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Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation (ENVISION)

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ClinicalTrials.gov Identifier: NCT00909727
Recruitment Status : Completed
First Posted : May 28, 2009
Results First Posted : August 21, 2012
Last Update Posted : August 21, 2012
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 52
Recruitment Details Part A started on 05 August 2009 (signing of first informed consent). Screening evaluations were completed during Day -28 to Day -2. All subjects completing Part A were offered the opportunity to participate in Part B, which started on 12 March 2010. Screening evaluations were completed during Day -35 to Day -15 before the first dose of study drug.
Pre-assignment Details Nine subjects were dosed and included in Part A. In Part B, 52 subjects were enrolled and all were randomized to ivacaftor (26 subjects) or placebo (26 subjects). 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 26 [1] 26 [2]
Completed Treatment Period, Week 24 23 26
Completed 22 [3] 26 [3]
Not Completed 4 0
Reason Not Completed
Adverse Event             1             0
Wrong Genotype             1             0
Withdrawal of Consent             1             0
Prohibited Medication             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 26 26 52
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 26 participants 26 participants 52 participants
8.9  (1.86) 8.9  (2.00) 8.9  (1.91)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 26 participants 26 participants 52 participants
6 to 8 Years 13 12 25
9 to 11 Years 12 11 23
> 11 Years 1 3 4
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants 26 participants 52 participants
Female
10
  38.5%
17
  65.4%
27
  51.9%
Male
16
  61.5%
9
  34.6%
25
  48.1%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 26 participants 26 participants 52 participants
White 23 22 45
Other 1 2 3
Not Allowed to Ask Per Local Regulations 2 2 4
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 26 participants 26 participants 52 participants
Hispanic or Latino 0 1 1
Not Hispanic or Latino 24 23 47
Not Allowed to Ask Per Local Regulations 2 2 4
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 26 participants 26 participants 52 participants
North America 15 12 27
Europe 5 6 11
Australia 6 8 14
Weight  
Mean (Standard Deviation)
Unit of measure:  Kilograms
Number Analyzed 26 participants 26 participants 52 participants
30.0  (7.16) 31.8  (9.95) 30.9  (8.63)
Body Mass Index  
Mean (Standard Deviation)
Unit of measure:  Kilograms per square meter
Number Analyzed 26 participants 26 participants 52 participants
16.8  (1.75) 17.1  (2.61) 17.0  (2.21)
Sweat Chloride  
Mean (Standard Deviation)
Unit of measure:  Millimoles per liter
Number Analyzed 26 participants 26 participants 52 participants
104.8  (8.87) 104.3  (14.54) 104.6  (11.92)
Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 26 participants 26 participants 52 participants
< 70% 8 4 12
≥ 70% to ≤ 90% 6 12 18
> 90% 12 10 22
[1]
Measure Description: Percent predicted for age, gender, and height
Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)   [1] 
Mean (Standard Deviation)
Unit of measure:  Percentage
Number Analyzed 26 participants 26 participants 52 participants
83.7  (20.37) 84.7  (15.83) 84.2  (18.07)
[1]
Measure Description: Percent predicted for age, gender, and height
1.Primary Outcome
Title Absolute 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 25 26
Least Squares Mean (Standard Error)
Unit of Measure: percent of predicted volume (L)
0.1  (2.1) 12.6  (2.1)
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 value of percent predicted FEV1.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments The primary endpoint and key secondary endpoints were tested in sequence. test 1: primary (α=0.05); test 2: using Hochberg's step-up procedure on weight (Wk 24) and sweat chloride (Wk 24)(α=0.05); test 3: CFQ-R respiratory domain score (Wk 24).
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 12.5
Confidence Interval (2-Sided) 95%
6.6 to 18.3
Parameter Dispersion
Type: Standard Error of the mean
Value: 2.9
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (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 tablet of 150 mg of ivacaftor q12h for up to 48 weeks
Overall Number of Participants Analyzed 25 26
Least Squares Mean (Standard Error)
Unit of Measure: percent of predicted volume (L)
0.7  (2.0) 10.7  (1.9)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, 150 mg Ivacaftor q12h
Comments Analysis for this variable was similar to that of the primary analysis of the primary efficacy endpoint, a Mixed-Effects Model for Repeated Measures (MMRM). Estimates were obtained from MMRM with dependent variable absolute change from baseline, fixed effects for categorical visit & treatment group, & adjustment for the continuous baseline value of percent predicted FEV1, using unstructured covariance matrix.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0006
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 10.0
Confidence Interval (2-Sided) 95%
4.5 to 15.5
Parameter Dispersion
Type: Standard Error of the mean
Value: 2.7
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Through Week 24 and Week 48 (Respiratory Domain Score, Children)
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 25 26
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
Change from Baseline Through Week 24 0.3  (2.6) 6.3  (2.5)
Change from Baseline Through Week 48 1.0  (2.3) 6.1  (2.2)
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, a Mixed-Effects Model for Repeated Measures (MMRM), with the addition of the baseline domain score as a covariate.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1092
Comments The primary endpoint and key secondary endpoints were tested in sequence. test 1: primary (α=0.05); test 2: using Hochberg's step-up procedure on weight (Wk 24) and sweat chloride (Wk 24)(α=0.05); test 3: CFQ-R respiratory domain score (Wk 24).
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 6.1
Confidence Interval (2-Sided) 95%
-1.4 to 13.5
Parameter Dispersion
Type: Standard Error of the mean
Value: 3.7
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, a Mixed-Effects Model for Repeated Measures (MMRM), with the addition of the baseline domain score as a covariate.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1354
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 5.1
Confidence Interval (2-Sided) 95%
-1.6 to 11.8
Parameter Dispersion
Type: Standard Error of the mean
Value: 3.3
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 23 23
Least Squares Mean (Standard Error)
Unit of Measure: millimoles per liter
Change from Baseline Through Week 24 -1.2  (2.6) -55.5  (2.6)
Change from Baseline Through Week 48 -2.6  (2.6) -56.0  (2.5)
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 sweat chloride 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 The primary endpoint and key secondary endpoints were tested in sequence. test 1: primary (α=0.05); test 2: using Hochberg's step-up procedure on weight (Wk 24) and sweat chloride (Wk 24)(α=0.05); test 3: CFQ-R respiratory domain score (Wk 24).
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -54.3
Confidence Interval (2-Sided) 95%
-61.8 to -46.8
Parameter Dispersion
Type: Standard Error of the mean
Value: 3.7
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 sweat chloride 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 [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -53.5
Confidence Interval (2-Sided) 95%
-60.9 to -46.0
Parameter Dispersion
Type: Standard Error of the mean
Value: 3.7
Estimation Comments [Not Specified]
5.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 26 26
Least Squares Mean (Standard Error)
Unit of Measure: kilograms
At Week 24 1.8  (0.4) 3.7  (0.4)
At Week 48 3.1  (0.5) 5.9  (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 Effect (LME) model with dependent variable weight; treatment as a fixed effect; and intercept, visit, and treatment by visit interaction as random effects, with adjustment for 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 0.0004
Comments The primary endpoint and key secondary endpoints were tested in sequence. test 1: primary (α=0.05); test 2: using Hochberg's step-up procedure on weight (Wk 24) and sweat chloride (Wk 24)(α=0.05); test 3: CFQ-R respiratory domain score (Wk 24).
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.9
Confidence Interval (2-Sided) 95%
0.9 to 2.9
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 Effect (LME) model with random intercept and random slope, treatment as a fixed effect, and visit (days on study) and treatment by visit interaction as random effects, with adjustment for categorical 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 0.0002
Comments P-value is for the treatment effect at Week 48 (obtained as a linear contrast of treatment at Day 336). 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 2.8
Confidence Interval (2-Sided) 95%
1.3 to 4.2
Parameter Dispersion
Type: Standard Error of the mean
Value: 0.7
Estimation Comments [Not Specified]
Time Frame For enrolled subjects, adverse events (AEs) were collected through the Follow-up visit in each study part. For subjects who completed 48 weeks of treatment and enrolled in the open-label extension study, AEs were only collected through the Week 48 visit.
Adverse Event Reporting Description [Not Specified]
 
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   6/26 (23.08%)      5/26 (19.23%)    
Congenital, familial and genetic disorders     
Cystic fibrosis lung  1 [1]  3/26 (11.54%)  3 2/26 (7.69%)  2
Gastrointestinal disorders     
Abdominal pain  1  0/26 (0.00%)  0 1/26 (3.85%)  1
Constipation  1  1/26 (3.85%)  1 0/26 (0.00%)  0
General disorders     
Pyrexia  1  0/26 (0.00%)  0 1/26 (3.85%)  1
Infections and infestations     
Pseudomonas infection  1  1/26 (3.85%)  1 0/26 (0.00%)  0
Injury, poisoning and procedural complications     
Muscle strain  1  0/26 (0.00%)  0 1/26 (3.85%)  1
Investigations     
Hepatic enzyme increased  1  0/26 (0.00%)  0 1/26 (3.85%)  1
Pulmonary function test decreased  1  1/26 (3.85%)  1 0/26 (0.00%)  0
Psychiatric disorders     
Adjustment disorder  1  1/26 (3.85%)  1 0/26 (0.00%)  0
Anxiety  1  1/26 (3.85%)  1 0/26 (0.00%)  0
Affective disorder  1  1/26 (3.85%)  1 0/26 (0.00%)  0
Conversion disorder  1  0/26 (0.00%)  0 1/26 (3.85%)  1
Respiratory, thoracic and mediastinal disorders     
Productive cough  1  1/26 (3.85%)  1 1/26 (3.85%)  1
Lung consolidation  1  1/26 (3.85%)  1 0/26 (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   25/26 (96.15%)      26/26 (100.00%)    
Blood and lymphatic system disorders     
Lymphadenopathy  1  2/26 (7.69%)  2 1/26 (3.85%)  1
Congenital, familial and genetic disorders     
Cystic fibrosis lung  1 [1]  6/26 (23.08%)  8 7/26 (26.92%)  9
Gastrointestinal disorders     
Abdominal pain upper  1  5/26 (19.23%)  5 6/26 (23.08%)  9
Vomiting  1  7/26 (26.92%)  11 2/26 (7.69%)  3
Abdominal pain  1  3/26 (11.54%)  5 3/26 (11.54%)  4
Constipation  1  2/26 (7.69%)  2 2/26 (7.69%)  2
Diarrhoea  1  0/26 (0.00%)  0 3/26 (11.54%)  3
Gastrooesophageal reflux disease  1  0/26 (0.00%)  0 2/26 (7.69%)  2
Nausea  1  2/26 (7.69%)  2 0/26 (0.00%)  0
General disorders     
Pyrexia  1  7/26 (26.92%)  16 6/26 (23.08%)  6
Fatigue  1  2/26 (7.69%)  2 1/26 (3.85%)  1
Immune system disorders     
Seasonal allergy  1  2/26 (7.69%)  2 1/26 (3.85%)  1
Infections and infestations     
Nasopharyngitis  1  2/26 (7.69%)  3 6/26 (23.08%)  8
Upper respiratory tract infection  1  2/26 (7.69%)  2 6/26 (23.08%)  9
Bronchitis  1  2/26 (7.69%)  2 3/26 (11.54%)  4
Otitis media  1  1/26 (3.85%)  2 4/26 (15.38%)  7
Sinusitis  1  3/26 (11.54%)  4 2/26 (7.69%)  3
Ear infection  1  2/26 (7.69%)  2 0/26 (0.00%)  0
Pharyngitis streptococcal  1  0/26 (0.00%)  0 2/26 (7.69%)  2
Rhinitis  1  0/26 (0.00%)  0 2/26 (7.69%)  3
Injury, poisoning and procedural complications     
Excoriation  1  2/26 (7.69%)  3 1/26 (3.85%)  2
Investigations     
Alanine aminotransferase increased  1  3/26 (11.54%)  6 2/26 (7.69%)  2
Aspartate aminotransferase increased  1  2/26 (7.69%)  3 3/26 (11.54%)  3
Bacteria sputum identified  1  3/26 (11.54%)  3 2/26 (7.69%)  2
Pulmonary function test decreased  1  3/26 (11.54%)  4 2/26 (7.69%)  2
Eosinophil count increased  1  1/26 (3.85%)  1 3/26 (11.54%)  5
Culture throat positive  1  1/26 (3.85%)  1 2/26 (7.69%)  2
Breath sounds abnormal  1  2/26 (7.69%)  3 0/26 (0.00%)  0
Forced expiratory volume decreased  1  0/26 (0.00%)  0 2/26 (7.69%)  2
Neutrophil count decreased  1  0/26 (0.00%)  0 2/26 (7.69%)  3
White blood cell count decreased  1  0/26 (0.00%)  0 2/26 (7.69%)  3
Musculoskeletal and connective tissue disorders     
Myalgia  1  0/26 (0.00%)  0 2/26 (7.69%)  4
Neck pain  1  0/26 (0.00%)  0 2/26 (7.69%)  2
Nervous system disorders     
Headache  1  4/26 (15.38%)  8 7/26 (26.92%)  15
Respiratory, thoracic and mediastinal disorders     
Cough  1  19/26 (73.08%)  40 13/26 (50.00%)  21
Oropharyngeal pain  1  4/26 (15.38%)  9 7/26 (26.92%)  10
Nasal congestion  1  4/26 (15.38%)  7 5/26 (19.23%)  7
Rhinorrhoea  1  4/26 (15.38%)  8 3/26 (11.54%)  3
Wheezing  1  4/26 (15.38%)  4 3/26 (11.54%)  4
Productive cough  1  4/26 (15.38%)  5 2/26 (7.69%)  2
Rales  1  4/26 (15.38%)  5 2/26 (7.69%)  2
Pharyngeal erythema  1  0/26 (0.00%)  0 2/26 (7.69%)  2
Respiratory tract congestion  1  2/26 (7.69%)  3 0/26 (0.00%)  0
Rhinitis allergic  1  0/26 (0.00%)  0 2/26 (7.69%)  2
Skin and subcutaneous tissue disorders     
Rash  1  3/26 (11.54%)  3 2/26 (7.69%)  3
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
Layout table for Results Point of Contact information
Name/Title: Medical Monitor
Organization: Vertex
Phone: 617-444-6777
EMail: medicalinfo@vrtx.com
Layout table for additonal information
Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT00909727     History of Changes
Other Study ID Numbers: VX08-770-103
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: August 21, 2012