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(Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability In The Sweat Gland Secretory Coil?

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ClinicalTrials.gov Identifier: NCT02310789
Recruitment Status : Completed
First Posted : December 8, 2014
Results First Posted : April 20, 2018
Last Update Posted : January 9, 2019
Sponsor:
Information provided by (Responsible Party):
Richard Barry Moss, Stanford University

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Basic Science
Condition Cystic Fibrosis
Interventions Drug: Ivacaftor
Drug: β-Adrenergic cocktail
Drug: Pilocarpine Nitrate 5%
Device: Macroduct sweat stimulator
Enrollment 8
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Ivacaftor
Hide Arm/Group Description Participants received ivacaftor orally for 3 days, followed by 35 days off drug. Participants repeated this cycle then received ivacaftor for 3 additional days. For sweat testing, participants received β-adrenergic cocktail to stimulated sweating, at both 1% stimulation strength and full stimulation strength. Each participant also received pilocarpine nitrate 5% administered by Macroduct sweat stimulator device. Sweat stimulation testing was done on- and off-ivacaftor.
Period Title: Overall Study
Started 8
Completed 7
Not Completed 1
Reason Not Completed
Adverse Event             1
Arm/Group Title Ivacaftor
Hide Arm/Group Description Participants received ivacaftor orally for 3 days, followed by 35 days off drug. Participants repeated this cycle then received ivacaftor for 3 additional days. For sweat testing, participants received β-adrenergic cocktail to stimulated sweating, at both 1% stimulation strength and full stimulation strength. Each participant also received pilocarpine nitrate 5% administered by Macroduct sweat stimulator device. Sweat stimulation testing was done on- and off-ivacaftor.
Overall Number of Baseline Participants 8
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
<=18 years
0
   0.0%
Between 18 and 65 years
7
  87.5%
>=65 years
1
  12.5%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 8 participants
46.5
(20 to 75)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Female
5
  62.5%
Male
3
  37.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Hispanic or Latino
1
  12.5%
Not Hispanic or Latino
7
  87.5%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
8
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 8 participants
8
Healthy Volunteers  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
8
 100.0%
1.Primary Outcome
Title Change in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Dependent Sweat Rate
Hide Description CFTR-dependent sweat rate (C-sweat) was analyzed using a linear mixed model, combining on- and off-ivacaftor data.
Time Frame Up to 79 days
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with available data were included in the analysis.
Arm/Group Title Ivacaftor
Hide Arm/Group Description:
Participants received ivacaftor orally for 3 days, followed by 35 days off drug. Participants repeated this cycle then received ivacaftor for 3 additional days. For sweat testing, participants received β-adrenergic cocktail to stimulated sweating, at both 1% stimulation strength and full stimulation strength. Each participant also received pilocarpine nitrate 5% administered by Macroduct sweat stimulator device. Sweat stimulation testing was done on- and off-ivacaftor.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: percent change
1% β-adrenergic stimulation 16.45  (2.25)
Full β-adrenergic stimulation 6.60  (2.97)
2.Secondary Outcome
Title Change Sweat Chloride Production
Hide Description Sweat chloride concentration was measured via the traditional sweat collection methods using the pilocarpine stimulation with the Macroduct device.
Time Frame Up to 79 days
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with available data were included in the analysis.
Arm/Group Title Ivacaftor
Hide Arm/Group Description:
Participants received ivacaftor orally for 3 days, followed by 35 days off drug. Participants repeated this cycle then received ivacaftor for 3 additional days. For sweat testing, participants received β-adrenergic cocktail to stimulated sweating, at both 1% stimulation strength and full stimulation strength. Each participant also received pilocarpine nitrate 5% administered by Macroduct sweat stimulator device. Sweat stimulation testing was done on- and off-ivacaftor.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: percent change
On ivacaftor 25.1  (11.4)
Off Ivacaftor 24.9  (6.1)
Time Frame Up to 79 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Ivacaftor
Hide Arm/Group Description Participants received ivacaftor orally for 3 days, followed by 35 days off drug. Participants repeated this cycle then received ivacaftor for 3 additional days. For sweat testing, participants received β-adrenergic cocktail to stimulated sweating, at both 1% stimulation strength and full stimulation strength. Each participant also received pilocarpine nitrate 5% administered by Macroduct sweat stimulator device. Sweat stimulation testing was done on- and off-ivacaftor.
All-Cause Mortality
Ivacaftor
Affected / at Risk (%)
Total   0/8 (0.00%) 
Hide Serious Adverse Events
Ivacaftor
Affected / at Risk (%)
Total   0/8 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Ivacaftor
Affected / at Risk (%)
Total   1/8 (12.50%) 
Skin and subcutaneous tissue disorders   
Subcutaneous induration   1/8 (12.50%) 
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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: Jeff Wine, PhD
Organization: Stanford University
Phone: 650-725-2462
EMail: wine@stanford.edu
Layout table for additonal information
Responsible Party: Richard Barry Moss, Stanford University
ClinicalTrials.gov Identifier: NCT02310789    
Other Study ID Numbers: 31238
First Submitted: September 3, 2014
First Posted: December 8, 2014
Results First Submitted: December 12, 2017
Results First Posted: April 20, 2018
Last Update Posted: January 9, 2019