Trial · NCT03525574
A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
NCT03525574, Phase 3, Completed, Vertex Pharmaceuticals Incorporated; results first posted 2024-05-08
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-445 Combination Therapy. Trials, record NCT03525574. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03525574
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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-445 Combination Therapy
- NCT number
- NCT03525574
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 507 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Cystic Fibrosis
- Interventions
- ELX/TEZ/IVA, IVA
- First posted
- 2018-05-15
- Last updated
- 2025-09-24
- Other registries' IDs
- eudract:2018-000185-11
- Results first posted
- 2024-05-08
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 196 | |
| Secondary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set | From Baseline at Week 192 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. |
| Secondary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set | From Baseline at Week 192 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. |
| Secondary | Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set | From Baseline at Week 192 | Sweat samples were collected using an approved collection device. This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. |
| Secondary | Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set | From Baseline at Week 192 | Sweat samples were collected using an approved collection device. This analysis set included study 103 parent study participants who received TEZ/IVA-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. |
| Secondary | Number of Pulmonary Exacerbations (PEx) for 102/105 Efficacy Set | From Baseline at Week 192 | 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. |
| Secondary | Number of Pulmonary Exacerbations (PEx) for 103/105 Efficacy Set | From Baseline at Week 192 | 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. |
| Secondary | Time to First PEx for 102/105 Efficacy Set | From Baseline at Week 192 | Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx 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. |
| Secondary | Time to First PEx for 103/105 Efficacy Set | From Baseline at Week 192 | Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx 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. |
| Secondary | Absolute Change in Body Mass Index (BMI) for 102/105 Efficacy Set | From Baseline at Week 192 | BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).This analysis set included study 102 parent study participants who received Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA. |
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 | Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 196 | The Open-Label Safety Set (OL-SS) included all participants who had received at least 1 dose of study drug in this open label study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set | From Baseline at Week 192 | OL-FAS is defined as all enrolled participants who received at least 1 dose of study drug in this open label study. Here Overall Number of Participants Analyzed signifies those participants who were evaluated for this outcome. Here Number Analyzed signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | percentage points | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set | From Baseline at Week 192 | OL-FAS. Here Overall Number of Participants Analyzed signifies those participants who were evaluated for this outcome. Here Number Analyzed signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | percentage points | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set | From Baseline at Week 192 | OL-FAS. Here Overall Number of Participants Analyzed signifies those participants who were evaluated for this outcome. Here Number Analyzed signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | millimole per liter (mmol/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set | From Baseline at Week 192 | OL-FAS. Here Overall Number of Participants Analyzed signifies those participants who were evaluated for this outcome. Here Number Analyzed signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | mmol/L | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Pulmonary Exacerbations (PEx) for 102/105 Efficacy Set | From Baseline at Week 192 | Cumulative TC Efficacy Set 102/105 included all participants who were randomized to TC ELX/TEZ/IVA and received at least 1 dose of study drug during the parent study 102 and/or received at least 1 dose of study drug during this open label study. Baseline was defined as the parent study baseline. | PEx events | NUMBER |
| SECONDARY | Number of Pulmonary Exacerbations (PEx) for 103/105 Efficacy Set | From Baseline at Week 192 | Cumulative TC Efficacy Set 103/105 included all participants who were randomized to TC ELX/TEZ/IVA and received at least 1 dose of study drug during the parent study 103 and/or received at least 1 dose of study drug during this open label study. Baseline was defined as the parent study baseline. | PEx events | NUMBER |
| SECONDARY | Time to First PEx for 102/105 Efficacy Set | From Baseline at Week 192 | Cumulative TC Efficacy Set 102/105 included all participants who were randomized to TC ELX/TEZ/IVA and received at least 1 dose of study drug during the parent study 102 and/or received at least 1 dose of study drug during this open label study. Baseline was defined as the parent study baseline. | days | MEDIAN |
| SECONDARY | Time to First PEx for 103/105 Efficacy Set | From Baseline at Week 192 | Cumulative TC Efficacy Set 103/105 included all participants who were randomized to TC ELX/TEZ/IVA and received at least 1 dose of study drug during the parent study 103 and/or received at least 1 dose of study drug during this open label study. Baseline was defined as the parent study baseline. | days | MEDIAN |
| SECONDARY | Absolute Change in Body Mass Index (BMI) for 102/105 Efficacy Set | From Baseline at Week 192 | OL-FAS. Here Overall Number of Participants Analyzed signifies those participants who were evaluated for this outcome. Here Number Analyzed signifies participants who were evaluable for the specified category. Baseline was defined as the parent study baseline. | kilogram per meter square (kg/m^2) | LEAST_SQUARES_MEAN |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group30
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | ELX/TEZ/IVA | Participants With TEAEs | 504 | Participants | |
| Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | ELX/TEZ/IVA | Participants With SAEs | 175 | Participants | |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set | ELX/TEZ/IVA | Placebo-ELX/TEZ/IVA | 15.3 | Standard Error 0.8 | percentage points |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set | ELX/TEZ/IVA | ELX/TEZ/IVA-ELX/TEZ/IVA | 13.8 | Standard Error 0.8 | percentage points |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set | ELX/TEZ/IVA | TEZ/IVA-ELX/TEZ/IVA | 10.9 | Standard Error 1.3 | percentage points |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set | ELX/TEZ/IVA | ELX/TEZ/IVA-ELX/TEZ/IVA | 10.7 | Standard Error 1.3 | percentage points |
| Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set | ELX/TEZ/IVA | Placebo-ELX/TEZ/IVA | -47.0 | Standard Error 1.6 | millimole per liter (mmol/L) |
| Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set | ELX/TEZ/IVA | ELX/TEZ/IVA-ELX/TEZ/IVA | -45.3 | Standard Error 1.6 | millimole per liter (mmol/L) |
| Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set | ELX/TEZ/IVA | TEZ/IVA-ELX/TEZ/IVA | -48.2 | Standard Error 3.8 | mmol/L |
| Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set | ELX/TEZ/IVA | ELX/TEZ/IVA-ELX/TEZ/IVA | -48.2 | Standard Error 3.5 | mmol/L |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | ELX/TEZ/IVA | 507 | |
| Overall Study | STARTED | Additional Participants for Cumulative TC Efficacy Set | 3 | |
| Overall Study | Open-Label Safety Set (OL-SS) | ELX/TEZ/IVA | 506 | |
| Overall Study | Open-Label Safety Set (OL-SS) | Additional Participants for Cumulative TC Efficacy Set | 0 | |
| Overall Study | OL-FAS 102/105 | ELX/TEZ/IVA | 399 | |
| Overall Study | OL-FAS 102/105 | Additional Participants for Cumulative TC Efficacy Set | 0 | |
| Overall Study | OL-FAS 103/105 | ELX/TEZ/IVA | 107 | |
| Overall Study | OL-FAS 103/105 | Additional Participants for Cumulative TC Efficacy Set | 0 | |
| Overall Study | Cumulative Triple Combination (TC) Efficacy Set 102/105 | ELX/TEZ/IVA | 400 | |
| Overall Study | Cumulative Triple Combination (TC) Efficacy Set 102/105 | Additional Participants for Cumulative TC Efficacy Set | 3 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events432
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 | Palpitations | Cardiac disorders | Treatment Period: ELX/TEZ/IVA | 11 | 506 | |
| other | Palpitations | Cardiac disorders | Extension Period: ELX/TEZ/IVA | 1 | 11 | |
| other | Abdominal distension | Gastrointestinal disorders | Treatment Period: ELX/TEZ/IVA | 31 | 506 | |
| other | Abdominal distension | Gastrointestinal disorders | Extension Period: ELX/TEZ/IVA | 0 | 11 | |
| other | Abdominal pain | Gastrointestinal disorders | Treatment Period: ELX/TEZ/IVA | 76 | 506 | |
| other | Abdominal pain | Gastrointestinal disorders | Extension Period: ELX/TEZ/IVA | 0 | 11 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Treatment Period: ELX/TEZ/IVA | 49 | 506 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Extension Period: ELX/TEZ/IVA | 0 | 11 | |
| other | Constipation | Gastrointestinal disorders | Treatment Period: ELX/TEZ/IVA | 72 | 506 | |
| other | Constipation | Gastrointestinal disorders | Extension Period: ELX/TEZ/IVA | 0 | 11 |
1 to 10 of 432Source ClinicalTrials.gov
See all 432 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Treatment Period: ELX/TEZ/IVA | 1 | 506 |
| Total, all-cause mortality | Extension Period: ELX/TEZ/IVA | 0 | 11 |
| Total, other adverse events | Treatment Period: ELX/TEZ/IVA | 501 | 506 |
| Total, other adverse events | Extension Period: ELX/TEZ/IVA | 7 | 11 |
| Total, serious adverse events | Treatment Period: ELX/TEZ/IVA | 175 | 506 |
| Total, serious adverse events | Extension Period: ELX/TEZ/IVA | 0 | 11 |
Source ClinicalTrials.gov
Papers3
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Treatment Period: ELX/TEZ/IVA vs Extension Period: ELX/TEZ/IVA: deaths from any cause | Trial comparisons |
| Treatment Period: ELX/TEZ/IVA vs Extension Period: ELX/TEZ/IVA: 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.
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