Trial · NCT05033080
A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
NCT05033080, Phase 3, Completed, Vertex Pharmaceuticals Incorporated; results first posted 2024-06-10
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF). Trials, record NCT05033080. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05033080
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
- NCT number
- NCT05033080
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 435 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Cystic Fibrosis
- Interventions
- VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, Placebo (matched to VX-121/TEZ/D-IVA), Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA)
- First posted
- 2021-09-02
- Last updated
- 2024-10-03
- Other registries' IDs
- eudract:2021-000712-31
- Results first posted
- 2024-06-10
Source ClinicalTrials.gov
Registered outcomes4
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 Through Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. |
| Secondary | Absolute Change in Sweat Chloride (SwCl) | From Baseline Through Week 24 | Sweat samples were collected using an approved collection device. |
| Secondary | Percentage of Participants With SwCl <60 mmol/L (Pooled With Data From Study VX20-121-103) | From Baseline Through Week 24 | Sweat samples were collected using an approved collection device. |
| Secondary | Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-103) | From Baseline Through Week 24 | Sweat samples were collected using an approved collection device. |
Source ClinicalTrials.gov
Outcomes4
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 Through Week 24 | The Full Analysis Set (FAS) included all randomized participants who carried the intended CFTR mutation(s) and received at least 1 dose of study drug during Treatment Period. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | percentage points | LEAST_SQUARES_MEAN |
| SECONDARY | Absolute Change in Sweat Chloride (SwCl) | From Baseline Through Week 24 | FAS. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | millimole per liter (mmol/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Participants With SwCl <60 mmol/L (Pooled With Data From Study VX20-121-103) | From Baseline Through Week 24 | The Pooled Full Analysis Set (PFAS) included all randomized participants from this study (VX20-121-102) and from Study VX20-121-103 who carried the intended CFTR mutation(s) and received at least 1 dose of study drug during the Treatment Period. Here Overall Number of Participants Analyzed signifies those participants who were evaluated for this specific outcome measure. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-103) | From Baseline Through Week 24 | PFAS. Here Overall Number of Participants Analyzed signifies those participants who were evaluated for this specific outcome measure. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group8
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) | ELX/TEZ/IVA | 0.3 | 95% Confidence Interval -0.3 to 0.9 | percentage points | |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | VX-121/TEZ/D-IVA | 0.5 | 95% Confidence Interval -0.1 to 1.1 | percentage points | |
| Absolute Change in Sweat Chloride (SwCl) | ELX/TEZ/IVA | 0.9 | 95% Confidence Interval -0.6 to 2.3 | millimole per liter (mmol/L) | |
| Absolute Change in Sweat Chloride (SwCl) | VX-121/TEZ/D-IVA | -7.5 | 95% Confidence Interval -9.0 to -6.0 | millimole per liter (mmol/L) | |
| Percentage of Participants With SwCl <60 mmol/L (Pooled With Data From Study VX20-121-103) | ELX/TEZ/IVA | 76.6 | percentage of participants | ||
| Percentage of Participants With SwCl <60 mmol/L (Pooled With Data From Study VX20-121-103) | VX-121/TEZ/D-IVA | 85.8 | percentage of participants | ||
| Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-103) | ELX/TEZ/IVA | 22.5 | percentage of participants | ||
| Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-103) | VX-121/TEZ/D-IVA | 30.5 | percentage of participants |
Source ClinicalTrials.gov
Participant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | ELX/TEZ/IVA | 202 | |
| Overall Study | STARTED | VX-121/TEZ/D-IVA | 196 | |
| Overall Study | Pooled Analysis Set | ELX/TEZ/IVA | 491 | |
| Overall Study | Pooled Analysis Set | VX-121/TEZ/D-IVA | 480 | |
| Overall Study | COMPLETED | ELX/TEZ/IVA | 191 | |
| Overall Study | COMPLETED | VX-121/TEZ/D-IVA | 184 | |
| Overall Study | NOT COMPLETED | ELX/TEZ/IVA | 11 | |
| Overall Study | NOT COMPLETED | VX-121/TEZ/D-IVA | 12 |
Source ClinicalTrials.gov
Adverse events148
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 distension | Gastrointestinal disorders | ELX/TEZ/IVA | 10 | 202 | |
| other | Abdominal distension | Gastrointestinal disorders | VNZ/TEZ/D-IVA | 5 | 196 | |
| other | Abdominal pain | Gastrointestinal disorders | ELX/TEZ/IVA | 14 | 202 | |
| other | Abdominal pain | Gastrointestinal disorders | VNZ/TEZ/D-IVA | 10 | 196 | |
| other | Abdominal pain upper | Gastrointestinal disorders | ELX/TEZ/IVA | 6 | 202 | |
| other | Abdominal pain upper | Gastrointestinal disorders | VNZ/TEZ/D-IVA | 12 | 196 | |
| other | Diarrhoea | Gastrointestinal disorders | ELX/TEZ/IVA | 15 | 202 | |
| other | Diarrhoea | Gastrointestinal disorders | VNZ/TEZ/D-IVA | 21 | 196 | |
| other | Nausea | Gastrointestinal disorders | ELX/TEZ/IVA | 17 | 202 | |
| other | Nausea | Gastrointestinal disorders | VNZ/TEZ/D-IVA | 7 | 196 |
1 to 10 of 148Source ClinicalTrials.gov
See all 148 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | ELX/TEZ/IVA | 0 | 202 |
| Total, all-cause mortality | VNZ/TEZ/D-IVA | 0 | 196 |
| Total, other adverse events | ELX/TEZ/IVA | 184 | 202 |
| Total, other adverse events | VNZ/TEZ/D-IVA | 177 | 196 |
| Total, serious adverse events | ELX/TEZ/IVA | 41 | 202 |
| Total, serious adverse events | VNZ/TEZ/D-IVA | 28 | 196 |
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 |
|---|---|
| ELX/TEZ/IVA vs VNZ/TEZ/D-IVA: deaths from any cause | Trial comparisons |
| ELX/TEZ/IVA vs VNZ/TEZ/D-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/NCT05033080
GET https://conductscience.org/api/trials/v1/NCT05033080/events?page=2