Trial · NCT01931839
A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis
NCT01931839, Phase 3, Completed, Vertex Pharmaceuticals Incorporated; results first posted 2017-05-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis. Trials, record NCT01931839. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01931839
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis
- NCT number
- NCT01931839
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 1,164 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
- Interventions
- Lumacaftor Plus Ivacaftor Combination, Ivacaftor
- First posted
- 2013-08-29
- Last updated
- 2017-05-12
- Results first posted
- 2017-05-12
- Limitations and caveats
- Analysis using baseline values of the current study 105 (NCT01931839) was not performed for Part B Treatment Cohort.
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 105 (Study 105) | AE: as 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 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, In-patient 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 was considered treatment-emergent. |
| Primary | Part B Treatment Cohort: Number of Participants With Treatment-Emergent AEs and SAEs | Day 1 up to Week 105 (Study 105) | AE: as 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 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, In-patient 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 was considered treatment-emergent. |
| Secondary | Part A Treatment Cohort: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) At Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 103/104/105); Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | 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. Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4. |
| Secondary | Part B Treatment Cohort: Absolute Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | 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. Analysis was performed using baseline of Cohort 4 of previous study VX09-809-102 (NCT01225211) for Arm 6 and 7. |
| Secondary | Part A Treatment Cohort: Relative Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | 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. Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4. |
| Secondary | Part B Treatment Cohort: Relative Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | 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. Analysis was performed using baseline of Cohort 4 of previous study VX09-809-102 (NCT01225211) for Arm 6 and 7. |
| Secondary | Part A Treatment Cohort: Absolute Change From Baseline in Body Mass Index (BMI) at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | BMI = (Weight in kilogram \[kg\]) divided by (Stature in meters \[m\]) \^2. Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4. |
| Secondary | Part B Treatment Cohort: Absolute Change From Baseline in BMI at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | BMI = (Weight \[in kg\]) divided by (Stature \[in meters\]) \^2. Analysis was performed using baseline of Cohort 4 of previous study VX09-809-102 (NCT01225211) for Arm 6 and 7. |
| Secondary | Part A Treatment Cohort: Number of Pulmonary Exacerbations Events Per Patient-Year | Baseline (Study 103/104) up to Week 100 (Study 105) for Arm 1 and 3 (Cumulative study period); Baseline (Study 105) up to Week 100 (Study 105) for Arm 2 and 4 (current study period) | Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. The number of events per patient year were reported, where patient years = total number of days on study/336. Analysis includes all events in the Cumulative Study Period for Arm 1 and 3, and all events in the Current Study period (Study 105) for Arm 2 and 4. As per planned analysis, endpoint evaluation included subjects from the parent study VX12-809-103 and VX12-809-104 as well for cumulative study period. |
| Secondary | Part A Treatment Cohort: Absolute Change From Baseline in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score at Day 15, Week 8, 16, 24, 48 and 72 | Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 48, 72 (Study 105) | 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. Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4. |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 105 (Study 105) | Safety Set (study 105) included all participants in Treatment Cohort Part A who were exposed to any amount of study drug. | participants | NUMBER |
| PRIMARY | Part B Treatment Cohort: Number of Participants With Treatment-Emergent AEs and SAEs | Day 1 up to Week 105 (Study 105) | Safety Set (study 105) included all participants in the Treatment Cohort Part B who were exposed to any amount of study drug. | participants | NUMBER |
| SECONDARY | Part A Treatment Cohort: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) At Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 103/104/105); Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | Full Analysis Set (FAS) study 105 (NCT01931839) included all participants randomized in the Part A Treatment Cohort and dosed. Here, 'Number of participants analyzed' = those participants who were evaluable for this endpoint and 'Number Analyzed' = those participants who were evaluable at the specified time points for each arm, respectively. | percent predicted of FEV1 | LEAST_SQUARES_MEAN |
| SECONDARY | Part B Treatment Cohort: Absolute Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | FAS (study 105) included all participants randomized in the Part B Treatment Cohort and dosed. Here, 'Number of participants analyzed' = those participants who were evaluable for this endpoint and 'Number Analyzed' = those participants who were evaluable at the specified time points for each arm, respectively. | percent predicted of FEV1 | MEAN |
| SECONDARY | Part A Treatment Cohort: Relative Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | FAS study 105 (NCT01931839) included all participants randomized in the Part A Treatment Cohort and dosed. Here, 'Number of participants analyzed' = those participants who were evaluable for this endpoint and 'Number Analyzed' = those participants who were evaluable at the specified time points for each arm, respectively. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Part B Treatment Cohort: Relative Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | FAS (study 105) included all participants randomized in the Part B Treatment Cohort and dosed. Here, 'Number of participants analyzed' = those participants who were evaluable for this endpoint and 'Number Analyzed' = those participants who were evaluable at the specified time points for each arm, respectively. | percent change | MEAN |
| SECONDARY | Part A Treatment Cohort: Absolute Change From Baseline in Body Mass Index (BMI) at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | FAS study 105 (NCT01931839) included all participants randomized in the Part A Treatment Cohort and dosed. Here, 'Number of participants analyzed' = those participants who were evaluable for this endpoint and 'Number Analyzed' = those participants who were evaluable at the specified time points for each arm, respectively. | Kilogram per square meter (kg/m^2) | LEAST_SQUARES_MEAN |
| SECONDARY | Part B Treatment Cohort: Absolute Change From Baseline in BMI at Day 15, Week 8, 16, 24, 36, 48, 60 and 72 | Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105) | FAS (study 105) included all participants randomized in the Part B Treatment Cohort and dosed. Here, 'Number Analyzed' = those participants who were evaluable at the specified time points for each arm, respectively. | kg/m^2 | MEAN |
| SECONDARY | Part A Treatment Cohort: Number of Pulmonary Exacerbations Events Per Patient-Year | Baseline (Study 103/104) up to Week 100 (Study 105) for Arm 1 and 3 (Cumulative study period); Baseline (Study 105) up to Week 100 (Study 105) for Arm 2 and 4 (current study period) | FAS (Study 103/104) was used for Arm 1 \& 3, \& included all participants randomized in previous studies \& dosed. FAS (Study 105) was used for Arm 2 \& 4, \& included all participants randomized in Part A Treatment Cohort \& dosed in current study 105. 'Number Analyzed'=those participants who were evaluable at specified time points for each arm. | events per patient year | NUMBER |
| SECONDARY | Part A Treatment Cohort: Absolute Change From Baseline in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score at Day 15, Week 8, 16, 24, 48 and 72 | Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 48, 72 (Study 105) | FAS study 105 (NCT01931839) included all participants randomized in the Part A Treatment Cohort and dosed. Here, 'Number of participants analyzed' = those participants who were evaluable for this endpoint and 'Number Analyzed' = those participants who were evaluable at the specified time points for each arm, respectively. | units on a scale | LEAST_SQUARES_MEAN |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group385
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Arm 1: Part A - LUM 600 mg qd/ IVA 250 mg q12h | Participants with any AEs | 331 | participants | |
| Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Arm 2: Part A - Placebo- LUM 600 mg qd/ IVA 250 mg q12h | Participants with any AEs | 177 | participants | |
| Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Arm 3: Part A - LUM 400 mg q12h/ IVA 250 mg q12h | Participants with any AEs | 333 | participants | |
| Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Arm 4: Part A - Placebo- LUM 400 mg q12h/ IVA 250 mg q12h | Participants with any AEs | 176 | participants | |
| Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Arm 1: Part A - LUM 600 mg qd/ IVA 250 mg q12h | Participants with SAEs | 156 | participants | |
| Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Arm 2: Part A - Placebo- LUM 600 mg qd/ IVA 250 mg q12h | Participants with SAEs | 77 | participants | |
| Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Arm 3: Part A - LUM 400 mg q12h/ IVA 250 mg q12h | Participants with SAEs | 143 | participants | |
| Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Arm 4: Part A - Placebo- LUM 400 mg q12h/ IVA 250 mg q12h | Participants with SAEs | 89 | participants | |
| Part B Treatment Cohort: Number of Participants With Treatment-Emergent AEs and SAEs | Arm 6: Part B LUM 400 mg q12h/ IVA 250 mg q12h | Participants with AEs | 52 | participants | |
| Part B Treatment Cohort: Number of Participants With Treatment-Emergent AEs and SAEs | Arm 7: Part B Placebo- LUM 400 mg q12h/ IVA 250 mg q12h | Participants with AEs | 57 | participants |
1 to 10 of 385Source ClinicalTrials.gov
See all 385 results by groupParticipant flow35
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Arm 1 Part A: LUM 600 mg qd/ IVA 250 mg q12h | 335 | |
| Overall Study | STARTED | Arm 2 Part A: Placebo - LUM 600 mg qd/ IVA 250 mg q12h | 178 | |
| Overall Study | STARTED | Arm 3 Part A: LUM 400 mg q12h/ IVA 250 mg q12h | 340 | |
| Overall Study | STARTED | Arm 4 Part A: Placebo - LUM 400 mg q12h/ IVA 250 mg q12h | 176 | |
| Overall Study | STARTED | Arm 5 Part A: Observational Cohort | 19 | |
| Overall Study | STARTED | Arm 6 Part B: LUM 400 mg q12h/ IVA 250 mg q12h | 55 | |
| Overall Study | STARTED | Arm 7 Part B: Placebo - LUM 400 mg q12h/ IVA 250 mg q12h | 60 | |
| Overall Study | Full Analysis Set (As Randomized) | Arm 1 Part A: LUM 600 mg qd/ IVA 250 mg q12h | 334 | |
| Overall Study | Full Analysis Set (As Randomized) | Arm 2 Part A: Placebo - LUM 600 mg qd/ IVA 250 mg q12h | 179 | |
| Overall Study | Full Analysis Set (As Randomized) | Arm 3 Part A: LUM 400 mg q12h/ IVA 250 mg q12h | 340 |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 participant flowAdverse events7,364
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 | Contusion | Injury, poisoning and procedural complications | Arm 1 Part A: LUM 600 mg qd/ IVA 250 mg q12h | 7 | 335 | |
| other | Contusion | Injury, poisoning and procedural complications | Arm 2 Part A: Placebo - LUM 600 mg qd/ IVA 250 mg q12h | 3 | 178 | |
| other | Contusion | Injury, poisoning and procedural complications | Arm 3 Part A: LUM 400 mg q12h/ IVA 250 mg q12h | 8 | 340 | |
| other | Contusion | Injury, poisoning and procedural complications | Arm 4 Part A: Placebo - LUM 400 mg q12h/ IVA 250 mg q12h | 3 | 176 | |
| other | Contusion | Injury, poisoning and procedural complications | Arm 5 Part A: Observational Cohort | 0 | 0 | |
| other | Contusion | Injury, poisoning and procedural complications | Arm 6 Part B: LUM 400 mg q12h/ IVA 250 mg q12h | 0 | 55 | |
| other | Contusion | Injury, poisoning and procedural complications | Arm 7 Part B: Placebo - LUM 400 mg q12h/ IVA 250 mg q12h | 0 | 60 | |
| other | Procedural pain | Injury, poisoning and procedural complications | Arm 1 Part A: LUM 600 mg qd/ IVA 250 mg q12h | 10 | 335 | |
| other | Procedural pain | Injury, poisoning and procedural complications | Arm 2 Part A: Placebo - LUM 600 mg qd/ IVA 250 mg q12h | 3 | 178 | |
| other | Procedural pain | Injury, poisoning and procedural complications | Arm 3 Part A: LUM 400 mg q12h/ IVA 250 mg q12h | 8 | 340 |
1 to 10 of 7,364Source ClinicalTrials.gov
See all 7,364 adverse eventsAdverse event totals21
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arm 1 Part A: LUM 600 mg qd/ IVA 250 mg q12h | ||
| Total, all-cause mortality | Arm 2 Part A: Placebo - LUM 600 mg qd/ IVA 250 mg q12h | ||
| Total, all-cause mortality | Arm 3 Part A: LUM 400 mg q12h/ IVA 250 mg q12h | ||
| Total, all-cause mortality | Arm 4 Part A: Placebo - LUM 400 mg q12h/ IVA 250 mg q12h | ||
| Total, all-cause mortality | Arm 5 Part A: Observational Cohort | ||
| Total, all-cause mortality | Arm 6 Part B: LUM 400 mg q12h/ IVA 250 mg q12h | ||
| Total, all-cause mortality | Arm 7 Part B: Placebo - LUM 400 mg q12h/ IVA 250 mg q12h | ||
| Total, other adverse events | Arm 1 Part A: LUM 600 mg qd/ IVA 250 mg q12h | 328 | 335 |
| Total, other adverse events | Arm 2 Part A: Placebo - LUM 600 mg qd/ IVA 250 mg q12h | 177 | 178 |
| Total, other adverse events | Arm 3 Part A: LUM 400 mg q12h/ IVA 250 mg q12h | 332 | 340 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse event totalsPapers3
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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GET https://conductscience.org/api/trials/v1/NCT01931839/results?page=2