Trial · NCT02871778
Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia
NCT02871778, Phase 2, Completed, Parion Sciences; results first posted 2021-12-16
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia. Trials, record NCT02871778. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02871778
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia
- NCT number
- NCT02871778
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 123 participants, actual
- Lead sponsor
- Parion Sciences
- Conditions
- Primary Ciliary Dyskinesia
- Interventions
- VX-371, Hypertonic Saline, Placebo (0.17% saline), VX-371 + HS, Ivacaftor
- First posted
- 2016-08-18
- Last updated
- 2021-12-16
- Other registries' IDs
- eudract:2015-004917-26
- Results first posted
- 2021-12-16
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part A: From first dose of study drug up 84 days | An 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 pulse oximetry 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 84 days that were absent before treatment or that worsened relative to pretreatment state. TEAEs included both serious and non-serious TEAEs. |
| Primary | Part B: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part B: Day 85 up to 28 days after last dose of study drug (56 days) | An 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 pulse oximetry examinations. 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 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 | Part A: Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 29 | Part A: Study Baseline, Day 29 of each treatment period | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The study baseline is defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of study drug in the study. |
| Primary | Part B: Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 29 | Study Baseline, Day 29 of Part B | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The study baseline is defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of study drug in the study. |
| Primary | Part B: Absolute Change From Part B Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 29 | Part B Baseline, Day 29 of Part B | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Part B baseline was defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of ivacaftor in Part B and after the last dose in Period 2. |
| Secondary | Part A: Change From Study Baseline in Quality of Life-Primary Ciliary Dyskinesia (QOL-PCD) (Adult Version) Lower Respiratory Symptoms Domain Score at Day 29 | Study Baseline, Day 29 of Part A | QOL- PCD adult version has following 10 domains: lower respiratory symptoms, emotional functioning, treatment burden, role, social functioning, vitality, health perception, upper respiratory symptoms, physical functioning and hearing symptoms. The total numbers of items in the lower respiratory symptoms domain are 6 in the questionnaire for adults. All items are scored using a 4-point Likert scale. Scaled score calculated as: \[Sum of scores - (n\*1)\] / \[(n\*4) - (n\*1)\]\*100. Where 'n' is the number of questions in domain. The total score range is from 0-100, where higher score indicates greater improvement. Change from study baseline \>0 indicated improvement. The study baseline is defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of study drug in the study. |
| Secondary | Part B: Change From Study Baseline in Quality of Life-Primary Ciliary Dyskinesia (QOL-PCD) (Adult Version) Lower Respiratory Symptoms Domain Score at Day 29 | Study Baseline, Day 29 of Part B | QOL- PCD adult version has following 10 domains: lower respiratory symptoms, emotional functioning, treatment burden, role, social functioning, vitality, health perception, upper respiratory symptoms, physical functioning and hearing symptoms. The total numbers of items in the lower respiratory symptoms domain are 6 in the questionnaire for adults. All items are scored using a 4-point Likert scale. Scaled score calculated as: \[Sum of scores - (n\*1)\] / \[(n\*4) - (n\*1)\]\*100. Where 'n' is the number of questions in domain. The total score range is from 0-100, where higher score indicates greater improvement. Change from study baseline \>0 indicated improvement. The study baseline is defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of study drug in the study. |
| Secondary | Part B: Change From Part B Baseline in Quality of Life-Primary Ciliary Dyskinesia (QOL-PCD) (Adult Version) Lower Respiratory Symptoms Domain Score at Day 29 | Part B Baseline, Day 29 of Part B | QOL- PCD adult version has following 10 domains: lower respiratory symptoms, emotional functioning, treatment burden, role, social functioning, vitality, health perception, upper respiratory symptoms, physical functioning and hearing symptoms. The total numbers of items in the lower respiratory symptoms domain are 6 in the questionnaire for adults. All items are scored using a 4-point Likert scale. Scaled score calculated as: \[Sum of scores - (n\*1)\] / \[(n\*4) - (n\*1)\]\*100. Where 'n' is the number of questions in domain. The total score range is from 0-100, where higher score indicates greater improvement. Change from Part B baseline \>0 indicated improvement. Part B baseline was defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of ivacaftor in Part B and after the last dose in Period 2. |
| Secondary | Part A: Change From Study Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score for Participants Aged Greater Than or Equals to (>=) 16 Years at Day 29 | Study Baseline, Day 29 of Part A | SGRQ measured health-related quality of life among participants with respiratory diseases. It is a 40 items questionnaire grouped into three domains (Symptoms, Activity, and Impacts). Total scores range from 0 to 100. Higher score reflected worse quality of life. The study baseline is defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of study drug in the study. |
| Secondary | Part B: Change From Study Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score for Participants Aged >=16 Years at Day 29 | Study Baseline, Day 29 of Part B | SGRQ measured health-related quality of life among participants with respiratory diseases. It is a 40 items questionnaire grouped into three domains (Symptoms, Activity, and Impacts). Total scores range from 0 to 100. Higher score reflected worse quality of life. The study baseline is defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of study drug in the study. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part A: From first dose of study drug up 84 days | Part A safety set included all participants who received at least 1 dose of study drug in Part A. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part B: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part B: Day 85 up to 28 days after last dose of study drug (56 days) | Part B safety set included all participants who received at least 1 dose of ivacaftor in Part B. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part A: Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 29 | Part A: Study Baseline, Day 29 of each treatment period | Part A FAS included all randomized participants who received at least 1 dose of study drug in Part A and had a confirmed diagnosis of PCD. Here, overall number of participants analyzed signifies participants evaluable for this outcome measure. | Percentage of predicted FEV1 | LEAST_SQUARES_MEAN |
| PRIMARY | Part B: Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 29 | Study Baseline, Day 29 of Part B | Part B FAS included all participants who had a confirmed diagnosis of PCD and received at least 1 dose of ivacaftor in Part B. Here, overall number of participants analyzed signifies participants evaluable for this outcome measure. | Percentage of predicted FEV1 | LEAST_SQUARES_MEAN |
| PRIMARY | Part B: Absolute Change From Part B Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 29 | Part B Baseline, Day 29 of Part B | Part B FAS included all participants who had a confirmed diagnosis of PCD and received at least 1 dose of ivacaftor in Part B. Here, overall number of participants analyzed signifies participants evaluable for this outcome measure. | Percentage of predicted FEV1 | LEAST_SQUARES_MEAN |
| SECONDARY | Part A: Change From Study Baseline in Quality of Life-Primary Ciliary Dyskinesia (QOL-PCD) (Adult Version) Lower Respiratory Symptoms Domain Score at Day 29 | Study Baseline, Day 29 of Part A | Part A FAS included all randomized participants who received at least 1 dose of study drug in Part A and had a confirmed diagnosis of PCD. Here, overall number of participants analyzed signifies adult participants evaluable for this outcome measure. | score on a scale | MEAN |
| SECONDARY | Part B: Change From Study Baseline in Quality of Life-Primary Ciliary Dyskinesia (QOL-PCD) (Adult Version) Lower Respiratory Symptoms Domain Score at Day 29 | Study Baseline, Day 29 of Part B | Part B FAS included all participants who had a confirmed diagnosis of PCD and received at least 1 dose of ivacaftor in Part B. Here, overall number of participants analyzed signifies adult participants evaluable for this outcome measure. | score on a scale | MEAN |
| SECONDARY | Part B: Change From Part B Baseline in Quality of Life-Primary Ciliary Dyskinesia (QOL-PCD) (Adult Version) Lower Respiratory Symptoms Domain Score at Day 29 | Part B Baseline, Day 29 of Part B | Part B FAS included all participants who had a confirmed diagnosis of PCD and received at least 1 dose of ivacaftor in Part B. Here, overall number of participants analyzed signifies adult participants evaluable for this outcome measure. | score on a scale | MEAN |
| SECONDARY | Part A: Change From Study Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score for Participants Aged Greater Than or Equals to (>=) 16 Years at Day 29 | Study Baseline, Day 29 of Part A | Part A FAS included all randomized participants who received at least 1 dose of study drug in Part A and had a confirmed diagnosis of PCD. Here, overall number of participants analyzed signifies participants \>=16 years of age evaluable for this outcome measure. | score on a scale | MEAN |
| SECONDARY | Part B: Change From Study Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score for Participants Aged >=16 Years at Day 29 | Study Baseline, Day 29 of Part B | Part B FAS included all participants who had a confirmed diagnosis of PCD and received at least 1 dose of ivacaftor in Part B. Here, overall number of participants analyzed signifies participants \>=16 years of age evaluable for this outcome measure. | score on a scale | MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group52
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part A: VX-371 in HS | Participants with TEAEs | 52 | Participants | |
| Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part A: HS | Participants with TEAEs | 46 | Participants | |
| Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part A: VX-371 | Participants with TEAEs | 22 | Participants | |
| Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part A: Placebo | Participants with TEAEs | 23 | Participants | |
| Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part A: VX-371 in HS | Participants with Serious TEAEs | 1 | Participants | |
| Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part A: HS | Participants with Serious TEAEs | 1 | Participants | |
| Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part A: VX-371 | Participants with Serious TEAEs | 1 | Participants | |
| Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part A: Placebo | Participants with Serious TEAEs | 1 | Participants | |
| Part B: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part B: VX-371 in HS + IVA | Participants with TEAEs | 5 | Participants | |
| Part B: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs | Part B: HS + IVA | Participants with TEAEs | 17 | Participants |
1 to 10 of 52Source ClinicalTrials.gov
See all 52 results by groupParticipant flow96
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part A: Treatment Period 1 | STARTED | Part A: VX-371 in Hypertonic Saline (HS), Then HS | 43 | |
| Part A: Treatment Period 1 | STARTED | Part A: HS, Then VX-371 in HS | 41 | |
| Part A: Treatment Period 1 | STARTED | Part A: VX-371, Then Placebo | 21 | |
| Part A: Treatment Period 1 | STARTED | Part A: Placebo, Then VX-371 | 18 | |
| Part A: Treatment Period 1 | STARTED | Part B: VX-371 in HS + Ivacaftor | 0 | |
| Part A: Treatment Period 1 | STARTED | Part B: HS + Ivacaftor | 0 | |
| Part A: Treatment Period 1 | STARTED | Part B: VX-371 + Ivacaftor | 0 | |
| Part A: Treatment Period 1 | STARTED | Part B: Placebo + Ivacaftor | 0 | |
| Part A: Treatment Period 1 | Safety Analysis Set | Part A: VX-371 in Hypertonic Saline (HS), Then HS | 44 | |
| Part A: Treatment Period 1 | Safety Analysis Set | Part A: HS, Then VX-371 in HS | 40 |
1 to 10 of 96Source ClinicalTrials.gov
See all 96 participant flowAdverse events376
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 | Hot flush | Vascular disorders | Part A: VX-371 in HS | 0 | 81 | |
| other | Hot flush | Vascular disorders | Part A: HS | 0 | 80 | |
| other | Hot flush | Vascular disorders | Part A: VX-371 | 0 | 37 | |
| other | Hot flush | Vascular disorders | Part A: Placebo | 0 | 36 | |
| other | Hot flush | Vascular disorders | Part B: VX-371 in HS + Ivacaftor | 0 | 11 | |
| other | Hot flush | Vascular disorders | Part B: HS + Ivacaftor | 2 | 27 | |
| other | Hot flush | Vascular disorders | Part B: VX-371 + Ivacaftor | 0 | 7 | |
| other | Hot flush | Vascular disorders | Part B: Placebo + Ivacaftor | 0 | 12 | |
| other | Haemophilus test positive | Investigations | Part A: VX-371 in HS | 1 | 81 | |
| other | Haemophilus test positive | Investigations | Part A: HS | 1 | 80 |
1 to 10 of 376Source ClinicalTrials.gov
See all 376 adverse eventsAdverse event totals24
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A: VX-371 in HS | 0 | 81 |
| Total, all-cause mortality | Part A: HS | 0 | 80 |
| Total, all-cause mortality | Part A: VX-371 | 0 | 37 |
| Total, all-cause mortality | Part A: Placebo | 0 | 36 |
| Total, all-cause mortality | Part B: VX-371 in HS + Ivacaftor | 0 | 11 |
| Total, all-cause mortality | Part B: HS + Ivacaftor | 0 | 27 |
| Total, all-cause mortality | Part B: VX-371 + Ivacaftor | 0 | 7 |
| Total, all-cause mortality | Part B: Placebo + Ivacaftor | 0 | 12 |
| Total, other adverse events | Part A: VX-371 in HS | 40 | 81 |
| Total, other adverse events | Part A: HS | 27 | 80 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37660715 | DERIVED |
Source PubMed
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.
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