Trial · NCT02709109
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
NCT02709109, Phase 2, Completed, Parion Sciences; results first posted 2021-07-29
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation. Trials, record NCT02709109. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02709109
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
- NCT number
- NCT02709109
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 147 participants, actual
- Lead sponsor
- Parion Sciences
- Conditions
- Cystic Fibrosis
- Interventions
- VX-371 + HS, Hypertonic saline, Placebo, Orkambi, VX-371
- First posted
- 2016-03-15
- Last updated
- 2021-07-29
- Other registries' IDs
- eudract:2015-004841-13
- Results first posted
- 2021-07-29
Source ClinicalTrials.gov
Registered outcomes4
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 (TEAEs) and Serious TEAEs | Baseline (Day 1) up to 28 days post last administration of study drug (up to 112 days) | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included abnormal clinically significant findings for spirometry, clinical laboratory parameters, standard 12-lead electrocardiograms (ECGs), vital signs and ophthalmologic examinations. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent were events between first dose of study drug and up to 112 days (up to 28 days after last dose) that were absent before treatment or that worsened relative to pretreatment state. TEAEs included both serious and non-serious TEAEs. |
| Primary | Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 | Study baseline, Day 28 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Study Baseline was defined as the most recent non-missing measurement before the first dose of inhaled study drug in the study. Day 28 measurements after treatment discontinuation from the treatment period in which discontinuation occurred were included in the analysis. |
| Secondary | Plasma Concentrations of VX-371 | Pre-dose (90 minutes prior inhaled study drug administration) and 60 minutes post-dose on Days 1, 14 and 28 within treatment period 1 and 2 | |
| Secondary | Urine Concentrations of VX-371 | Pre-dose (90 minutes prior inhaled study drug administration) and 60 minutes post-dose on Days 1, 14 and 28 within treatment period 1 and 2 |
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 | Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Baseline (Day 1) up to 28 days post last administration of study drug (up to 112 days) | Safety set included all participants who received at least 1 dose of inhaled study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 | Study baseline, Day 28 | The full analysis set (FAS) included all randomized participants who carried the intended homozygous F508del-cystic fibrosis transmembrane conductance regulator (CFTR) mutation and received at least 1 dose of inhaled study drug. Here, overall number of participants analyzed signifies participants evaluable for this outcome measure. | percentage points | LEAST_SQUARES_MEAN |
| SECONDARY | Plasma Concentrations of VX-371 | Pre-dose (90 minutes prior inhaled study drug administration) and 60 minutes post-dose on Days 1, 14 and 28 within treatment period 1 and 2 | The Pharmacokinetic (PK) analysis set included all participants who received at least 1 dose of VX-371. Here, overall number of participants analyzed signifies participants evaluable for this outcome measure and number analyzed signifies those participants who were evaluable at specified time points. | Picograms per milliliter (pg/mL) | MEAN |
| SECONDARY | Urine Concentrations of VX-371 | Pre-dose (90 minutes prior inhaled study drug administration) and 60 minutes post-dose on Days 1, 14 and 28 within treatment period 1 and 2 | The PK analysis set included all participants who received at least 1 dose of VX-371. Here, overall number of participants analyzed signifies participants evaluable for this outcome measure and number analyzed signifies those participants who were evaluable at specified time points. | pg/mL | MEAN |
Source ClinicalTrials.gov
Results by group36
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 (TEAEs) and Serious TEAEs | VX-371 + Hypertonic Saline | Participants With TEAEs | 65 | Participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Hypertonic Saline | Participants With TEAEs | 66 | Participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | VX-371 + Placebo | Participants With TEAEs | 32 | Participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Placebo | Participants With TEAEs | 28 | Participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | VX-371 + Hypertonic Saline | Participants With Serious TEAEs | 8 | Participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Hypertonic Saline | Participants With Serious TEAEs | 4 | Participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | VX-371 + Placebo | Participants With Serious TEAEs | 6 | Participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Placebo | Participants With Serious TEAEs | 3 | Participants | |
| Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 | VX-371 + Hypertonic Saline | 0.1 | Standard Error 0.8 | percentage points | |
| Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 | Hypertonic Saline | -0.1 | Standard Error 0.8 | percentage points |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 results by groupParticipant flow36
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period 1 (28 Days) | STARTED | Sequence 1: VX-371 + HS, Then HS | 49 | |
| Treatment Period 1 (28 Days) | STARTED | Sequence 2: HS, Then VX-371 + HS | 47 | |
| Treatment Period 1 (28 Days) | STARTED | Sequence 3: VX-371 + Placebo, Then Placebo | 22 | |
| Treatment Period 1 (28 Days) | STARTED | Sequence 4: Placebo, Then VX-371 + Placebo | 24 | |
| Treatment Period 1 (28 Days) | COMPLETED | Sequence 1: VX-371 + HS, Then HS | 45 | |
| Treatment Period 1 (28 Days) | COMPLETED | Sequence 2: HS, Then VX-371 + HS | 42 | |
| Treatment Period 1 (28 Days) | COMPLETED | Sequence 3: VX-371 + Placebo, Then Placebo | 21 | |
| Treatment Period 1 (28 Days) | COMPLETED | Sequence 4: Placebo, Then VX-371 + Placebo | 24 | |
| Treatment Period 1 (28 Days) | NOT COMPLETED | Sequence 1: VX-371 + HS, Then HS | 4 | |
| Treatment Period 1 (28 Days) | NOT COMPLETED | Sequence 2: HS, Then VX-371 + HS | 5 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 participant flowAdverse events104
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 | VX-371 + Hypertonic Saline | 20 | 89 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Hypertonic Saline | 13 | 90 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | VX-371 + Placebo | 13 | 46 | |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Placebo | 5 | 42 | |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | VX-371 + Hypertonic Saline | 8 | 89 | |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Hypertonic Saline | 6 | 90 | |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | VX-371 + Placebo | 4 | 46 | |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Placebo | 1 | 42 | |
| other | Nasal congestion | Respiratory, thoracic and mediastinal disorders | VX-371 + Hypertonic Saline | 7 | 89 | |
| other | Nasal congestion | Respiratory, thoracic and mediastinal disorders | Hypertonic Saline | 5 | 90 |
1 to 10 of 104Source ClinicalTrials.gov
See all 104 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | VX-371 + Hypertonic Saline | 0 | 89 |
| Total, all-cause mortality | Hypertonic Saline | 0 | 90 |
| Total, all-cause mortality | VX-371 + Placebo | 0 | 46 |
| Total, all-cause mortality | Placebo | 0 | 42 |
| Total, other adverse events | VX-371 + Hypertonic Saline | 49 | 89 |
| Total, other adverse events | Hypertonic Saline | 55 | 90 |
| Total, other adverse events | VX-371 + Placebo | 26 | 46 |
| Total, other adverse events | Placebo | 20 | 42 |
| Total, serious adverse events | VX-371 + Hypertonic Saline | 8 | 89 |
| Total, serious adverse events | Hypertonic Saline | 4 | 90 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT02709109
GET https://conductscience.org/api/trials/v1/NCT02709109/results?page=2