Trial · NCT03447262
A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
NCT03447262, Phase 3, Terminated, Vertex Pharmaceuticals Incorporated; results first posted 2022-01-25
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy. Trials, record NCT03447262. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03447262
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
- NCT number
- NCT03447262
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 484 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Cystic Fibrosis
- Interventions
- VX-659/TEZ/IVA, IVA
- First posted
- 2018-02-27
- Last updated
- 2022-01-25
- Other registries' IDs
- eudract:2017-004134-29
- Results first posted
- 2022-01-25
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100) | |
| Secondary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102 | From Baseline at Week 72 (Study 659-105) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. |
| Secondary | Absolute Change in ppFEV1 for Participants From the Parent Study 659-103 | From Baseline at Week 72 (Study 659-105) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. |
| Secondary | Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102 | From Baseline at Week 24 (Study 659-105) | Sweat samples were collected using an approved collection device. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. |
| Secondary | Absolute Change in SwCl for Participants From the Parent Study 659-103 | From Baseline at Week 24 (Study 659-105) | Sweat samples were collected using an approved collection device. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. |
| Secondary | Number of Pulmonary Exacerbations (PEx) for Participants From the Parent Study 659-102 | From Baseline up to Week 96 (Study 659-105) | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-102 or/and VX-659/TEZ/IVA in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for Placebo - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. |
| Secondary | Number of PEx for Participants From the Parent Study 659-103 | From Baseline up to Week 96 (Study 659-105) | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported for overall participants from the parent study 659-103, that is combined for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. |
| Secondary | Number of Participants With at Least One PEx for Participants From the Parent Study 659-102 | From Baseline up to Week 96 (Study 659-105) | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the Kaplan-Meier (KM) method. However, because way less than 50% of participants had events, median time-to-first event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported separately for those in Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-102 or/and VX-659/TEZ/IVA in the current study 659-105). Baseline was defined as the parent study baseline except for Placebo - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. |
| Secondary | Number of Participants With at Least One PEx for Participants From the Parent Study 659-103 | From Baseline up to Week 96 (Study 659-105) | PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the KM method. However, because way less than 50% of participants had events, median time-to-first-event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported for all participants from the parent study 659-103, that is combined for those in the TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105). Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline. |
| Secondary | Absolute Change in Body Mass Index (BMI) for Participants From the Parent Study 659-102 | From Baseline at Week 72 (Study 659-105) | BMI was defined as weight in kilograms (kg) divided by squared height in meters (m\^2). The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100) | Open label safety set (OL-SS) included all participants who received at least 1 dose of study drug in the current study 659-105. | participants | NUMBER |
| SECONDARY | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102 | From Baseline at Week 72 (Study 659-105) | Open label full analysis set (OL-FAS) included all rolled over participants from the parent study 659-102 who received at least 1 dose of study drug in the current study 659-105. Here, number analyzed signifies participants who were evaluable for the specified category. | percentage points | MEAN |
| SECONDARY | Absolute Change in ppFEV1 for Participants From the Parent Study 659-103 | From Baseline at Week 72 (Study 659-105) | OL-FAS included all rolled over participants from the parent study 659-103 who received at least 1 dose of study drug in the current study 659-105. Here, number analyzed signifies participants who were evaluable for the specified category. | percentage points | MEAN |
| SECONDARY | Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102 | From Baseline at Week 24 (Study 659-105) | OL-FAS included all rolled over participants from the parent study 659-102 who received at least 1 dose of study drug in the current study 659-105. Here, number analyzed signifies participants who were evaluable for the specified category. | millimole per liter (mmol/L) | MEAN |
| SECONDARY | Absolute Change in SwCl for Participants From the Parent Study 659-103 | From Baseline at Week 24 (Study 659-105) | OL-FAS included all rolled over participants from the parent study 659-103 who received at least 1 dose of study drug in the current study 659-105. Here, number analyzed signifies participants who were evaluable for the specified category. | mmol/L | MEAN |
| SECONDARY | Number of Pulmonary Exacerbations (PEx) for Participants From the Parent Study 659-102 | From Baseline up to Week 96 (Study 659-105) | The cumulative TC efficacy set for PEx analysis included all participants who were randomized to VX-659/TEZ/IVA arm and received at least one dose of study drug during the parent study 659-102 and/or received at least one dose of study drug during the current study 659-105. Here, number analyzed signifies participants who were evaluable for the specified category. | PEx events | NUMBER |
| SECONDARY | Number of PEx for Participants From the Parent Study 659-103 | From Baseline up to Week 96 (Study 659-105) | The cumulative TC efficacy set for PEx analysis included all participants who were randomized to VX-659/TEZ/IVA arm and received at least one dose of study drug during the parent study 659-103 and/or received at least one dose of study drug during the current study 659-105. | PEx events | NUMBER |
| SECONDARY | Number of Participants With at Least One PEx for Participants From the Parent Study 659-102 | From Baseline up to Week 96 (Study 659-105) | The cumulative TC efficacy set for PEx analysis included all participants who were randomized to VX-659/TEZ/IVA arm and received at least one dose of study drug during the parent study 659-102 and/or received at least one dose of study drug during the current study 659-105. Here, number analyzed signifies participants who were evaluable for the specified category. | participants | NUMBER |
| SECONDARY | Number of Participants With at Least One PEx for Participants From the Parent Study 659-103 | From Baseline up to Week 96 (Study 659-105) | The cumulative TC efficacy set for PEx analysis included all participants who were randomized to VX-659/TEZ/IVA arm and received at least one dose of study drug during the parent study 659-103 and/or received at least one dose of study drug during the current study 659-105. | participants | NUMBER |
| SECONDARY | Absolute Change in Body Mass Index (BMI) for Participants From the Parent Study 659-102 | From Baseline at Week 72 (Study 659-105) | OL-FAS included all rolled over participants from the parent study 659-102 who received at least 1 dose of study drug in the current study 659-105. Here, number analyzed signifies participants who were evaluable for the specified category. | kilogram per meter square (kg/m^2) | MEAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group32
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | VX-659/TEZ/IVA TC | Participants With AEs | 470 | participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | VX-659/TEZ/IVA TC | Participants With SAEs | 99 | participants | |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102 | VX-659/TEZ/IVA TC | Placebo - VX-659/TEZ/IVA | 14.2 | Standard Deviation 7.8 | percentage points |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102 | VX-659/TEZ/IVA TC | VX-659/TEZ/IVA - VX-659/TEZ/IVA | 10.3 | Standard Deviation 9.3 | percentage points |
| Absolute Change in ppFEV1 for Participants From the Parent Study 659-103 | VX-659/TEZ/IVA TC | TEZ/IVA - VX-659/TEZ/IVA | 15.1 | Standard Deviation 11.9 | percentage points |
| Absolute Change in ppFEV1 for Participants From the Parent Study 659-103 | VX-659/TEZ/IVA TC | VX-659/TEZ/IVA - VX-659/TEZ/IVA | 11.5 | Standard Deviation 9.8 | percentage points |
| Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102 | VX-659/TEZ/IVA TC | Placebo - VX-659/TEZ/IVA | -48.9 | Standard Deviation 20.4 | millimole per liter (mmol/L) |
| Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102 | VX-659/TEZ/IVA TC | VX-659/TEZ/IVA - VX-659/TEZ/IVA | -49.7 | Standard Deviation 20.1 | millimole per liter (mmol/L) |
| Absolute Change in SwCl for Participants From the Parent Study 659-103 | VX-659/TEZ/IVA TC | TEZ/IVA - VX-659/TEZ/IVA | -45.7 | Standard Deviation 16.8 | mmol/L |
| Absolute Change in SwCl for Participants From the Parent Study 659-103 | VX-659/TEZ/IVA TC | VX-659/TEZ/IVA - VX-659/TEZ/IVA | -53.5 | Standard Deviation 16.2 | mmol/L |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | VX-659/TEZ/IVA Triple Combination (TC) | 481 | |
| Overall Study | COMPLETED | VX-659/TEZ/IVA Triple Combination (TC) | 2 | |
| Overall Study | NOT COMPLETED | VX-659/TEZ/IVA Triple Combination (TC) | 479 |
Source ClinicalTrials.gov
Adverse events94
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 | Abdominal pain | Gastrointestinal disorders | VX-659/TEZ/IVA TC | 42 | 481 | |
| other | Abdominal pain upper | Gastrointestinal disorders | VX-659/TEZ/IVA TC | 26 | 481 | |
| other | Diarrhoea | Gastrointestinal disorders | VX-659/TEZ/IVA TC | 42 | 481 | |
| other | Nausea | Gastrointestinal disorders | VX-659/TEZ/IVA TC | 36 | 481 | |
| other | Vomiting | Gastrointestinal disorders | VX-659/TEZ/IVA TC | 27 | 481 | |
| other | Fatigue | General disorders | VX-659/TEZ/IVA TC | 31 | 481 | |
| other | Pyrexia | General disorders | VX-659/TEZ/IVA TC | 56 | 481 | |
| other | Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | VX-659/TEZ/IVA TC | 149 | 481 | |
| other | Influenza | Infections and infestations | VX-659/TEZ/IVA TC | 40 | 481 | |
| other | Nasopharyngitis | Infections and infestations | VX-659/TEZ/IVA TC | 97 | 481 |
1 to 10 of 94Source ClinicalTrials.gov
See all 94 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | VX-659/TEZ/IVA TC | 2 | 481 |
| Total, other adverse events | VX-659/TEZ/IVA TC | 446 | 481 |
| Total, serious adverse events | VX-659/TEZ/IVA TC | 99 | 481 |
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 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.
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/NCT03447262
GET https://conductscience.org/api/trials/v1/NCT03447262/results?page=2