Chronic Obstructive Pulmonary DiseaseEarly Phase 1NCT02135432
TOPIC Trial for COPD
A Early Phase 1 study testing Ivacaftor in people with Chronic Obstructive Pulmonary Disease, run by University of Alabama at Birmingham at 1 site in United States.
Trial summary
The study is a randomized, double-blind, placebo-controlled, multiple-dose, pilot study of orally-administered ivacaftor in subjects with chronic obstructive pulmonary disease. Subjects will be administered the study drug ivacaftor 150 mg (or placebo) twice daily (BID). Read more about this study
- Who is it for?
- Ages 40 to 65 years
- Conditions the study is for
- Chronic Obstructive Pulmonary Disease
- What is being tested?
- Ivacaftor
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- May 2015to Nov 2015
- Phase
- Early Phase 1
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or Female age 40-65
- A clinical diagnosis of COPD as defined by GOLD
- At Least a 10 pack year smoking history
- Exhibit symptoms of chronic bronchitis defined by MRC
- FEV1% predicted ≥ 35% and ≤70% Post Bronchodilator
- Clinically stable in the last 4 weeks with no evidence of COPD exacerbation
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- 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
- Willing to monitor blood glucose if known history of diabetes mellitus requiring insulin or medical therapy
Who cannot join
- Current Diagnosis of Asthma
- Daytime use of Oxygen Therapy
- 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
- GFR < 50 estimated by Cockcroft-Gault
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- Any illness of abnormal lab finding that, in the opinion of the investigator or the subject's general practitioner, might confound the results of the study or pose an additional risk in administering study drug to the subject.
- Pregnant or Breastfeeding
- Subjects taking any inhibitors or inducers of CYP3A4, including certain herbal medications and grapefruit/grapefruit juice.
- Uncontrolled Diabetes
- Excluded medications and foods include the drugs and foods provided in the appendix document.
- Clinically significant arrhythmias or conduction abnormalities that in the opinion of the investigator affect patient safety have been added as exclusion criteria and criteria for withdrawal.
- Patients who have not been stable or have been hospitalized in the past 3 months with any clinically significant cardiac conditions.
- Subjects with history of cancer (current or past, unless remote (>5years))except for localized non-melanomatous skin cancers History of Stroke/CVA History of myocardial infarction/acute coronary syndrome Cardiac Failure NYHC grade III-IV Diabetes Type I Uncontrolled Hypertension Primary or secondary pulmonary hypertension
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Trial locations and contacts
1 site in 1 country.
United States1 site
University of Alabama at Birmingham
Birmingham, Alabama, 35233
About this study
Official title: A Randomized, Double-Blind, Placebo Controlled Pilot 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 randomized, double-blind, placebo-controlled, multiple-dose, pilot study of orally-administered ivacaftor in subjects with chronic obstructive pulmonary disease. Subjects will be administered the study drug ivacaftor 150 mg (or placebo) twice daily (BID).
- Conditions
- Chronic Obstructive Pulmonary Disease
- Being tested
- Ivacaftor, Placebo
- Keywords
- Chronic Obstructive pulmonary disease, Ivacaftor
- Medical subject headings
- Pulmonary Disease, Chronic Obstructive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IvacaftorDrug
Also called Kalydeco
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Ivacaftor (VX-770) | Experimental | twice a day administration of Ivacaftor: 150mg Receives: Drug: Ivacaftor |
| 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.
Change in COPD as Measured by the Sweat Analysis in Each Group
sweat analysis is measured by performing a sweat test in each participant. The primary analysis will compare the within group change in sweat chloride before (day 1) and after (day 14) ivacaftor or placebo administration and will be used to test the null hypothesis of no change in sweat chloride using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.
Measured: baseline to 2 weeks
Secondary outcomes (3)
Change in COPD as Measured by Nasal Potential Difference
Evaluate the efficacy of ivacaftor treatment in patients with COPD including measures of CFTR activity and clinical outcome as measured by change in nasal potential difference measurement in each group. These data will be used to test the null hypothesis of no change in nasal potential difference (ΔLow Chloride plus isoproterenol) using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.
Measured: baseline to 2 weeks
Number of Adverse Events Experienced by the Ivacaftor Subjects and Placebo Subjects.
Number of adverse events per subject in each the Ivacaftor subjects and placebo subjects
Measured: baseline to 2 weeks
Change in COPD as Measured by Change in Percentage of FEV1 as Measured in Each Group
Spirometry will be analyzed by ATS criteria, and the best of three reproducible efforts will be used to calculate FEV1 in comparison to Hankinson standards. The primary analysis will be the change in FEV1% from day 0 to day 14 within subject, and will be tested against the null hypothesis that no change occurs using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.
Measured: baseline to 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2014 (estimated)
- Study startMay 2015
- Primary completionOct 2015
- Study completionNov 2015
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.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dr. Steven M Rowe, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02135432
- Sponsor's protocol ID
- F140319002
- Other IDs
- TOPIC Trial
- Status last verified
- Jan 2017
- First submitted
- 15 Apr 2014
- Last updated
- 24 Feb 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo Controlled Pilot 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
- NCT02135432
- Acronym / sponsor ID
- F140319002
- Conditions
- Chronic Obstructive Pulmonary Disease
- Keywords
- Chronic Obstructive pulmonary disease, Ivacaftor
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Study start
- May 2015
- Primary completion
- Oct 2015 (actual)
- Study completion
- Nov 2015 (actual)
- First posted
- 12 May 2014 (estimated)
- Results
- Posted 24 Feb 2017
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 randomized, double-blind, placebo-controlled, multiple-dose, pilot study of orally-administered ivacaftor in subjects with chronic obstructive pulmonary disease. Subjects will be administered the study drug ivacaftor 150 mg (or placebo) twice daily (BID).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ivacaftor (VX-770) | Experimental | Drug: Ivacaftortwice a day administration of Ivacaftor: 150mg |
| Placebo | Placebo comparator | Drug: Placebomatching placebo |
Who may qualify
- Ages
- 40 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male or Female age 40-65
- A clinical diagnosis of COPD as defined by GOLD
- At Least a 10 pack year smoking history
- Exhibit symptoms of chronic bronchitis defined by MRC
- FEV1% predicted ≥ 35% and ≤70% Post Bronchodilator
Key exclusion criteria (15 listed)
- Current Diagnosis of Asthma
- Daytime use of Oxygen Therapy
- 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
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 |
|---|---|---|
| IvacaftorAlso: Kalydeco | Drug | Ivacaftor (VX-770) |
| Placebo | Drug | Placebo |
Primary outcome
Change in COPD as Measured by the Sweat Analysis in Each Group
Time frame: baseline to 2 weeks
sweat analysis is measured by performing a sweat test in each participant. The primary analysis will compare the within group change in sweat chloride before (day 1) and after (day 14) ivacaftor or placebo administration and will be used to test the null hypothesis of no change in sweat chloride using a paired t-test unless the distributions are notably skewed, in which case the non-parametric Wilcoxon signed-rank test will be implemented due to small sample size.
3 secondary outcomes
Change in COPD as Measured by Nasal Potential Difference
Time frame: baseline to 2 weeks
Number of Adverse Events Experienced by the Ivacaftor Subjects and Placebo Subjects.
Time frame: baseline to 2 weeks
Change in COPD as Measured by Change in Percentage of FEV1 as Measured in Each Group
Time frame: baseline to 2 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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 Early Phase 1 study testing Ivacaftor in people with Chronic Obstructive Pulmonary Disease, 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 9 inclusion and 15 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 24 Feb 2017. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 24 Feb 2017). 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?
Change in COPD as Measured by the Sweat Analysis in Each Group (time frame: baseline to 2 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 24 Feb 2017.