Chronic Obstructive Pulmonary DiseasePhase 2NCT03085485
The Topic Trial - Study to Determine the Safety and Efficacy of Ivacaftor
A Phase 2 study testing Ivacaftor 150 MG in people with Chronic Obstructive Pulmonary Disease and Chronic Bronchitis, run by University of Alabama at Birmingham at 1 site in United States.
Trial summary
The study is a Phase 2 Study to establish the safety and efficacy of a drug called Ivacaftor (VX-770) in patients with chronic obstructive pulmonary disease (COPD), chronic bronchitis, and acquired CFTR dysfunction as detected by sweat chloride analysis. Read more about this study
- Who is it for?
- Ages 40 to 80 years
- Conditions the study is for
- Chronic Obstructive Pulmonary Disease, Chronic Bronchitis
- What is being tested?
- Ivacaftor 150 MG
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Mar 2017to 30 Sep 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or Female age 40-80
- A Clinical diagnosis of COPD as defined by GOLD
- At Least a 10 pack year smoking history
- Exhibit symptoms of chronic bronchitis as defined by the Medical Research Council
- FEV1% predicted ≥ 35% and ≤70% Post Bronchodilator
- Clinically stable in the last 4 weeks with no evidence of COPD exacerbation
Show all 10Show fewer
- Weight of 40 kg-120 kg
- Willingness to use at least one form of acceptable birth control including abstinence, condom with spermicide, or hormonal contraceptives from time of signing ICF through study follow up visit
- Willing to monitor blood glucose if known history of diabetes mellitus requiring insulin or medical therapy
- Element of CFTR Dysfunction, as defined by Sweat Chloride > 30 mEq/L)
Who cannot join
- Current Diagnosis of Asthma
- Known Diagnosis of Cystic Fibrosis
- Use of Continuous Oxygen Therapy of greater than 2 liters per minute - patients on 2 liters of Oxygen or less will be excluded if they have been hospitalized for COPD in the prior year or had more than 2 exacerbations requiring steroids and/or antibiotics in the prior year.
- Documented history of drug abuse within the last year
- Subjects should not have a pulmonary exacerbation or changes in therapy for pulmonary disease within 28 days before receiving the first dose of study drug.
- Cirrhosis or elevated liver transaminases > 3X ULN
Show all 13Show fewer
- GFR < 50 estimated by Cockroft-Gault
- Any illness or abnormal lab finding that, in the opinion of the investigator might confound the results of the study or pose an additional risk in administering study drug to the subject.
- Pregnant or Breastfeeding
- Subjects taking moderate or strong inhibitors or inducers of CYP3A4, including certain herbal medications and grapefruit juice. (Excluded medications and foods including the drugs and foods listed in the IRB HSP application.)
- Uncontrolled Diabetes
- Recent (e.g 1year) arterial thrombotic events (peripheral arterial disease, thrombotic stroke)
- Clinically significant arrhythmias requiring anti-arrhythmic agent(s) or conduction abnormalities that in the opinion of the investigator that affect patient safety such as the abnormalities listed below (patients with stable coronary artery disease are eligible) : (1) Angina symptoms (2) History of MI (3) Revascularization procedure in the last year prior to screening (4) Clinically significant congestive heart failure (known LVEF <= 45%, cor pulmonale, diastolic heart failure, etc)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Double-blind, Phase 2, Placebo Controlled Study to Determine the Safety and Efficacy of Ivacaftor (VX-770) for the Treatment of Chronic Obstructive Pulmonary Disease (The Topic Trial)
The study is a Phase 2 Study to establish the safety and efficacy of a drug called Ivacaftor (VX-770) in patients with chronic obstructive pulmonary disease (COPD), chronic bronchitis, and acquired CFTR dysfunction as detected by sweat chloride analysis. The design is a pilot, randomized (3:1, active:placebo), double-blind, placebo-controlled study. Approximately 40 subjects with COPD will be randomized.
Read the detailed description
Like CF, COPD is characterized by small airway mucus obstruction that is associated with accelerated loss of lung function and mortality. Preliminary data indicate that cigarette smoke exerts deleterious effects on airway epithelial function including the reduction of CFTR activity, enhanced mucus expression, and a pronounced reduction in mucociliary transport (MCT). Preliminary data also indicate that approximately 50% of patients with COPD have reduced CFTR activity, as detected in the upper airways, lower airways and sweat glands. Furthermore, CFTR dysfunction is independently associated with chronic bronchitis, can persist despite smoking cessation, and can be reversed by the CFTR potentiator ivacaftor (VX-770) in vitro by activating wild-type CFTR, resulting in a robust increase in MCT. Combined with unprecedented clinical improvement via augmented mucociliary clearance in CF patients with a responsive CFTR mutation treated with ivacaftor, these data indicate that CFTR represents a viable therapeutic target to address mucus stasis in a large subset of COPD patients (potentially representing over 4 million patients in the U.S. alone). This project will investigate the hypothesis that ivacaftor can augment CFTR activity in individuals with COPD who exhibit chronic bronchitis, resulting in meaningful improvements in epithelial function and respiratory health. The investigators' initial pilot study in patients with COPD and chronic bronchitis demonstrated that ivacaftor was safe, demonstrated stable pharmacokinetics, and exhibited a trend towards efficacy in measures of PROs and sweat chloride. The current trial will test the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in a larger number of COPD patients with chronic bronchitis and for a longer treatment period, evaluating the potential of CFTR potentiator therapy to address acquired CFTR dysfunction in this population and set the stage for larger and longer-term trials in the future. Based on an IND already in place in the Rowe laboratory, an IRB familiar with the proposed study, an experienced clinical investigation team with expertise in all of the endpoints proposed, and a well characterized COPD population prioritized for the presence chronic bronchitis, CFTR dysfunction, and the absence of congenital CFTR mutations, the investigators are poised to deliver the trial.
Enrollment is planned at a single center, The University of Alabama at Birmingham. Patients will be randomized 3:1 to active drug (n=30) and placebo (10) to achieve the enrollment goal.
A sufficient number of subjects will be screened to randomize up to 50 subjects to achieve 40 completed subjects to receive either ivacaftor 150 mg BID (n=30) or placebo (n=10) for 84 days.
Ivacaftor and matching placebo will be orally administered as capsules according to the following guidelines:
Between study visit Day 1 and study visit Day 84, subjects will take 1 dose of study drug each day in the morning, beginning any time between 08:00 h (8:00 AM) and 12:00 h (12:00 PM). Whenever possible, subjects should take the study drug at the same time each day.
On the study visit days when PK samples are collected (study visit Days 1, 28, 56, and 84), the study drug is to be taken by the subject while he/she is at the study site
For visits after the Day 1 visit, subjects will be instructed to bring all remaining study drug materials to the site; study drug will be dispensed at each visit.
Ivacaftor will be prepared and dispensed by an unblinded pharmacist.
Subjects will be instructed to continue their standard COPD medication regimen.
- Being tested
- Ivacaftor 150 MG, Placebo
- Keywords
- COPD, CFTR, ivacaftor, topic trial
- Medical subject headings
- Pulmonary Disease, Chronic Obstructive, Bronchitis, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ivacaftor 150 MGDrug
Also called KALYDECO
Ivacaftor is a CFTR potentiator
PlaceboDrug
placebo pills
| Study group | Role | What they receive |
|---|---|---|
| Ivacaftor | Active comparator | Ivacaftor, 150 mg PO every 12 hrs for 84 days Receives: Drug: Ivacaftor 150 MG |
| Placebo | Placebo comparator | matching placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of Ivacaftor - Number of Participants With Adverse Events
Safety of ivacaftor will be determined by number of participants with adverse events (including serious adverse events).
Measured: From Screening to Day 98
Safety of Ivacaftor - Number of Participants With Abnormal Serum Chemistry
Number of participants with abnormal serum chemistry values compared to screening values will also be used to determine safety of ivacaftor.
Measured: From Screening to Day 98
Safety of Ivacaftor - Number of Participants With Abnormal Hematology
Safety of ivacaftor will also be determined by number of participants with abnormal changes in their screening hematology values.
Measured: From Screening to Day 98
Safety of Ivacaftor - Number of Participants With Abnormal ECG (Prolonged QT Intervals)
Number of participants with abnormal changes in their screening ECGs is another factor that will be used to evaluate the safety of ivacaftor.
Measured: From Screening to Day 98
Secondary outcomes (7)
Central CFTR Activity Measured by Mucociliary Clearance (MCC) Percentage Clearance at 60 Mins
Clearance of Tc99 sulfur colloid is a measure of MCC of the lungs, and is calculated by a standard protocol developed by the Cystic Fibrosis Therapeutics Development Network. The method provides a robust measure of MCC, and has been sensitive to the effects of inhaled pharmacologic agents in CF and COPD including improvements of an unprecedentedly large magnitude in CF patients with the G551D-CFTR mutation treated with ivacaftor measured in a multicenter study. The technique allows estimates of MCC in both the small and large airway compartments.
Measured: From Screening to Day 84
Peripheral CFTR Activity Measured by Change in Sweat Chloride
Sweat chloride abnormality is correlated with COPD severity and symptoms, and is a highly sensitive outcome measure for CFTR-directed therapeutics. We have shown sweat chloride is sensitive to the presence of cigarette smoking and COPD, and the test has been successfully used as an endpoint in multiple CF trials, including studies to detect the efficacy of ivacaftor therapy.
Measured: From Screening to Day 84
Indicators of Respiratory Function and COPD Health : Change in FEV1 Predict %
Spirometry is a standard outcome measure in COPD and a major indicator of efficacy and safety in COPD clinical trials. Post-bronchodilator spirometry will be performed by ATS criteria. FEV1 will be measured in predict percentage.
Measured: From Screening to Day 84
San Diego Shortness of Breath Questionnaire (SOBQ)
The SOBQ is a self-reported questionnaire that assesses shortness of breath while performing a variety of activities of daily living. The Minimum Clinically Important Difference (MCID) is 5.
The SOBQ includes 24 items, using 6-point scale with 0 = "not at all" to 5 = "maximal or unable to do because of breathlessness". The sum of SOBQ score ranges from 0 to 120, with higher score indicating more breathlessness.
Measured: From D1 to Day 84
Breathlessness, Cough, and Sputum Scale (BCSS)
The BCSS is a three-item questionnaire rating breathlessness, cough and sputum on a 5-point Likert scale from 0 (no symptoms) to 4 (severe symptoms).
The BCSS score ranges from 0 to 12; the higher the score indicates the worse of symptoms.
Measured: From D1 to Day 84
COPD Assessment Test (CAT)
CAT is a self-reported questionnaire that measures COPD related quality of life. The MCID is 2.
CAT composes of 8 questions, and the scores range from 0 - 40. Higher scores denote a more severe impact of COPD on a patient's life.
Measured: From D1 to Day 84
St. George Respiratory Questionnaire (SGRQ)
The SGRQ is a disease-specific measure of health status for use in COPD with an MCID of 4.
The scores range from 0 to 100, with higher scores indicating more limitations.
Measured: From D1 to Day 84
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2017
- Study start16 Mar 2017
- Primary completion30 Sep 2022
- Study completion30 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Obstructive Pulmonary DiseaseStudies of the same condition, open and under way.
- More trials run by University of Alabama at BirminghamThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The design is a pilot, randomized (3:1, active:placebo), double-blind, placebo-controlled study
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mark Dransfield, MD, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03085485
- Sponsor's protocol ID
- F160920009
- Other IDs
- 1R34HL127166-01
- Status last verified
- Dec 2024
- First submitted
- 16 Feb 2017
- Last updated
- 12 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Vijaykumar K, Solomon GM, Guimbellot J, Acosta EP, Bhambhavni PG, White S, Kim H, Raju SV, Rasmussen LW, Harris N, Liu B, Hathorne H, Rowe SM, Dransfield MT. Ivacaftor for Chronic Obstructive Pulmonary Disease: Results from a Phase 2, Randomized Controlled Trial. Am J Respir Crit Care Med. 2025 May;211(5):823-831. doi: 10.1164/rccm.202407-1302OC.
PubMed 39316773
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Phase 2, Placebo Controlled Study to Determine the Safety and Efficacy of Ivacaftor (VX-770) for the Treatment of Chronic Obstructive Pulmonary Disease (The Topic Trial)
- ClinicalTrials.gov ID
- NCT03085485
- Acronym / sponsor ID
- F160920009
- Keywords
- COPD, CFTR, ivacaftor, topic trial
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Vertex Pharmaceuticals Incorporated
- Study start
- 16 Mar 2017 (actual)
- Primary completion
- 30 Sep 2022 (actual)
- Study completion
- 30 Sep 2022 (actual)
- First posted
- 21 Mar 2017 (actual)
- Results
- Posted 21 Dec 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is a Phase 2 Study to establish the safety and efficacy of a drug called Ivacaftor (VX-770) in patients with chronic obstructive pulmonary disease (COPD), chronic bronchitis, and acquired CFTR dysfunction as detected by sweat chloride analysis. The design is a pilot, randomized (3:1, active:placebo), double-blind, placebo-controlled study. Approximately 40 subjects with COPD will be randomized.
Detailed description
Like CF, COPD is characterized by small airway mucus obstruction that is associated with accelerated loss of lung function and mortality. Preliminary data indicate that cigarette smoke exerts deleterious effects on airway epithelial function including the reduction of CFTR activity, enhanced mucus expression, and a pronounced reduction in mucociliary transport (MCT). Preliminary data also indicate that approximately 50% of patients with COPD have reduced CFTR activity, as detected in the upper airways, lower airways and sweat glands. Furthermore, CFTR dysfunction is independently associated with chronic bronchitis, can persist despite smoking cessation, and can be reversed by the CFTR potentiator ivacaftor (VX-770) in vitro by activating wild-type CFTR, resulting in a robust increase in MCT. Combined with unprecedented clinical improvement via augmented mucociliary clearance in CF patients with a responsive CFTR mutation treated with ivacaftor, these data indicate that CFTR represents a viable therapeutic target to address mucus stasis in a large subset of COPD patients (potentially representing over 4 million patients in the U.S. alone). This project will investigate the hypothesis that ivacaftor can augment CFTR activity in individuals with COPD who exhibit chronic bronchitis, resulting in meaningful improvements in epithelial function and respiratory health. The investigators' initial pilot study in patients with COPD and chronic bronchitis demonstrated that ivacaftor was safe, demonstrated stable pharmacokinetics, and exhibited a trend towards efficacy in measures of PROs and sweat chloride. The current trial will test the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in a larger number of COPD patients with chronic bronchitis and for a longer treatment period, evaluating the potential of CFTR potentiator therapy to address acquired CFTR dysfunction in this population and set the stage for larger and longer-term trials in the future. Based on an IND already in place in the Rowe laboratory, an IRB familiar with the proposed study, an experienced clinical investigation team with expertise in all of the endpoints proposed, and a well characterized COPD population prioritized for the presence chronic bronchitis, CFTR dysfunction, and the absence of congenital CFTR mutations, the investigators are poised to deliver the trial.
Enrollment is planned at a single center, The University of Alabama at Birmingham. Patients will be randomized 3:1 to active drug (n=30) and placebo (10) to achieve the enrollment goal.
A sufficient number of subjects will be screened to randomize up to 50 subjects to achieve 40 completed subjects to receive either ivacaftor 150 mg BID (n=30) or placebo (n=10) for 84 days.
Ivacaftor and matching placebo will be orally administered as capsules according to the following guidelines:
Between study visit Day 1 and study visit Day 84, subjects will take 1 dose of study drug each day in the morning, beginning any time between 08:00 h (8:00 AM) and 12:00 h (12:00 PM). Whenever possible, subjects should take the study drug at the same time each day.
On the study visit days when PK samples are collected (study visit Days 1, 28, 56, and 84), the study drug is to be taken by the subject while he/she is at the study site
For visits after the Day 1 visit, subjects will be instructed to bring all remaining study drug materials to the site; study drug will be dispensed at each visit.
Ivacaftor will be prepared and dispensed by an unblinded pharmacist.
Subjects will be instructed to continue their standard COPD medication regimen.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- The design is a pilot, randomized (3:1, active:placebo), double-blind, placebo-controlled study
| Arm | Type | Receives |
|---|---|---|
| Ivacaftor | Active comparator | Drug: Ivacaftor 150 MGIvacaftor, 150 mg PO every 12 hrs for 84 days |
| Placebo | Placebo comparator | Drug: Placebomatching placebo |
Who may qualify
- Ages
- 40 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Male or Female age 40-80
- A Clinical diagnosis of COPD as defined by GOLD
- At Least a 10 pack year smoking history
- Exhibit symptoms of chronic bronchitis as defined by the Medical Research Council
- FEV1% predicted ≥ 35% and ≤70% Post Bronchodilator
Key exclusion criteria (13 listed)
- Current Diagnosis of Asthma
- Known Diagnosis of Cystic Fibrosis
- Use of Continuous Oxygen Therapy of greater than 2 liters per minute - patients on 2 liters of Oxygen or less will be excluded if they have been hospitalized for COPD in the prior year or had more than 2 exacerbations requiring steroids and/or antibiotics in the prior year.
- Documented history of drug abuse within the last year
- Subjects should not have a pulmonary exacerbation or changes in therapy for pulmonary disease within 28 days before receiving the first dose of study drug.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ivacaftor 150 MGAlso: KALYDECOIvacaftor is a CFTR potentiator | Drug | Ivacaftor |
| Placeboplacebo pills | Drug | Placebo |
Primary outcome
Safety of Ivacaftor - Number of Participants With Adverse Events
Time frame: From Screening to Day 98
Safety of ivacaftor will be determined by number of participants with adverse events (including serious adverse events).
Safety of Ivacaftor - Number of Participants With Abnormal Serum Chemistry
Time frame: From Screening to Day 98
Number of participants with abnormal serum chemistry values compared to screening values will also be used to determine safety of ivacaftor.
Safety of Ivacaftor - Number of Participants With Abnormal Hematology
Time frame: From Screening to Day 98
Safety of ivacaftor will also be determined by number of participants with abnormal changes in their screening hematology values.
Safety of Ivacaftor - Number of Participants With Abnormal ECG (Prolonged QT Intervals)
Time frame: From Screening to Day 98
Number of participants with abnormal changes in their screening ECGs is another factor that will be used to evaluate the safety of ivacaftor.
7 secondary outcomes
Central CFTR Activity Measured by Mucociliary Clearance (MCC) Percentage Clearance at 60 Mins
Time frame: From Screening to Day 84
Peripheral CFTR Activity Measured by Change in Sweat Chloride
Time frame: From Screening to Day 84
Indicators of Respiratory Function and COPD Health : Change in FEV1 Predict %
Time frame: From Screening to Day 84
San Diego Shortness of Breath Questionnaire (SOBQ)
Time frame: From D1 to Day 84
Breathlessness, Cough, and Sputum Scale (BCSS)
Time frame: From D1 to Day 84
COPD Assessment Test (CAT)
Time frame: From D1 to Day 84
St. George Respiratory Questionnaire (SGRQ)
Time frame: From D1 to Day 84
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39316773Vijaykumar K, Solomon GM, Guimbellot J, Acosta EP, Bhambhavni PG, White S, Kim H, Raju SV, Rasmussen LW, Harris N, Liu B, Hathorne H, Rowe SM, Dransfield MT. Ivacaftor for Chronic Obstructive Pulmonary Disease: Results from a Phase 2, Randomized Controlled Trial. Am J Respir Crit Care Med. 2025 May;211(5):823-831. doi: 10.1164/rccm.202407-1302OC.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ivacaftor 150 MG in people with Chronic Obstructive Pulmonary Disease and Chronic Bronchitis, run by University of Alabama at Birmingham at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Dec 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of Ivacaftor - Number of Participants With Adverse Events (time frame: From Screening to Day 98). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03085485, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Dec 2024.