement is with range from -infinity to + infinity; 0: same mean, \>0: a greater mean, and \<0: a lesser mean than the standard. BMI-for-age z-score was calculated by using Centers for Disease Control and Prevention (CDC) growth charts for the pediatric population. |
| Secondary | Time-to-First Pulmonary Exacerbation | through Week 24 | Time to first pulmonary exacerbation was assessed using Cox Regression. For participants who completed 24 weeks of treatment, participants without a pulmonary exacerbation before treatment completion were considered censored at the time of treatment completion or at the Week 24 Visit (whichever occurred last). For participants who prematurely discontinued study treatment, participants without a pulmonary exacerbation through the Week 24 Visit were considered censored at the time of the Week 24 Visit. |
| Secondary | Percentage of Participants With At Least 1 Pulmonary Exacerbation Event | through Week 24 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
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 in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24 | Baseline, Week 16 and 24 | Full Analysis Set (FAS) included all randomized participants who received any amount of study drug. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. | percent predicted of FEV1 | LEAST_SQUARES_MEAN |
| SECONDARY | Relative Change From Baseline in Percent Predicted FEV1 at Week 24 | Baseline, Week 16 and 24 | FAS. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 | Baseline, Week 24 | FAS. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. | kilogram per square meter (kg/m^2) | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24 | Baseline, Week 24 | FAS. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Participants With Response Based on Percent Predicted FEV1 | Week 16 and 24 | FAS. | percentage of participants | NUMBER |
| SECONDARY | Number of Pulmonary Exacerbation Events | through Week 24 | FAS. | pulmonary exacerbation events per year | NUMBER |
| SECONDARY | Absolute Change From Baseline in Weight at Week 24 | Baseline, Week 24 | FAS. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. | kilograms (kg) | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change From Baseline in BMI-for-age Z-score at Week 24 | Baseline, Week 24 | FAS. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Only participants who were \<20 years of age were analyzed. | z-score | LEAST_SQUARES_MEAN |
| SECONDARY | Time-to-First Pulmonary Exacerbation | through Week 24 | FAS. | days | MEDIAN |
| SECONDARY | Percentage of Participants With At Least 1 Pulmonary Exacerbation Event | through Week 24 | FAS. | percentage of participants | NUMBER |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group154
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 in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24 | Placebo | -0.44 | Standard Error 0.524 | percent predicted of FEV1 | |
| Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24 | LUM 600 mg qd/IVA 250 mg q12h | 3.59 | Standard Error 0.525 | percent predicted of FEV1 | |
| Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24 | LUM 400 mg q12h/ IVA 250 mg q12h | 2.16 | Standard Error 0.530 | percent predicted of FEV1 | |
| Relative Change From Baseline in Percent Predicted FEV1 at Week 24 | Placebo | -0.34 | Standard Error 0.913 | percent change | |
| Relative Change From Baseline in Percent Predicted FEV1 at Week 24 | LUM 600 mg qd/IVA 250 mg q12h | 6.39 | Standard Error 0.914 | percent change | |
| Relative Change From Baseline in Percent Predicted FEV1 at Week 24 | LUM 400 mg q12h/ IVA 250 mg q12h | 3.99 | Standard Error 0.923 | percent change | |
| Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 | Placebo | 0.19 | Standard Error 0.070 | kilogram per square meter (kg/m^2) | |
| Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 | LUM 600 mg qd/IVA 250 mg q12h | 0.35 | Standard Error 0.070 | kilogram per square meter (kg/m^2) | |
| Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 | LUM 400 mg q12h/ IVA 250 mg q12h | 0.32 | Standard Error 0.071 | kilogram per square meter (kg/m^2) | |
| Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24 | Placebo | 1.10 | Standard Error 1.161 | units on a scale |
1 to 10 of 154Source ClinicalTrials.gov
See all 154 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 187 | |
| Overall Study | STARTED | LUM 600 mg qd/IVA 250 mg q12h | 185 | |
| Overall Study | STARTED | LUM 400 mg q12h/ IVA 250 mg q12h | 187 | |
| Overall Study | COMPLETED | Placebo | 182 | |
| Overall Study | COMPLETED | LUM 600 mg qd/IVA 250 mg q12h | 179 | |
| Overall Study | COMPLETED | LUM 400 mg q12h/ IVA 250 mg q12h | 176 | |
| Overall Study | NOT COMPLETED | Placebo | 5 | |
| Overall Study | NOT COMPLETED | LUM 600 mg qd/IVA 250 mg q12h | 6 | |
| Overall Study | NOT COMPLETED | LUM 400 mg q12h/ IVA 250 mg q12h | 11 |
Source ClinicalTrials.gov
Adverse events1,356
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 | 58 | 184 | |
| other | Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | LUM 600 mg qd/IVA 250 mg q12h | 57 | 183 | |
| other | Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | LUM 400 mg q12h/ IVA 250 mg q12h | 54 | 182 | |
| other | Nasopharyngitis | Infections and infestations | Placebo | 20 | 184 | |
| other | Nasopharyngitis | Infections and infestations | LUM 600 mg qd/IVA 250 mg q12h | 9 | 183 | |
| other | Nasopharyngitis | Infections and infestations | LUM 400 mg q12h/ IVA 250 mg q12h | 26 | 182 | |
| other | Upper respiratory tract infection | Infections and infestations | Placebo | 10 | 184 | |
| other | Upper respiratory tract infection | Infections and infestations | LUM 600 mg qd/IVA 250 mg q12h | 16 | 183 | |
| other | Upper respiratory tract infection | Infections and infestations | LUM 400 mg q12h/ IVA 250 mg q12h | 17 | 182 | |
| other | Viral upper respiratory tract infection | Infections and infestations | Placebo | 12 | 184 |
1 to 10 of 1,356Source ClinicalTrials.gov
See all 1,356 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | ||
| Total, all-cause mortality | LUM 600 mg qd/IVA 250 mg q12h | ||
| Total, all-cause mortality | LUM 400 mg q12h/ IVA 250 mg q12h | ||
| Total, other adverse events | Placebo | 173 | 184 |
| Total, other adverse events | LUM 600 mg qd/IVA 250 mg q12h | 175 | 183 |
| Total, other adverse events | LUM 400 mg q12h/ IVA 250 mg q12h | 172 | 182 |
| Total, serious adverse events | Placebo | 49 | 184 |
| Total, serious adverse events | LUM 600 mg qd/IVA 250 mg q12h | 33 | 183 |
| Total, serious adverse events | LUM 400 mg q12h/ IVA 250 mg q12h | 33 | 182 |
Source ClinicalTrials.gov
Papers7
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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.
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