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TOPIC Trial for COPD

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ClinicalTrials.gov Identifier: NCT02135432
Recruitment Status : Completed
First Posted : May 12, 2014
Results First Posted : February 24, 2017
Last Update Posted : February 24, 2017
Sponsor:
Information provided by (Responsible Party):
Dr. Steven M Rowe, University of Alabama at Birmingham

Brief 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).

Condition or disease Intervention/treatment Phase
Chronic Obstructive Pulmonary Disease Drug: Ivacaftor Drug: Placebo Early Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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)
Study Start Date : May 2015
Actual Primary Completion Date : October 2015
Actual Study Completion Date : November 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: COPD Lung Diseases
Drug Information available for: Ivacaftor

Arm Intervention/treatment
Experimental: Ivacaftor (VX-770)
twice a day administration of Ivacaftor: 150mg
Drug: Ivacaftor
Other Name: Kalydeco

Placebo Comparator: Placebo
matching placebo
Drug: Placebo



Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Change in COPD as Measured by Nasal Potential Difference [ Time Frame: baseline to 2 weeks ]
    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.

  2. Number of Adverse Events Experienced by the Ivacaftor Subjects and Placebo Subjects. [ Time Frame: baseline to 2 weeks ]
    Number of adverse events per subject in each the Ivacaftor subjects and placebo subjects

  3. Change in COPD as Measured by Change in Percentage of FEV1 as Measured in Each Group [ Time Frame: baseline to 2 weeks ]
    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.


Other Outcome Measures:
  1. Pharmacokinetics as Described by AUC12 of Subjects Receiving Ivacaftor [ Time Frame: baseline to 2 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   40 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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
  • 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

Exclusion Criteria:

  • 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
  • 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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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02135432


Locations
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United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Sponsors and Collaborators
University of Alabama at Birmingham
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Responsible Party: Dr. Steven M Rowe, MD, University of Alabama at Birmingham
ClinicalTrials.gov Identifier: NCT02135432    
Other Study ID Numbers: F140319002
TOPIC Trial ( Other Identifier: UAB )
First Posted: May 12, 2014    Key Record Dates
Results First Posted: February 24, 2017
Last Update Posted: February 24, 2017
Last Verified: January 2017
Keywords provided by Dr. Steven M Rowe, University of Alabama at Birmingham:
Chronic Obstructive pulmonary disease, Ivacaftor
Additional relevant MeSH terms:
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Lung Diseases
Lung Diseases, Obstructive
Pulmonary Disease, Chronic Obstructive
Respiratory Tract Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action