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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

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Single Group Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Chronic Obstructive Pulmonary Disease
Interventions Drug: Ivacaftor
Drug: Placebo
Enrollment 12
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Ivacaftor (VX-770) Placebo
Hide Arm/Group Description

twice a day administration of Ivacaftor: 150mg

Ivacaftor

matching placebo

Placebo

Period Title: Overall Study
Started 8 4
Completed 8 4
Not Completed 0 0
Arm/Group Title Ivacaftor (VX-770) Placebo Total
Hide Arm/Group Description

twice a day administration of Ivacaftor: 150mg

Ivacaftor

matching placebo

Placebo

Total of all reporting groups
Overall Number of Baseline Participants 8 4 12
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 8 participants 4 participants 12 participants
55
(49 to 65)
62
(60 to 65)
55
(49 to 65)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 8 participants 4 participants 12 participants
<=18 years 0 0 0
Between 18 and 65 years 6 3 9
>=65 years 2 1 3
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 4 participants 12 participants
Female
4
  50.0%
1
  25.0%
5
  41.7%
Male
4
  50.0%
3
  75.0%
7
  58.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 8 participants 4 participants 12 participants
8 4 12
1.Primary Outcome
Title Change in COPD as Measured by the Sweat Analysis in Each Group
Hide Description 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.
Time Frame baseline to 2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
8 patients were analyzed in the ivacaftor Arm because 8 patients were randomized to study drug and only 4 patients were randomized in the placebo arm because 4 patients received placebl
Arm/Group Title Ivacaftor (VX-770) Placebo
Hide Arm/Group Description:

twice a day administration of Ivacaftor: 150mg

Ivacaftor

matching placebo

Placebo

Overall Number of Participants Analyzed 8 4
Mean (Standard Deviation)
Unit of Measure: mmol/L
-8.0  (4.4) 2.0  (1.5)
2.Secondary Outcome
Title Change in COPD as Measured by Nasal Potential Difference
Hide Description 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.
Time Frame baseline to 2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Ivacaftor Placebo
Hide Arm/Group Description:
patients randomized to ivacaftor twice daily

matching placebo

Placebo

Overall Number of Participants Analyzed 8 4
Mean (Standard Deviation)
Unit of Measure: millivolts
-4.9  (3.9) 1.0  (6.4)
3.Secondary Outcome
Title Number of Adverse Events Experienced by the Ivacaftor Subjects and Placebo Subjects.
Hide Description Number of adverse events per subject in each the Ivacaftor subjects and placebo subjects
Time Frame baseline to 2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Ivacaftor (VX-770) Placebo
Hide Arm/Group Description:

twice a day administration of Ivacaftor: 150mg

Ivacaftor

matching placebo

Placebo

Overall Number of Participants Analyzed 8 4
Measure Type: Number
Unit of Measure: adverse events
22 14
4.Secondary Outcome
Title Change in COPD as Measured by Change in Percentage of FEV1 as Measured in Each Group
Hide Description 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.
Time Frame baseline to 2 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Ivacaftor (VX-770) Placebo
Hide Arm/Group Description:

twice a day administration of Ivacaftor: 150mg

Ivacaftor

matching placebo

Placebo

Overall Number of Participants Analyzed 8 4
Mean (Standard Deviation)
Unit of Measure: percentage of FEV1
-2.3  (5.2) 1.8  (9.3)
5.Other Pre-specified Outcome
Title Pharmacokinetics as Described by AUC12 of Subjects Receiving Ivacaftor
Hide Description [Not Specified]
Time Frame baseline to 2 weeks
Outcome Measure Data Not Reported
Time Frame 1 year, 5 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Ivacaftor (VX-770) Placebo
Hide Arm/Group Description

twice a day administration of Ivacaftor: 150mg

Ivacaftor

matching placebo

Placebo

All-Cause Mortality
Ivacaftor (VX-770) Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Hide Serious Adverse Events
Ivacaftor (VX-770) Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/8 (12.50%)      0/4 (0.00%)    
Respiratory, thoracic and mediastinal disorders     
COPD Exacerbation   1/8 (12.50%)  1 0/4 (0.00%)  0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Ivacaftor (VX-770) Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/8 (87.50%)      3/4 (75.00%)    
Gastrointestinal disorders     
Loose Stools   2/8 (25.00%)  2 0/4 (0.00%)  0
General disorders     
Headache   2/8 (25.00%)  2 0/4 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
decreased/diminished breath sounds   4/8 (50.00%)  4 3/4 (75.00%)  3
COPD Exacerbation   3/8 (37.50%)  3 0/4 (0.00%)  0
Indicates events were collected by systematic assessment
this pilot trial was under powered and potentially too brief to detect definitive changes in lung function
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Steven M. Rowe
Organization: University of Alabama at Birmingham
Phone: 205-975-6385
EMail: smrowe@uab.edu
Layout table for additonal information
Responsible Party: Dr. Steven M Rowe, University of Alabama at Birmingham
ClinicalTrials.gov Identifier: NCT02135432    
Other Study ID Numbers: F140319002
TOPIC Trial ( Other Identifier: UAB )
First Submitted: April 15, 2014
First Posted: May 12, 2014
Results First Submitted: May 25, 2016
Results First Posted: February 24, 2017
Last Update Posted: February 24, 2017