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Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation (STRIVE)

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ClinicalTrials.gov Identifier: NCT00909532
Recruitment Status : Completed
First Posted : May 28, 2009
Results First Posted : August 21, 2012
Last Update Posted : January 18, 2013
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Interventions Drug: Ivacaftor
Drug: Placebo
Enrollment 167
Recruitment Details The study started on 10 June 2009 (signing of first informed consent). After obtaining consent and assent (where applicable), screening evaluations were completed during a period of 2 to 5 weeks (Day -35 to Day -15) before the first dose of study drug.
Pre-assignment Details A total of 167 subjects were randomized; 161 subjects received at least 1 dose of the study drug. A 2-week run-in period was included to establish the baseline assessments on Day 1 after ensuring that subjects were properly taking their cystic fibrosis (CF) medication regimens.
Arm/Group Title Placebo 150 mg Ivacaftor q12h
Hide Arm/Group Description Oral tablet every 12 hours (q12h) for up to 48 weeks. Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.
Period Title: Overall Study
Started 78 [1] 83 [2]
Completed Treatment Period, Week 24 71 80
Completed 68 [3] 77 [3]
Not Completed 10 6
Reason Not Completed
Adverse Event             4             1
Pregnancy             0             1
Prohibited Medication             2             1
Withdrawal of Consent             1             1
Noncompliance with Study Requirements             0             2
Physician Decision             1             0
Wrong Genotype             1             0
Increased Lab Draws, Difficult Lab Stick             1             0
[1]
All subjects who received at least 1 dose of study drug (placebo).
[2]
All subjects who received at least 1 dose of study drug (ivacaftor).
[3]
Completed Treatment and Extension Periods (Through Week 48)
Arm/Group Title Placebo 150 mg Ivacaftor q12h Total
Hide Arm/Group Description Oral tablet every 12 hours (q12h) for up to 48 weeks. Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks. Total of all reporting groups
Overall Number of Baseline Participants 78 83 161
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 78 participants 83 participants 161 participants
<=18 years
17
  21.8%
19
  22.9%
36
  22.4%
Between 18 and 65 years
61
  78.2%
64
  77.1%
125
  77.6%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 78 participants 83 participants 161 participants
24.7  (9.21) 26.2  (9.85) 25.5  (9.54)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 78 participants 83 participants 161 participants
Female
40
  51.3%
44
  53.0%
84
  52.2%
Male
38
  48.7%
39
  47.0%
77
  47.8%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 78 participants 83 participants 161 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
77
  98.7%
81
  97.6%
158
  98.1%
Unknown or Not Reported
1
   1.3%
2
   2.4%
3
   1.9%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 78 participants 83 participants 161 participants
White 77 81 158
Not Allowed to Ask Per Local Regulations 1 2 3
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 78 participants 83 participants 161 participants
North America 50 50 100
Europe 19 23 42
Australia 9 10 19
Percent Predicted Forced Expiratory Volume in 1 Second (FEV1), Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Percentage
Number Analyzed 78 participants 83 participants 161 participants
63.7  (16.83) 63.5  (16.14) 63.6  (16.43)
[1]
Measure Description: Percent predicted for age, gender, and height.
Percent Predicted FEV1, Categorical   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 78 participants 83 participants 161 participants
< 70% predicted FEV1 45 49 94
≥ 70% predicted FEV1 33 34 67
[1]
Measure Description: Percent predicted for age, gender, and height.
Weight  
Mean (Standard Deviation)
Unit of measure:  Kilograms
Number Analyzed 78 participants 83 participants 161 participants
61.2  (13.93) 61.7  (14.26) 61.5  (14.06)
Body Mass Index  
Mean (Standard Deviation)
Unit of measure:  Kilograms per square meter
Number Analyzed 78 participants 83 participants 161 participants
21.9  (3.49) 21.7  (3.65) 21.8  (3.56)
Sweat Chloride  
Mean (Standard Deviation)
Unit of measure:  Millimoles per liter
Number Analyzed 78 participants 83 participants 161 participants
100.1  (10.63) 100.4  (10.00) 100.2  (10.28)
1.Primary Outcome
Title Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24
Hide Description Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
Time Frame baseline through 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.
Arm/Group Title Placebo 150 mg Ivacaftor q12h
Hide Arm/Group Description:
Oral tablet every 12 hours (q12h) for up to 48 weeks.
Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.
Overall Number of Participants Analyzed 78 83
Least Squares Mean (Standard Error)
Unit of Measure: percent of predicted volume (L)
-0.2  (0.7) 10.4  (0.7)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments The primary analysis for the primary efficacy variable was based on a Mixed-Effects Model for Repeated Measures (MMRM). The model included absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) as the dependent variable, treatment (ivacaftor versus placebo) and visit (Day 15, Week 8, Week 16, and Week 24) as fixed effects, and subject as a random effect, with adjustment for the continuous baseline values of age and percent predicted FEV1.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments The primary and key secondary endpoints were analyzed using Hochberg’s step-up procedure: test 1, primary (α=0.05); test 2, CFQ-R resp domain (Wk24) and sweat chloride (Wk24)(α=0.05).
Method Mixed Models Analysis
Comments Denominator degrees of freedom were estimated using the Kenward-Roger approximation.
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 10.6
Confidence Interval (2-Sided) 95%
8.6 to 12.6
Parameter Dispersion
Type: Standard Error of the mean
Value: 1.0
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48
Hide Description Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
Time Frame baseline through 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame..
Arm/Group Title Placebo 150 mg Ivacaftor q12h
Hide Arm/Group Description:
Oral tablet every 12 hours (q12h) for up to 48 weeks.
Oral tablets of 150 mg of ivacaftor q12h for up to 48 weeks.
Overall Number of Participants Analyzed 78 83
Least Squares Mean (Standard Error)
Unit of Measure: percent of predicted volume (L)
-0.4  (0.7) 10.1  (0.7)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments Analysis of this variable was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were obtained from Mixed-Effects Model for Repeated Measures (MMRM) with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for the continuous baseline values of age and percent predicted forced expiratory volume in 1 second (FEV1),using unstructured covariance matrix.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments There was no adjustment for multiple comparisons.
Method Mixed Models Analysis
Comments Denominator degrees of freedom were estimated using the Kenward-Roger approximation. No imputation of missing data was done.
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 10.5
Confidence Interval (2-Sided) 95%
8.5 to 12.5
Parameter Dispersion
Type: Standard Error of the mean
Value: 1.0
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled)
Hide Description The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID).
Time Frame baseline through 24 weeks and 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.
Arm/Group Title Placebo 150 mg Ivacaftor q12h
Hide Arm/Group Description:
Oral tablet every 12 hours (q12h) for up to 48 weeks.
Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.
Overall Number of Participants Analyzed 71 80
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
Change from Baseline Through Week 24 -2.1  (1.3) 6.0  (1.2)
Change from Baseline Through Week 48 -2.7  (1.2) 6.0  (1.1)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments Through Week 24: Analysis for the respiratory domain score endpoint was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were from Mixed-Effects Model for Repeated Measures (MMRM) with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for continuous baseline value for age, domain score, and percent predicted FEV1, using unstructured covariance matrix.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Analyzed in sequence: test 1, primary (α=0.05); test 2, using Hochberg’s step-up procedure on CFQ-R resp domain(Wk 24) and sweat chloride (Wk 24) (α=0.05).
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 8.1
Confidence Interval (2-Sided) 95%
4.7 to 11.4
Parameter Dispersion
Type: Standard Error of the mean
Value: 1.7
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments Through Week 48: Analysis for the CFQ-R respiratory domain score endpoint was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were from MMRM with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for continuous baseline value for age,sweat chloride, and percent predicted FEV1,using unstructured covariance matrix.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments There was no adjustment for multiple comparisons.
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 8.6
Confidence Interval (2-Sided) 95%
5.3 to 11.9
Parameter Dispersion
Type: Standard Error of the mean
Value: 1.7
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48
Hide Description The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity.
Time Frame baseline through 24 weeks and 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame..
Arm/Group Title Placebo 150 mg Ivacaftor q12h
Hide Arm/Group Description:
Oral tablet every 12 hours (q12h) for up to 48 weeks.
Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.
Overall Number of Participants Analyzed 74 78
Least Squares Mean (Standard Error)
Unit of Measure: millimoles per liter
Change from Baseline Through Week 24 -0.8  (1.3) -48.7  (1.2)
Change from Baseline Through Week 48 -0.6  (1.3) -48.7  (1.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments Through Week 24: Analysis for this variable was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were from Mixed-Effects Model for Repeated Measures (MMRM) with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for continuous baseline value for age, sweat chloride, and percent predicted FEV1, using unstructured covariance matrix.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments Analyzed in sequence: test 1, primary (α=0.05); test 2, using Hochberg’s step-up procedure on CFQ-R resp domain(Wk 24) and sweat chloride (Wk 24) (α=0.05); test 3 using Hochberg’s on time to pulmonary exacerbation (Wk 48) and weight (Wk 48).
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -47.9
Confidence Interval (2-Sided) 95%
-51.3 to -44.5
Parameter Dispersion
Type: Standard Error of the mean
Value: 1.7
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments Through Week 48: Analysis for this variable was similar to that of the primary analysis of the primary efficacy endpoint. Estimates were from Mixed-Effects Model for Repeated Measures (MMRM) with dependent variable absolute change from baseline, fixed effects for categorical visit and treatment group, and adjustment for continuous baseline value for age, sweat chloride, and percent predicted FEV1, using unstructured covariance matrix.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments There was no adjustment for multiple comparisons.
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -48.1
Confidence Interval (2-Sided) 95%
-51.5 to -44.7
Parameter Dispersion
Type: Standard Error of the mean
Value: 1.7
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48
Hide Description Pulmonary exacerbation was defined as a change in antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of signs/symptoms such as change in sputum; new or increased hemoptysis; increased cough or dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees C; anorexia or weight loss; sinus pain/tenderness and discharge; change in physical examination of the chest; decreased pulmonary function by 10%; and radiographic changes indicative of pulmonary infection.
Time Frame baseline through 24 weeks and 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.
Arm/Group Title Placebo 150 mg Ivacaftor q12h
Hide Arm/Group Description:
Oral tablet every 12 hours (q12h) for up to 48 weeks.
Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.
Overall Number of Participants Analyzed 78 83
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: proportion of event-free participants
0 to 15 Days
0.97
(0.90 to 0.99)
0.98
(0.91 to 0.99)
16 to 56 Days
0.87
(0.77 to 0.93)
0.89
(0.80 to 0.94)
57 to 112 Days
0.72
(0.61 to 0.81)
0.83
(0.73 to 0.90)
113 to 168 Days
0.53
(0.41 to 0.64)
0.78
(0.68 to 0.86)
169 to 224 Days
0.51
(0.39 to 0.61)
0.75
(0.64 to 0.83)
225 to 280 Days
0.44
(0.32 to 0.55)
0.70
(0.58 to 0.78)
281 to 336 Days
0.41
(0.29 to 0.52)
0.67
(0.55 to 0.76)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments Time to first pulmonary exacerbation through Week 24 was analyzed using Cox regression. The model included a covariate for treatment and adjustments for the age group and percent predicted forced expiratory volume in 1 second (FEV1) severity at baseline.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0016
Comments There was no adjustment for multiple comparisons.
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Cox Proportional Hazard at Week 24
Estimated Value 0.40
Confidence Interval (2-Sided) 95%
0.23 to 0.71
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments Time to first pulmonary exacerbation through Week 48 was analyzed using Cox regression. The model included a covariate for treatment and adjustments for the age group and percent predicted forced expiratory volume in 1 second (FEV1) severity at baseline.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0012
Comments Analyzed in sequence: test 1, primary (α=0.05); test 2, using Hochberg’s step-up procedure on CFQ-R resp domain(Wk 24) and sweat chloride (Wk 24) (α=0.05); test 3 using Hochberg’s on time to pulmonary exacerbation (Wk 48) and weight (Wk 48).
Method Regression, Cox
Comments [Not Specified]
Method of Estimation Estimation Parameter Cox Proportional Hazard at 48 Weeks
Estimated Value 0.46
Confidence Interval (2-Sided) 95%
0.28 to 0.73
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Absolute Change From Baseline in Weight at Week 24 and Week 48
Hide Description As malnutrition is common in patients with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status.
Time Frame baseline to 24 weeks and 48 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.
Arm/Group Title Placebo 150 mg Ivacaftor q12h
Hide Arm/Group Description:
Oral tablet every 12 hours (q12h) for up to 48 weeks.
Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.
Overall Number of Participants Analyzed 78 83
Least Squares Mean (Standard Error)
Unit of Measure: kilograms
At Week 24 0.2  (0.4) 3.0  (0.4)
At Week 48 0.4  (0.5) 3.1  (0.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments At Week 24: Analysis for this variable was based on a linear mixed effects (LME) model with treatment as a fixed effect, and intercept, visit (days on study) and treatment by visit interaction as random effects, with adjustment for age group and baseline percent predicted forced expiratory volume in 1 second (FEV1) severity.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Mixed Models Analysis
Comments There was no adjustment for multiple comparisons.
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 2.8
Confidence Interval (2-Sided) 95%
1.8 to 3.7
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.5
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments At Week 48: Analysis for this variable was based on a linear mixed effects (LME) model with treatment as a fixed effect and visit (days on study) and treatment by visit interaction as random effects, with adjustment for age group and baseline percent predicted forced expiratory volume in 1 second (FEV1) severity.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments Analyzed in sequence: test 1, primary (α=0.05); test 2, using Hochberg’s step-up procedure on CFQ-R resp domain(Wk 24) and sweat chloride (Wk 24) (α=0.05).
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 2.7
Confidence Interval (2-Sided) 95%
1.3 to 4.1
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.7
Estimation Comments [Not Specified]
Time Frame For enrolled subjects, adverse events were collected through the Follow-up Visit (4 weeks [± 7 days] after the last dose of study drug).
Adverse Event Reporting Description For subjects who were screened but were not subsequently enrolled in the study, non-serious AEs were not collected, but SAEs were reported. For subjects who completed 48 weeks of study drug treatment and enrolled in the open-label extension study, adverse events were only collected through the Week 48 Visit.
 
Arm/Group Title Placebo 150 mg Ivacaftor q12h
Hide Arm/Group Description Oral tablet every 12 hours (q12h) for up to 48 weeks. Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks.
All-Cause Mortality
Placebo 150 mg Ivacaftor q12h
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Placebo 150 mg Ivacaftor q12h
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   33/78 (42.31%)      20/83 (24.10%)    
Cardiac disorders     
Atrioventricular block complete  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Congenital, familial and genetic disorders     
Cystic fibrosis lung  1 [1]  26/78 (33.33%)  37 11/83 (13.25%)  22
Gastrointestinal disorders     
Gastrooesophageal reflux disease  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Pancreatic pseudocyst  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Pancreatitis  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Vomiting  1  1/78 (1.28%)  1 0/83 (0.00%)  0
General disorders     
Catheter related complication  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Immune system disorders     
Anaphylactic shock  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Infections and infestations     
Implant site infection  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Influenza  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Lung infection pseudomonal  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Myringitis bullous  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Pneumonia  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Sinusitis  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Investigations     
Hepatic enzyme increased  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Weight decreased  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Metabolism and nutrition disorders     
Hypoglycaemia  1  0/78 (0.00%)  0 2/83 (2.41%)  2
Musculoskeletal and connective tissue disorders     
Musculoskeletal chest pain  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Pain in extremity  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Cervix carcinoma  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Pregnancy, puerperium and perinatal conditions     
Abortion spontaneous  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Renal and urinary disorders     
Haematuria  1  0/78 (0.00%)  0 1/83 (1.20%)  1
IgA nephropathy  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Nephrolithiasis  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Renal colic  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Reproductive system and breast disorders     
Testicular torsion  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Cough  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Hemoptysis  1  4/78 (5.13%)  4 1/83 (1.20%)  1
Pleuritic pain  1  0/78 (0.00%)  0 1/83 (1.20%)  1
Pulmonary embolism  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Respiratory distress  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Respiratory failure  1  1/78 (1.28%)  1 0/83 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 12.0
[1]
CF exacerbations were coded as "cystic fibrosis lung."
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo 150 mg Ivacaftor q12h
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   78/78 (100.00%)      82/83 (98.80%)    
Congenital, familial and genetic disorders     
Cystic fibrosis lung  1 [1]  39/78 (50.00%)  77 29/83 (34.94%)  47
Gastrointestinal disorders     
Abdominal pain  1  10/78 (12.82%)  13 13/83 (15.66%)  14
Nausea  1  9/78 (11.54%)  12 13/83 (15.66%)  22
Diarrhoea  1  10/78 (12.82%)  12 11/83 (13.25%)  12
Vomiting  1  10/78 (12.82%)  10 9/83 (10.84%)  13
Abdominal pain upper  1  6/78 (7.69%)  7 4/83 (4.82%)  5
Constipation  1  5/78 (6.41%)  5 1/83 (1.20%)  1
General disorders     
Pyrexia  1  9/78 (11.54%)  11 10/83 (12.05%)  13
Fatigue  1  7/78 (8.97%)  9 7/83 (8.43%)  8
Infections and infestations     
Upper respiratory tract infection  1  12/78 (15.38%)  16 19/83 (22.89%)  26
Nasopharyngitis  1  10/78 (12.82%)  14 10/83 (12.05%)  13
Sinusitis  1  7/78 (8.97%)  12 6/83 (7.23%)  8
Rhinitis  1  4/78 (5.13%)  4 6/83 (7.23%)  6
Viral infection  1  5/78 (6.41%)  5 2/83 (2.41%)  2
Injury, poisoning and procedural complications     
Joint sprain  1  4/78 (5.13%)  5 0/83 (0.00%)  0
Investigations     
Pulmonary function test decreased  1  11/78 (14.10%)  14 3/83 (3.61%)  3
Alanine aminotransferase increased  1  5/78 (6.41%)  6 5/83 (6.02%)  9
C-reactive protein increased  1  5/78 (6.41%)  6 4/83 (4.82%)  4
Blood glucose increased  1  3/78 (3.85%)  3 5/83 (6.02%)  6
Aspartate aminotransferase increased  1  2/78 (2.56%)  2 5/83 (6.02%)  7
Bacteria sputum identified  1  1/78 (1.28%)  1 6/83 (7.23%)  7
Breath sounds abnormal  1  4/78 (5.13%)  5 2/83 (2.41%)  2
Weight decreased  1  4/78 (5.13%)  4 2/83 (2.41%)  2
Metabolism and nutrition disorders     
Hypoglycaemia  1  4/78 (5.13%)  4 3/83 (3.61%)  3
Musculoskeletal and connective tissue disorders     
Arthralgia  1  5/78 (6.41%)  5 7/83 (8.43%)  11
Back pain  1  5/78 (6.41%)  7 5/83 (6.02%)  5
Nervous system disorders     
Headache  1  13/78 (16.67%)  31 19/83 (22.89%)  39
Dizziness  1  1/78 (1.28%)  1 10/83 (12.05%)  11
Sinus headache  1  4/78 (5.13%)  4 6/83 (7.23%)  6
Respiratory, thoracic and mediastinal disorders     
Cough  1  33/78 (42.31%)  59 27/83 (32.53%)  38
Oropharyngeal pain  1  15/78 (19.23%)  23 17/83 (20.48%)  25
Nasal congestion  1  12/78 (15.38%)  17 17/83 (20.48%)  22
Haemoptysis  1  15/78 (19.23%)  21 9/83 (10.84%)  17
Productive cough  1  11/78 (14.10%)  19 12/83 (14.46%)  15
Rales  1  8/78 (10.26%)  14 9/83 (10.84%)  17
Respiratory tract congestion  1  6/78 (7.69%)  9 6/83 (7.23%)  7
Sinus congestion  1  4/78 (5.13%)  4 8/83 (9.64%)  9
Rhinorrhoea  1  6/78 (7.69%)  6 4/83 (4.82%)  4
Paranasal sinus hypersecretion  1  6/78 (7.69%)  7 3/83 (3.61%)  3
Wheezing  1  3/78 (3.85%)  3 5/83 (6.02%)  8
Dyspnoea  1  5/78 (6.41%)  5 2/83 (2.41%)  2
Pleuritic pain  1  2/78 (2.56%)  2 5/83 (6.02%)  7
Respiration abnormal  1  4/78 (5.13%)  5 2/83 (2.41%)  2
Skin and subcutaneous tissue disorders     
Rash  1  4/78 (5.13%)  4 12/83 (14.46%)  22
Acne  1  3/78 (3.85%)  3 6/83 (7.23%)  6
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 12.0
[1]
CF exacerbations were coded as "cystic fibrosis lung."
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
Name/Title: Medical Monitor
Organization: Vertex
Phone: 617-444-6777
Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT00909532     History of Changes
Other Study ID Numbers: VX08-770-102
First Submitted: May 26, 2009
First Posted: May 28, 2009
Results First Submitted: February 27, 2012
Results First Posted: August 21, 2012
Last Update Posted: January 18, 2013