Trial · NCT04058366
Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
NCT04058366, Phase 3, Completed, Vertex Pharmaceuticals Incorporated; results first posted 2024-01-16
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy. Trials, record NCT04058366. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04058366
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
- NCT number
- NCT04058366
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 251 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Cystic Fibrosis
- Interventions
- ELX/TEZ/IVA, IVA
- First posted
- 2019-08-15
- Last updated
- 2024-01-16
- Other registries' IDs
- eudract:2019-000833-37
- Results first posted
- 2024-01-16
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Baseline up to Week 100 | |
| Secondary | Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | From Baseline at Week 96 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. |
| Secondary | Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) | From Baseline at Week 96 | Sweat samples were collected using an approved collection device. |
| Secondary | Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI) | From Baseline at Week 96 | BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2). |
| Secondary | Part A: Absolute Change From Parent Study Baseline in BMI Z-score | From Baseline at Week 96 | BMI was defined as weight in kilogram (kg) divided by squared height in meters (m\^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. |
| Secondary | Part A: Absolute Change From Parent Study Baseline in Body Weight | From Baseline at Week 96 | |
| Secondary | Part A: Absolute Change From Parent Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score | From Baseline at Week 96 | 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. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Baseline up to Week 100 | The open label (OL) Safety Set for Part A was defined as all participants who received at least 1 dose of study drug in the OLS Part A (participants were assigned based on parent study actual treatment). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | From Baseline at Week 96 | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. 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. | percentage points | LEAST_SQUARES_MEAN |
| SECONDARY | Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) | From Baseline at Week 96 | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. 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. | millimole per liter (mmol/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI) | From Baseline at Week 96 | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. 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. | kg/m^2 | LEAST_SQUARES_MEAN |
| SECONDARY | Part A: Absolute Change From Parent Study Baseline in BMI Z-score | From Baseline at Week 96 | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. 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. | z-score | LEAST_SQUARES_MEAN |
| SECONDARY | Part A: Absolute Change From Parent Study Baseline in Body Weight | From Baseline at Week 96 | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled subjects who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. 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. | Kilogram (Kg) | LEAST_SQUARES_MEAN |
| SECONDARY | Part A: Absolute Change From Parent Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score | From Baseline at Week 96 | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. 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. | units on a scale | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Part A: ELX/TEZ/IVA | Participants with TEAEs | 241 | Participants | |
| Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Part A: ELX/TEZ/IVA | Participants with SAEs | 38 | Participants | |
| Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | ELX/TEZ/IVA | Control-IVA or TEZ/IVA: Change at Week 96 | 4.1 | 95% Confidence Interval 2.5 to 5.7 | percentage points |
| Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | ELX/TEZ/IVA | ELX/TEZ/IVA: Change at Week 96 | 3.7 | 95% Confidence Interval 2.2 to 5.2 | percentage points |
| Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) | ELX/TEZ/IVA | Control-IVA or TEZ/IVA: Change at Week 96 | -23.0 | 95% Confidence Interval -25.8 to -20.1 | millimole per liter (mmol/L) |
| Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) | ELX/TEZ/IVA | ELX/TEZ/IVA: Change at Week 96 | -22.6 | 95% Confidence Interval -25.4 to -19.9 | millimole per liter (mmol/L) |
| Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI) | ELX/TEZ/IVA | Control-IVA or TEZ/IVA: Change at Week 96 | 1.15 | 95% Confidence Interval 0.84 to 1.45 | kg/m^2 |
| Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI) | ELX/TEZ/IVA | ELX/TEZ/IVA: Change at Week 96 | 0.83 | 95% Confidence Interval 0.54 to 1.11 | kg/m^2 |
| Part A: Absolute Change From Parent Study Baseline in BMI Z-score | ELX/TEZ/IVA | Control-IVA or TEZ/IVA: Change at Week 96 | 0.11 | 95% Confidence Interval -0.17 to 0.40 | z-score |
| Part A: Absolute Change From Parent Study Baseline in BMI Z-score | ELX/TEZ/IVA | ELX/TEZ/IVA: Change at Week 96 | 0.40 | 95% Confidence Interval 0.17 to 0.62 | z-score |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | ELX/TEZ/IVA | 251 | |
| Overall Study | COMPLETED | ELX/TEZ/IVA | 215 | |
| Overall Study | NOT COMPLETED | ELX/TEZ/IVA | 36 |
Source ClinicalTrials.gov
Adverse events136
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 | Part A: ELX/TEZ/IVA | 13 | 251 | |
| other | Abdominal pain | Gastrointestinal disorders | Part B: ELX/TEZ/IVA | 2 | 84 | |
| other | Constipation | Gastrointestinal disorders | Part A: ELX/TEZ/IVA | 17 | 251 | |
| other | Constipation | Gastrointestinal disorders | Part B: ELX/TEZ/IVA | 1 | 84 | |
| other | Diarrhoea | Gastrointestinal disorders | Part A: ELX/TEZ/IVA | 48 | 251 | |
| other | Diarrhoea | Gastrointestinal disorders | Part B: ELX/TEZ/IVA | 4 | 84 | |
| other | Gastrooesophageal reflux disease | Gastrointestinal disorders | Part A: ELX/TEZ/IVA | 13 | 251 | |
| other | Gastrooesophageal reflux disease | Gastrointestinal disorders | Part B: ELX/TEZ/IVA | 1 | 84 | |
| other | Nausea | Gastrointestinal disorders | Part A: ELX/TEZ/IVA | 28 | 251 | |
| other | Nausea | Gastrointestinal disorders | Part B: ELX/TEZ/IVA | 2 | 84 |
1 to 10 of 136Source ClinicalTrials.gov
See all 136 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A: ELX/TEZ/IVA | 1 | 251 |
| Total, all-cause mortality | Part B: ELX/TEZ/IVA | 0 | 84 |
| Total, other adverse events | Part A: ELX/TEZ/IVA | 223 | 251 |
| Total, other adverse events | Part B: ELX/TEZ/IVA | 48 | 84 |
| Total, serious adverse events | Part A: ELX/TEZ/IVA | 38 | 251 |
| Total, serious adverse events | Part B: ELX/TEZ/IVA | 3 | 84 |
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Part A: ELX/TEZ/IVA vs Part B: ELX/TEZ/IVA: deaths from any cause | Trial comparisons |
| Part A: ELX/TEZ/IVA vs Part B: 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.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04058366
GET https://conductscience.org/api/trials/v1/NCT04058366/events?page=2