TOPIC Trial for COPD

NCT02135432 | Completed Early Phase 1 Results DMC

• 2 other identifiers: F140319002TOPIC Trial
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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).

Conditions

Chronic Obstructive Pulmonary Disease

Keywords

Chronic Obstructive pulmonary disease, Ivacaftor

Outcome Measures

Primary Outcomes (1)

• 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.

  baseline to 2 weeks

Secondary Outcomes (3)

• Change in COPD as Measured by Nasal Potential Difference

  baseline to 2 weeks

• Number of Adverse Events Experienced by the Ivacaftor Subjects and Placebo Subjects.

  baseline to 2 weeks

• Change in COPD as Measured by Change in Percentage of FEV1 as Measured in Each Group

  baseline to 2 weeks

Other Outcomes (1)

• Pharmacokinetics as Described by AUC12 of Subjects Receiving Ivacaftor

  baseline to 2 weeks

Study Arms (2)

Ivacaftor (VX-770)

EXPERIMENTAL

twice a day administration of Ivacaftor: 150mg

Drug: Ivacaftor

Placebo

PLACEBO COMPARATOR

matching placebo

Drug: Placebo

Interventions

Ivacaftor DRUG

Also known as: Kalydeco

Ivacaftor (VX-770)

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 40 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.
• 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

Sponsors & Collaborators

• University of Alabama at Birmingham: lead

Study Sites (1)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

MeSH Terms

Conditions

Pulmonary Disease, Chronic Obstructive

Interventions

ivacaftor

Condition Hierarchy (Ancestors)

Lung Diseases, Obstructive; Lung Diseases; Respiratory Tract Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Limitations and Caveats

this pilot trial was under powered and potentially too brief to detect definitive changes in lung function

Results Point of Contact

• Title: Steven M. Rowe
• Organization: University of Alabama at Birmingham

smrowe@uab.edu

205-975-6385

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: April 15, 2014
• First Posted: May 12, 2014
• Study Start: May 1, 2015
• Primary Completion: October 1, 2015
• Study Completion: November 1, 2015
• Last Updated: February 24, 2017
• Results First Posted: February 24, 2017

Record last verified: 2017-01

Locations

US (1)