Trial · NCT03085485

The Topic Trial - Study to Determine the Safety and Efficacy of Ivacaftor

NCT03085485, Phase 2, Completed, University of Alabama at Birmingham; results first posted 2023-12-21

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. The Topic Trial - Study to Determine the Safety and Efficacy of Ivacaftor. Trials, record NCT03085485. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03085485

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: The Topic Trial - Study to Determine the Safety and Efficacy of Ivacaftor
NCT number: NCT03085485
Status: Completed
Phases: Phase 2
Enrollment: 40 participants, actual
Lead sponsor: University of Alabama at Birmingham
Conditions: Chronic Obstructive Pulmonary Disease, Chronic Bronchitis
Interventions: Ivacaftor 150 MG, Placebo
First posted: 2017-03-21
Last updated: 2024-12-12
Results first posted: 2023-12-21

Source ClinicalTrials.gov

Registered outcomes11

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Safety of Ivacaftor - Number of Participants With Adverse Events	From Screening to Day 98	Safety of ivacaftor will be determined by number of participants with adverse events (including serious adverse events).
Primary	Safety of Ivacaftor - Number of Participants With Abnormal Serum Chemistry	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.
Primary	Safety of Ivacaftor - Number of Participants With Abnormal Hematology	From Screening to Day 98	Safety of ivacaftor will also be determined by number of participants with abnormal changes in their screening hematology values.
Primary	Safety of Ivacaftor - Number of Participants With Abnormal ECG (Prolonged QT Intervals)	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.
Secondary	Central CFTR Activity Measured by Mucociliary Clearance (MCC) Percentage Clearance at 60 Mins	From Screening to Day 84	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.
Secondary	Peripheral CFTR Activity Measured by Change in Sweat Chloride	From Screening to Day 84	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.
Secondary	Indicators of Respiratory Function and COPD Health : Change in FEV1 Predict %	From Screening to Day 84	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.
Secondary	San Diego Shortness of Breath Questionnaire (SOBQ)	From D1 to Day 84	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.
Secondary	Breathlessness, Cough, and Sputum Scale (BCSS)	From D1 to Day 84	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.
Secondary	COPD Assessment Test (CAT)	From D1 to Day 84	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.

1 to 10 of 11Source ClinicalTrials.gov

See all 11 registered outcomes

Outcomes11

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Safety of Ivacaftor - Number of Participants With Adverse Events	From Screening to Day 98	One subject dropped out of study after being randomized.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Safety of Ivacaftor - Number of Participants With Abnormal Serum Chemistry	From Screening to Day 98		Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Safety of Ivacaftor - Number of Participants With Abnormal Hematology	From Screening to Day 98		Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Safety of Ivacaftor - Number of Participants With Abnormal ECG (Prolonged QT Intervals)	From Screening to Day 98		Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Central CFTR Activity Measured by Mucociliary Clearance (MCC) Percentage Clearance at 60 Mins	From Screening to Day 84		percentage of clearance	MEAN
SECONDARY	Peripheral CFTR Activity Measured by Change in Sweat Chloride	From Screening to Day 84		mmol/L	MEAN
SECONDARY	Indicators of Respiratory Function and COPD Health : Change in FEV1 Predict %	From Screening to Day 84		predict percentage	MEAN
SECONDARY	San Diego Shortness of Breath Questionnaire (SOBQ)	From D1 to Day 84		units on a scale	MEAN
SECONDARY	Breathlessness, Cough, and Sputum Scale (BCSS)	From D1 to Day 84		units on a scale	MEAN
SECONDARY	COPD Assessment Test (CAT)	From D1 to Day 84		units on a scale	MEAN

1 to 10 of 11Source ClinicalTrials.gov

See all 11 outcomes

Results by group28

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Safety of Ivacaftor - Number of Participants With Adverse Events	Ivacaftor	Subject with any AE	19	Participants
Safety of Ivacaftor - Number of Participants With Adverse Events	Placebo	Subject with any AE	6	Participants
Safety of Ivacaftor - Number of Participants With Adverse Events	Ivacaftor	AE resulting in drug interruption	7	Participants
Safety of Ivacaftor - Number of Participants With Adverse Events	Placebo	AE resulting in drug interruption	3	Participants
Safety of Ivacaftor - Number of Participants With Adverse Events	Ivacaftor	AE resulting in drug discontinuation	1	Participants
Safety of Ivacaftor - Number of Participants With Adverse Events	Placebo	AE resulting in drug discontinuation	1	Participants
Safety of Ivacaftor - Number of Participants With Abnormal Serum Chemistry	Ivacaftor		0	Participants
Safety of Ivacaftor - Number of Participants With Abnormal Serum Chemistry	Placebo		0	Participants
Safety of Ivacaftor - Number of Participants With Abnormal Hematology	Ivacaftor		0	Participants
Safety of Ivacaftor - Number of Participants With Abnormal Hematology	Placebo		0	Participants

1 to 10 of 28Source ClinicalTrials.gov

See all 28 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Ivacaftor	30
Overall Study	STARTED	Placebo	10
Overall Study	COMPLETED	Ivacaftor	28
Overall Study	COMPLETED	Placebo	10
Overall Study	NOT COMPLETED	Ivacaftor	2
Overall Study	NOT COMPLETED	Placebo	0

Source ClinicalTrials.gov

Adverse events10

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
serious	Pneumonia	Respiratory, thoracic and mediastinal disorders	Ivacaftor	2	29	2
serious	Pneumonia	Respiratory, thoracic and mediastinal disorders	Placebo	0	10	0
serious	Altered mental status	Psychiatric disorders	Ivacaftor	1	29	1
serious	Altered mental status	Psychiatric disorders	Placebo	0	10	0
serious	Diarrhea	Gastrointestinal disorders	Ivacaftor	1	29	1
serious	Diarrhea	Gastrointestinal disorders	Placebo	0	10	0
serious	AECOPD	Respiratory, thoracic and mediastinal disorders	Ivacaftor	2	29	2
serious	AECOPD	Respiratory, thoracic and mediastinal disorders	Placebo	0	10	0
serious	Chest pain	Respiratory, thoracic and mediastinal disorders	Ivacaftor	0	29	0
serious	Chest pain	Respiratory, thoracic and mediastinal disorders	Placebo	1	10	1

Source ClinicalTrials.gov

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Ivacaftor	19	29
Total, all-cause mortality	Placebo	6	10
Total, other adverse events	Ivacaftor	0	29
Total, other adverse events	Placebo	0	10
Total, serious adverse events	Ivacaftor	6	29
Total, serious adverse events	Placebo	1	10

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
39316773	DERIVED

Source PubMed

Grants1

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.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
R34HL127166	A Pilot Study of the Effect of the CFTR Potentiator Ivacaftor in COPD (P-TOPIC)	ROWE, STEVEN MARK; DRANSFIELD, MARK T	UNIVERSITY OF ALABAMA AT BIRMINGHAM	NATIONAL HEART, LUNG, AND BLOOD INSTITUTE	2015 to 2016

Source NIH RePORTER

Trial comparisons5

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Ivacaftor vs Placebo: Safety of Ivacaftor - Number of Participants With Abnormal Serum Chemistry	Trial comparisons
Ivacaftor vs Placebo: Safety of Ivacaftor - Number of Participants With Abnormal Hematology	Trial comparisons
Ivacaftor vs Placebo: deaths from any cause	Trial comparisons
Ivacaftor vs Placebo: Safety of Ivacaftor - Number of Participants With Abnormal ECG (Prolonged QT Intervals)	Trial comparisons
Ivacaftor 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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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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Read this trial from software.

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