Clinical trial NCT02135432: TOPIC Trial for COPD
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Chronic Obstructive Pulmonary Disease
Keywords: Chronic Obstructive pulmonary disease, Ivacaftor
Lead sponsor: University of Alabama at Birmingham
Sponsor class: OTHER
First posted: 2014-05-12
Enrollment: 12
Sex: ALL
Minimum age (years): 40
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility 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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Primary outcomes:
measure: Change in COPD as Measured by the Sweat Analysis in Each Group
timeFrame: baseline to 2 weeks
Secondary outcomes:
measure: Change in COPD as Measured by Nasal Potential Difference
timeFrame: baseline to 2 weeks
measure: Number of Adverse Events Experienced by the Ivacaftor Subjects and Placebo Subjects.
timeFrame: baseline to 2 weeks
measure: Change in COPD as Measured by Change in Percentage of FEV1 as Measured in Each Group
timeFrame: baseline to 2 weeks
Other outcomes:
measure: Pharmacokinetics as Described by AUC12 of Subjects Receiving Ivacaftor
timeFrame: baseline to 2 weeks
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
Study officials:
References: