Trial · NCT03525444
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
NCT03525444, Phase 3, Completed, Vertex Pharmaceuticals Incorporated; results first posted 2020-05-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF). Trials, record NCT03525444. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03525444
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
- NCT number
- NCT03525444
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 405 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Cystic Fibrosis
- Interventions
- VX-445/TEZ/IVA, IVA, Placebo
- First posted
- 2018-05-15
- Last updated
- 2020-05-19
- Other registries' IDs
- eudract:2018-000183-28
- Results first posted
- 2020-05-19
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | From Baseline at Week 4 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. |
| Secondary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | From Baseline through Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. |
| Secondary | Number of Pulmonary Exacerbations (PEx) | From Baseline through Week 24 | 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 | Absolute Change in Sweat Chloride (SwCl) | From Baseline through Week 24 | Sweat samples were collected using an approved collection device. |
| Secondary | Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | From Baseline through 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, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. |
| Secondary | Absolute Change in Body Mass Index (BMI) | From Baseline at Week 24 | BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2). |
| Secondary | Absolute Change in Sweat Chloride | From Baseline at Week 4 | Sweat samples were collected using an approved collection device. |
| Secondary | Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | From Baseline at Week 4 | 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. |
| Secondary | Time-to-first Pulmonary Exacerbation (PEx) | From Baseline through Week 24 | 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 | Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline | From Baseline at Week 24 | BMI was defined as weight in kg divided by height in m\^2. Z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | From Baseline at Week 4 | Analysis population included all participants in the Full Analysis Set (all randomized participants who carried the intended CFTR allele mutation and received at least 1 dose of study drug) who completed the Week 4 Visit or were randomized at least 28 days before the data cutoff date. | percentage points | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | From Baseline through Week 24 | FAS. | percentage points | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Pulmonary Exacerbations (PEx) | From Baseline through Week 24 | FAS. | pulmonary exacerbation events | NUMBER |
| SECONDARY | Absolute Change in Sweat Chloride (SwCl) | From Baseline through Week 24 | FAS. | millimole per liter (mmol/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | From Baseline through Week 24 | FAS. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Body Mass Index (BMI) | From Baseline at Week 24 | FAS. | kilogram per meter square (kg/m^2) | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Sweat Chloride | From Baseline at Week 4 | FAS. | mmol/L | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | From Baseline at Week 4 | FAS. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Time-to-first Pulmonary Exacerbation (PEx) | From Baseline through Week 24 | FAS. | days | MEDIAN |
| SECONDARY | Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline | From Baseline at Week 24 | FAS. Here Overall Number of Participants Analyzed signifies those participants who were \<=20 years of age at Baseline. | z-score | LEAST_SQUARES_MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group42
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | Placebo | -0.2 | Standard Error 0.6 | percentage points | |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | VX-445/TEZ/IVA TC | 13.6 | Standard Error 0.6 | percentage points | |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | Placebo | -0.4 | Standard Error 0.5 | percentage points | |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | VX-445/TEZ/IVA TC | 13.9 | Standard Error 0.6 | percentage points | |
| Number of Pulmonary Exacerbations (PEx) | Placebo | 113 | pulmonary exacerbation events | ||
| Number of Pulmonary Exacerbations (PEx) | VX-445/TEZ/IVA TC | 41 | pulmonary exacerbation events | ||
| Absolute Change in Sweat Chloride (SwCl) | Placebo | -0.4 | Standard Error 0.9 | millimole per liter (mmol/L) | |
| Absolute Change in Sweat Chloride (SwCl) | VX-445/TEZ/IVA TC | -42.2 | Standard Error 0.9 | millimole per liter (mmol/L) | |
| Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | Placebo | -2.7 | Standard Error 1.0 | units on a scale | |
| Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | VX-445/TEZ/IVA TC | 17.5 | Standard Error 1.0 | units on a scale |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 203 | |
| Overall Study | STARTED | VX-445/TEZ/IVA TC | 200 | |
| Overall Study | Safety Set | Placebo | 201 | |
| Overall Study | Safety Set | VX-445/TEZ/IVA TC | 202 | |
| Overall Study | COMPLETED | Placebo | 203 | |
| Overall Study | COMPLETED | VX-445/TEZ/IVA TC | 197 | |
| Overall Study | NOT COMPLETED | Placebo | 0 | |
| Overall Study | NOT COMPLETED | VX-445/TEZ/IVA TC | 3 |
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 | Cough | Respiratory, thoracic and mediastinal disorders | Placebo | 77 | 201 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | VX-445/TEZ/IVA TC | 34 | 202 | |
| other | Sputum increased | Respiratory, thoracic and mediastinal disorders | Placebo | 39 | 201 | |
| other | Sputum increased | Respiratory, thoracic and mediastinal disorders | VX-445/TEZ/IVA TC | 40 | 202 | |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Placebo | 25 | 201 | |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | VX-445/TEZ/IVA TC | 20 | 202 | |
| other | Haemoptysis | Respiratory, thoracic and mediastinal disorders | Placebo | 27 | 201 | |
| other | Haemoptysis | Respiratory, thoracic and mediastinal disorders | VX-445/TEZ/IVA TC | 9 | 202 | |
| other | Nasal congestion | Respiratory, thoracic and mediastinal disorders | Placebo | 15 | 201 | |
| other | Nasal congestion | Respiratory, thoracic and mediastinal disorders | VX-445/TEZ/IVA TC | 19 | 202 |
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 | Placebo | 0 | 201 |
| Total, all-cause mortality | VX-445/TEZ/IVA TC | 0 | 202 |
| Total, other adverse events | Placebo | 180 | 201 |
| Total, other adverse events | VX-445/TEZ/IVA TC | 168 | 202 |
| Total, serious adverse events | Placebo | 42 | 201 |
| Total, serious adverse events | VX-445/TEZ/IVA TC | 28 | 202 |
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 |
|---|---|
| VX-445/TEZ/IVA TC vs Placebo: deaths from any cause | Trial comparisons |
| VX-445/TEZ/IVA TC vs Placebo: 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/NCT03525444
GET https://conductscience.org/api/trials/v1/NCT03525444/results?page=2