KONTINUE: Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Completed
CT.gov ID
NCT01707290
Collaborator
Cystic Fibrosis Foundation (Other)
125
38
2
37.9
3.3
0.1
Study Details
Study Description
Brief Summary
The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Detailed Description
Ivacaftor is the first Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator to show an improvement in CFTR function and clinical benefit in participants with CF. Results from Phase 3 studies (NCT00909532 [Study 102] and NCT00909727 [Study 103]) showed that ivacaftor is effective in the treatment of participants with CF who have the G551D-CFTR mutation, as evidenced by sustained improvements in CFTR channel function (measured by reduction in sweat chloride concentration) and corresponding substantial, durable improvements in lung function, pulmonary exacerbations, respiratory symptoms, and weight gain. Ivacaftor was also well tolerated, as evidenced by the rates and reasons for premature discontinuation and results of safety assessments.
Ivacaftor (Trade Name Kalydeco; 150 mg tablets) was initially approved in the United States for the treatment of CF in participants 6 years of age and older who have a G551D mutation in the CFTR gene.
Study Design
Study Type:
Interventional
Actual Enrollment
:
125 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 3, Two-Arm, Rollover Study to Evaluate the Safety of Long Term Ivacaftor Treatment in Subjects 6 Years of Age and Older With Cystic Fibrosis and a Non-G551D CFTR Mutation
Study Start Date
:
Feb 1, 2013
Actual Primary Completion Date
:
Apr 1, 2016
Actual Study Completion Date
:
Apr 1, 2016
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Ivacaftor Participants who received Ivacaftor 150 milligram (mg) tablet and/or Placebo matched to Ivacaftor tablet orally, every 12 hours (q12h) in the previous study VX11-770-110 (Study 110; NCT01614457), VX12-770-111 (Study 111; NCT01614470) or VX12-770-113 (Study 113; NCT01685801); received Ivacaftor 150 mg tablet q12h in this VX12-770-112 (Study 112; NCT01707290) up to 104 weeks. |
Drug: Ivacaftor
150 mg tablet, oral use, every 12 hours (q12h)
Other Names:
|
| No Intervention: Observational Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet, orally, q12h in the previous Study 110 (NCT01614457) or Study 111 (NCT01614470), were observed (did not receive study drug) in this Study 112 (NCT01707290) for up to 2 years. |
Outcome Measures
Primary Outcome Measures
- Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm [Day 1 up to Week 108 (Study 112)]
AE: any untoward medical occurrence in a participants during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. TEAEs were defined as adverse events with start date or increased severity on and after the first dose of study drug through Week 108.
Secondary Outcome Measures
- Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 [Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)]
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 6 to 17 years and for female participants aged 6 to 15 years. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
- Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 [Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)]
BMI was defined as weight in kg divided by height in m^2. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
- Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 [Baseline, Week 2, 24, 48 and 104 (Study 112)]
Sweat samples were collected using an approved collection device. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
- Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 [Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)]
The CFQ-R is a validated participant reported outcome measuring health related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health related quality of life. Baseline was defined as the most recent measurement before intake of the first dose of study drug (ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
- Number of Pulmonary Exacerbations Events [Through Week 104 (Study 112)]
Pulmonary exacerbation events include those events which require treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. The number of events were reported. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
- Number of Participants With Serious Adverse Events (SAEs) in Observational Arm [up to 2 years (Study 112)]
SAE was defined as a medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event.
Eligibility Criteria
Criteria
Ages Eligible for Study:
6 Years
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
-
Participants from Study 110 or Study 111 entering the ivacaftor arm must have completed the assigned study drug treatment duration in the previous study.
-
Participants from Study 113 entering the ivacaftor arm must have completed all study related treatments through the Follow-up Visit and met the Study 113 responder criteria during the previous study.
-
Participants entering the observational arm must have completed at least 4 weeks of study drug treatment in their previous study (Study 110 or Study 111), must have completed the previous study but do not wish to enroll in the ivacaftor arm, or must have completed the previous study but do not meet the inclusion criteria of the ivacaftor arm.
-
Participants of childbearing potential entering the ivacaftor arm must not be pregnant.
-
Participants entering the ivacaftor arm must be willing to comply with contraception requirements.
Exclusion Criteria (Ivacaftor Arm Only):
-
History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the Participant.
-
Use of moderate or strong inhibitors or inducers of cytochrome P450 (CYP) 3A.
-
Evidence of cataract or lens opacity at or before the Day 1 Visit.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Birmingham | Alabama | United States | ||
| 2 | Palo Alto | California | United States | ||
| 3 | Denver | Colorado | United States | ||
| 4 | Hartford | Connecticut | United States | ||
| 5 | Tampa | Florida | United States | ||
| 6 | Atlanta | Georgia | United States | ||
| 7 | Chicago | Illinois | United States | ||
| 8 | Iowa City | Iowa | United States | ||
| 9 | Lexington | Kentucky | United States | ||
| 10 | Baltimore | Maryland | United States | ||
| 11 | Boston | Massachusetts | United States | ||
| 12 | Ann Arbor | Michigan | United States | ||
| 13 | Detroit | Michigan | United States | ||
| 14 | Grand Rapids | Michigan | United States | ||
| 15 | Minneapolis | Minnesota | United States | ||
| 16 | St. Louis | Missouri | United States | ||
| 17 | Omaha | Nebraska | United States | ||
| 18 | New York | New York | United States | ||
| 19 | Syracuse | New York | United States | ||
| 20 | Valhalla | New York | United States | ||
| 21 | Chapel Hill | North Carolina | United States | ||
| 22 | Philadelphia | Pennsylvania | United States | ||
| 23 | Pittsburgh | Pennsylvania | United States | ||
| 24 | Charleston | South Carolina | United States | ||
| 25 | Nashville | Tennessee | United States | ||
| 26 | Dallas | Texas | United States | ||
| 27 | Fort Worth | Texas | United States | ||
| 28 | Houston | Texas | United States | ||
| 29 | Salt Lake City | Utah | United States | ||
| 30 | Richmond | Virginia | United States | ||
| 31 | Seattle | Washington | United States | ||
| 32 | Morgantown | West Virginia | United States | ||
| 33 | Madison | Wisconsin | United States | ||
| 34 | Leuven | Belgium | |||
| 35 | Montpellier | France | |||
| 36 | Paris | France | |||
| 37 | Belfast | United Kingdom | |||
| 38 | Edinburgh | United Kingdom |
Sponsors and Collaborators
- Vertex Pharmaceuticals Incorporated
- Cystic Fibrosis Foundation
Investigators
- Principal Investigator: Joseph Pilewski, MD, University of Pittsburgh
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.Responsible Party:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01707290
Other Study ID Numbers:
- VX12-770-112
- 2012-000389-39
First Posted:
Oct 16, 2012
Last Update Posted:
May 12, 2017
Last Verified:
Apr 1, 2017
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail | The study consisted of 2 arms: Ivacaftor arm and Observational arm. The Ivacaftor arm enrolled subjects from Study VX11-770-110 (NCT01614457), Study VX12-770-111 (NCT01614470) and Study VX12-770-113 (NCT01685801). The Observational arm enrolled subjects from Study VX11-770-110 (NCT01614457) and Study VX12-770-111 (NCT01614470). |
| Arm/Group Title | Ivacaftor Arm | Observational Arm |
|---|---|---|
| Arm/Group Description | Participants who received Ivacaftor 150 milligram (mg) tablet and/or Placebo matched to Ivacaftor tablet, every 12 hours (q12h) in the previous study VX11-770-110 (Study 110; NCT01614457), VX12-770-111 (Study 111; NCT01614470) or VX12-770-113 (Study 113; NCT01685801); received Ivacaftor 150 mg tablet orally q12h in this VX12-770-112 (Study 112; NCT01707290) up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet q12h in the previous Study 110 (NCT01614457) or Study 111 (NCT01614470), were observed (did not receive study drug) in this Study 112 (NCT01707290) for up to 2 years. |
| Period Title: Overall Study | ||
| STARTED | 121 | 4 |
| COMPLETED | 35 | 3 |
| NOT COMPLETED | 86 | 1 |
Baseline Characteristics
| Arm/Group Title | Ivacaftor Arm | Observational Arm | Total |
|---|---|---|---|
| Arm/Group Description | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet, q12h in the previous study VX11-770-110 (Study 110; NCT01614457), VX12-770-111 (Study 111; NCT01614470) or VX12-770-113 (Study 113; NCT01685801); received Ivacaftor 150 mg tablet orally q12h in this VX12-770-112 (Study 112; NCT01707290) up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet q12h in the previous Study 110 (NCT01614457) or Study 111 (NCT01614470), were observed (did not receive study drug) in this Study 112 (NCT01707290) for up to 2 years. | Total of all reporting groups |
| Overall Participants | 121 | 4 | 125 |
| Age, Customized (participants) [Number] | |||
| 6 to 11 years |
21
17.4%
|
1
25%
|
22
17.6%
|
| 12 to 17 years |
11
9.1%
|
1
25%
|
12
9.6%
|
| 18 years and over |
89
73.6%
|
2
50%
|
91
72.8%
|
| Sex: Female, Male (Count of Participants) | |||
| Female |
61
50.4%
|
3
75%
|
64
51.2%
|
| Male |
60
49.6%
|
1
25%
|
61
48.8%
|
Outcome Measures
| Title | Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm |
|---|---|
| Description | AE: any untoward medical occurrence in a participants during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. TEAEs were defined as adverse events with start date or increased severity on and after the first dose of study drug through Week 108. |
| Time Frame | Day 1 up to Week 108 (Study 112) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety Set included all participants who received at least 1 dose of study drug (Ivacaftor). |
| Arm/Group Title | Ivacaftor Arm |
|---|---|
| Arm/Group Description | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet q12h in the previous study VX11-770-110 (Study 110; NCT01614457), VX12-770-111 (Study 111; NCT01614470) or VX12-770-113 (Study 113; NCT01685801); received Ivacaftor 150 mg tablet orally q12h in this VX12-770-112 (Study 112; NCT01707290) up to 104 weeks. |
| Measure Participants | 121 |
| Participants with TEAEs |
117
96.7%
|
| Participants with SAEs |
27
22.3%
|
| Title | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104 |
|---|---|
| Description | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 6 to 17 years and for female participants aged 6 to 15 years. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113. |
| Time Frame | Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Full Analysis Set (FAS) included all participants who received at least 1 dose of study drug (Ivacaftor). Here, "Number Analyzed" signifies those participants who were evaluable for this measure at the specified time point for each arm, respectively. |
| Arm/Group Title | Ivacaftor Arm: Study 110 | Ivacaftor Arm: Study 111 | Ivacaftor Arm: Study 113 |
|---|---|---|---|
| Arm/Group Description | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 110; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 111; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 113; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. |
| Measure Participants | 65 | 35 | 21 |
| Baseline |
71.8
(20.4)
|
78.3
(21.1)
|
62.8
(22.2)
|
| Change at Week 2 |
3.8
(8)
|
4.4
(7.7)
|
4
(7.2)
|
| Change at Week 12 |
5.4
(8.9)
|
5.5
(8.3)
|
4.9
(6.7)
|
| Change at Week 24 |
4.5
(8.4)
|
7.1
(8.3)
|
4.6
(8.5)
|
| Change at Week 36 |
4.4
(7.1)
|
7.5
(8.2)
|
4.9
(8.2)
|
| Change at Week 48 |
4.4
(7.5)
|
6.4
(10.7)
|
4.9
(9)
|
| Change at Week 60 |
4.5
(8.4)
|
8.2
(8.3)
|
4.5
(8.8)
|
| Change at Week 72 |
3.6
(8.9)
|
7.8
(9.2)
|
5.9
(9.2)
|
| Change at Week 84 |
5.4
(13.5)
|
7.7
(7.6)
|
4.8
(7.2)
|
| Change at Week 96 |
4.1
(14.6)
|
9.9
(6.4)
|
5.9
(8.1)
|
| Change at Week 104 |
4.6
(13.2)
|
4.9
(5.8)
|
6.3
(10)
|
| Title | Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104 |
|---|---|
| Description | BMI was defined as weight in kg divided by height in m^2. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113. |
| Time Frame | Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112) |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS included all participants who received at least 1 dose of study drug (Ivacaftor). Here, "Number analyzed" signifies those participants who were evaluable for this measure at the specified time point for each arm, respectively. |
| Arm/Group Title | Ivacaftor Arm: Study 110 | Ivacaftor Arm: Study 111 | Ivacaftor Arm: Study 113 |
|---|---|---|---|
| Arm/Group Description | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 110; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 111; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 113; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. |
| Measure Participants | 65 | 35 | 21 |
| Baseline |
23.75
(5.63)
|
23.15
(5.28)
|
24.81
(5.76)
|
| Change at Week 2 |
0.06
(0.43)
|
0.16
(0.47)
|
0.07
(0.43)
|
| Change at Week 12 |
0.32
(0.59)
|
0.39
(0.62)
|
0.22
(0.6)
|
| Change at Week 24 |
0.43
(0.92)
|
0.65
(0.96)
|
0.17
(0.91)
|
| Change at Week 36 |
0.62
(1.04)
|
0.64
(1.07)
|
0.02
(1.27)
|
| Change at Week 48 |
0.54
(1.39)
|
0.58
(1.22)
|
0.28
(1.26)
|
| Change at Week 60 |
0.72
(1.32)
|
0.25
(1.09)
|
0.38
(1.22)
|
| Change at Week 72 |
1.05
(1.73)
|
0.45
(1.34)
|
0.22
(1.22)
|
| Change at Week 84 |
0.72
(1.51)
|
0.33
(1.42)
|
0.23
(0.92)
|
| Change at Week 96 |
0.49
(1.44)
|
0.1
(1.49)
|
0.52
(1.28)
|
| Change at Week 104 |
0.42
(1.49)
|
0.16
(1.37)
|
0.88
(1.39)
|
| Title | Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104 |
|---|---|
| Description | Sweat samples were collected using an approved collection device. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113. |
| Time Frame | Baseline, Week 2, 24, 48 and 104 (Study 112) |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS included all participants who received at least 1 dose of study drug (Ivacaftor). Here, "Number of participants analyzed" signifies those participants who were evaluable for this outcome and "Number Analyzed" signifies those participants who were evaluable for this measure at the specified time point for each arm respectively. |
| Arm/Group Title | Ivacaftor Arm: Study 110 | Ivacaftor Arm: Study 111 | Ivacaftor Arm: Study 113 |
|---|---|---|---|
| Arm/Group Description | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 110; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 111; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 113; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. |
| Measure Participants | 59 | 33 | 21 |
| Baseline |
60.9
(19.4)
|
80.2
(22.8)
|
55.7
(22.2)
|
| Change at Week 2 |
-19.3
(10.7)
|
-38.4
(27.5)
|
-4.5
(10.9)
|
| Change at Week 24 |
-13
(18.4)
|
-39.2
(27)
|
1.1
(15.8)
|
| Change at Week 48 |
-13.5
(16.2)
|
-40.6
(26.1)
|
-1.9
(17.8)
|
| Change at Week 104 |
-13.7
(22.9)
|
-32.9
(26.8)
|
5.4
(18.1)
|
| Title | Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 |
|---|---|
| Description | The CFQ-R is a validated participant reported outcome measuring health related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health related quality of life. Baseline was defined as the most recent measurement before intake of the first dose of study drug (ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113. |
| Time Frame | Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112) |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS included all participants who received at least 1 dose of study drug (ivacaftor). Here, "Number Analyzed" signifies those participants who were evaluable for this measure at the specified time point for each arm respectively. |
| Arm/Group Title | Ivacaftor Arm: Study 110 | Ivacaftor Arm: Study 111 | Ivacaftor Arm: Study 113 |
|---|---|---|---|
| Arm/Group Description | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 110; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 111; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 113; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. |
| Measure Participants | 65 | 35 | 21 |
| Baseline |
68.6
(21.6)
|
72.4
(20.3)
|
65.9
(18.8)
|
| Change at Week 2 |
5.5
(17.9)
|
4.5
(17.5)
|
13.2
(16.2)
|
| Change at Week 12 |
11.6
(18)
|
5.7
(18.5)
|
13.5
(20)
|
| Change at Week 24 |
6.7
(19)
|
3.4
(18.7)
|
8.5
(14.9)
|
| Change at Week 36 |
12.1
(19.3)
|
5.7
(13.8)
|
7.9
(20.5)
|
| Change at Week 48 |
9.5
(17.6)
|
4.4
(13.2)
|
11.4
(15.7)
|
| Change at Week 60 |
7.1
(18.6)
|
4.4
(10.9)
|
8.6
(19.3)
|
| Change at Week 72 |
10.4
(22.2)
|
5.6
(11.8)
|
8.6
(19)
|
| Change at Week 84 |
10.5
(19.5)
|
8.8
(14.1)
|
4.9
(14.6)
|
| Change at Week 96 |
8.8
(21.1)
|
0
(9.5)
|
9.4
(14.7)
|
| Change at Week 104 |
9.2
(14)
|
1.9
(8.8)
|
19.9
(16)
|
| Title | Number of Pulmonary Exacerbations Events |
|---|---|
| Description | Pulmonary exacerbation events include those events which require treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. The number of events were reported. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113. |
| Time Frame | Through Week 104 (Study 112) |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS included all participants who received at least 1 dose of study drug (Ivacaftor). |
| Arm/Group Title | Ivacaftor Arm: Study 110 | Ivacaftor Arm: Study 111 | Ivacaftor Arm: Study 113 |
|---|---|---|---|
| Arm/Group Description | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 110; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 111; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet orally, q12h in the previous Study 113; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks. |
| Measure Participants | 65 | 35 | 21 |
| Number [pulmonary exacerbation events] |
47
|
30
|
6
|
| Title | Number of Participants With Serious Adverse Events (SAEs) in Observational Arm |
|---|---|
| Description | SAE was defined as a medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. |
| Time Frame | up to 2 years (Study 112) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Analysis population included all participants who were included in the observational arm. |
| Arm/Group Title | Observational Arm |
|---|---|
| Arm/Group Description | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet, orally, q12h in the previous Study 110 (NCT01614457) or Study 111 (NCT01614470), were observed (did not receive study drug) in this Study 112 (NCT01707290) for up to 2 years. |
| Measure Participants | 4 |
| Number [participants] |
1
0.8%
|
Adverse Events
| Time Frame | Day 1 up to Week 108 for Ivacaftor arm; up to 2 years for Observational arm (Study 112) | |||
|---|---|---|---|---|
| Adverse Event Reporting Description | Non-SAEs were not collected for Observational arm. Only SAEs were planned to be collected for the Observational arm. | |||
| Arm/Group Title | Ivacaftor Arm | Observational Arm | ||
| Arm/Group Description | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet q12h in the previous study VX11-770-110 (Study 110; NCT01614457), VX12-770-111 (Study 111; NCT01614470) or VX12-770-113 (Study 113; NCT01685801); received Ivacaftor 150 mg tablet orally q12h in this VX12-770-112 (Study 112; NCT01707290) up to 104 weeks. | Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet q12h in the previous Study 110 (NCT01614457) or Study 111 (NCT01614470), were observed (did not receive study drug) in this Study 112 (NCT01707290) for up to 2 years. | ||
All Cause Mortality |
||||
| Ivacaftor Arm | Observational Arm | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | / (NaN) | / (NaN) | ||
Serious Adverse Events |
||||
| Ivacaftor Arm | Observational Arm | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 27/121 (22.3%) | 1/4 (25%) | ||
| Cardiac disorders | ||||
| Cardiomyopathy acute | 1/121 (0.8%) | 0/4 (0%) | ||
| Gastrointestinal disorders | ||||
| Crohn's disease | 1/121 (0.8%) | 0/4 (0%) | ||
| Abdominal pain | 1/121 (0.8%) | 0/4 (0%) | ||
| Flatulence | 1/121 (0.8%) | 0/4 (0%) | ||
| Pancreatitis | 1/121 (0.8%) | 0/4 (0%) | ||
| Infections and infestations | ||||
| Infective pulmonary exacerbation of cystic fibrosis | 21/121 (17.4%) | 1/4 (25%) | ||
| Gastroenteritis | 2/121 (1.7%) | 0/4 (0%) | ||
| Pneumonia | 2/121 (1.7%) | 0/4 (0%) | ||
| Pharyngitis bacterial | 1/121 (0.8%) | 0/4 (0%) | ||
| Gastroenteritis viral | 1/121 (0.8%) | 0/4 (0%) | ||
| Sinusitis | 1/121 (0.8%) | 0/4 (0%) | ||
| Investigations | ||||
| Influenza A virus test positive | 1/121 (0.8%) | 0/4 (0%) | ||
| Renal and urinary disorders | ||||
| Renal failure acute | 1/121 (0.8%) | 0/4 (0%) | ||
| Respiratory, thoracic and mediastinal disorders | ||||
| Haemoptysis | 1/121 (0.8%) | 0/4 (0%) | ||
| Respiratory failure | 1/121 (0.8%) | 0/4 (0%) | ||
| Skin and subcutaneous tissue disorders | ||||
| Angioedema | 1/121 (0.8%) | 0/4 (0%) | ||
| Urticaria | 1/121 (0.8%) | 0/4 (0%) | ||
Other (Not Including Serious) Adverse Events |
||||
| Ivacaftor Arm | Observational Arm | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 117/121 (96.7%) | 0/0 (NaN) | ||
| Blood and lymphatic system disorders | ||||
| Iron deficiency anaemia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Ear and labyrinth disorders | ||||
| Ear pain | 3/121 (2.5%) | 0/0 (NaN) | ||
| Middle ear effusion | 1/121 (0.8%) | 0/0 (NaN) | ||
| Tinnitus | 2/121 (1.7%) | 0/0 (NaN) | ||
| Vertigo | 1/121 (0.8%) | 0/0 (NaN) | ||
| Otorrhoea | 1/121 (0.8%) | 0/0 (NaN) | ||
| Endocrine disorders | ||||
| Cushingoid | 1/121 (0.8%) | 0/0 (NaN) | ||
| Eye disorders | ||||
| Blepharospasm | 1/121 (0.8%) | 0/0 (NaN) | ||
| Conjunctivitis | 2/121 (1.7%) | 0/0 (NaN) | ||
| Conjunctivitis allergic | 1/121 (0.8%) | 0/0 (NaN) | ||
| Conjunctival haemorrhage | 1/121 (0.8%) | 0/0 (NaN) | ||
| Photophobia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Eye swelling | 1/121 (0.8%) | 0/0 (NaN) | ||
| Visual impairment | 1/121 (0.8%) | 0/0 (NaN) | ||
| Vision blurred | 1/121 (0.8%) | 0/0 (NaN) | ||
| Vitreous floaters | 1/121 (0.8%) | 0/0 (NaN) | ||
| Gastrointestinal disorders | ||||
| Diarrhoea | 17/121 (14%) | 0/0 (NaN) | ||
| Constipation | 17/121 (14%) | 0/0 (NaN) | ||
| Vomiting | 12/121 (9.9%) | 0/0 (NaN) | ||
| Abdominal pain | 12/121 (9.9%) | 0/0 (NaN) | ||
| Nausea | 9/121 (7.4%) | 0/0 (NaN) | ||
| Gastrooesophageal reflux disease | 7/121 (5.8%) | 0/0 (NaN) | ||
| Abdominal pain upper | 6/121 (5%) | 0/0 (NaN) | ||
| Abdominal distension | 3/121 (2.5%) | 0/0 (NaN) | ||
| Tooth impacted | 3/121 (2.5%) | 0/0 (NaN) | ||
| Abdominal discomfort | 2/121 (1.7%) | 0/0 (NaN) | ||
| Dental caries | 2/121 (1.7%) | 0/0 (NaN) | ||
| Flatulence | 2/121 (1.7%) | 0/0 (NaN) | ||
| Toothache | 2/121 (1.7%) | 0/0 (NaN) | ||
| Steatorrhoea | 2/121 (1.7%) | 0/0 (NaN) | ||
| Abdominal pain lower | 1/121 (0.8%) | 0/0 (NaN) | ||
| Barrett's oesophagus | 1/121 (0.8%) | 0/0 (NaN) | ||
| Colitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Defaecation urgency | 1/121 (0.8%) | 0/0 (NaN) | ||
| Dental discomfort | 1/121 (0.8%) | 0/0 (NaN) | ||
| Dyspepsia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Frequent bowel movements | 1/121 (0.8%) | 0/0 (NaN) | ||
| Palatal disorder | 1/121 (0.8%) | 0/0 (NaN) | ||
| Gastritis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Pancreatitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Proctitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Rectal haemorrhage | 1/121 (0.8%) | 0/0 (NaN) | ||
| Rectal polyp | 1/121 (0.8%) | 0/0 (NaN) | ||
| Stomatitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Tooth loss | 1/121 (0.8%) | 0/0 (NaN) | ||
| Food poisoning | 1/121 (0.8%) | 0/0 (NaN) | ||
| General disorders | ||||
| Pyrexia | 17/121 (14%) | 0/0 (NaN) | ||
| Chest pain | 4/121 (3.3%) | 0/0 (NaN) | ||
| Influenza like illness | 4/121 (3.3%) | 0/0 (NaN) | ||
| Fatigue | 12/121 (9.9%) | 0/0 (NaN) | ||
| Chills | 2/121 (1.7%) | 0/0 (NaN) | ||
| Feeling of body temperature change | 2/121 (1.7%) | 0/0 (NaN) | ||
| Pain | 2/121 (1.7%) | 0/0 (NaN) | ||
| Oedema peripheral | 2/121 (1.7%) | 0/0 (NaN) | ||
| Device leakage | 1/121 (0.8%) | 0/0 (NaN) | ||
| Asthenia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Implant site rash | 1/121 (0.8%) | 0/0 (NaN) | ||
| Local swelling | 1/121 (0.8%) | 0/0 (NaN) | ||
| Vaccination site pain | 1/121 (0.8%) | 0/0 (NaN) | ||
| Non-cardiac chest pain | 1/121 (0.8%) | 0/0 (NaN) | ||
| Hepatobiliary disorders | ||||
| Cholelithiasis | 2/121 (1.7%) | 0/0 (NaN) | ||
| Bile duct stone | 1/121 (0.8%) | 0/0 (NaN) | ||
| Immune system disorders | ||||
| Seasonal allergy | 4/121 (3.3%) | 0/0 (NaN) | ||
| Allergy to arthropod bite | 1/121 (0.8%) | 0/0 (NaN) | ||
| Infections and infestations | ||||
| Infective pulmonary exacerbation of cystic fibrosis | 49/121 (40.5%) | 0/0 (NaN) | ||
| Nasopharyngitis | 21/121 (17.4%) | 0/0 (NaN) | ||
| Sinusitis | 18/121 (14.9%) | 0/0 (NaN) | ||
| Viral upper respiratory tract infection | 17/121 (14%) | 0/0 (NaN) | ||
| Gastroenteritis viral | 9/121 (7.4%) | 0/0 (NaN) | ||
| Upper respiratory tract infection | 15/121 (12.4%) | 0/0 (NaN) | ||
| Influenza | 7/121 (5.8%) | 0/0 (NaN) | ||
| Oral candidiasis | 8/121 (6.6%) | 0/0 (NaN) | ||
| Gastroenteritis | 5/121 (4.1%) | 0/0 (NaN) | ||
| Acute sinusitis | 4/121 (3.3%) | 0/0 (NaN) | ||
| Lower respiratory tract infection | 5/121 (4.1%) | 0/0 (NaN) | ||
| Vulvovaginal mycotic infection | 3/121 (2.5%) | 0/0 (NaN) | ||
| Bacterial disease carrier | 3/121 (2.5%) | 0/0 (NaN) | ||
| Lower respiratory tract infection bacterial | 3/121 (2.5%) | 0/0 (NaN) | ||
| Pharyngitis streptococcal | 3/121 (2.5%) | 0/0 (NaN) | ||
| Rhinitis | 3/121 (2.5%) | 0/0 (NaN) | ||
| Respiratory tract infection viral | 3/121 (2.5%) | 0/0 (NaN) | ||
| Upper respiratory tract infection bacterial | 3/121 (2.5%) | 0/0 (NaN) | ||
| Tonsillitis | 3/121 (2.5%) | 0/0 (NaN) | ||
| Urinary tract infection | 3/121 (2.5%) | 0/0 (NaN) | ||
| Bronchopulmonary aspergillosis allergic | 2/121 (1.7%) | 0/0 (NaN) | ||
| Otitis externa | 2/121 (1.7%) | 0/0 (NaN) | ||
| Laryngitis | 2/121 (1.7%) | 0/0 (NaN) | ||
| Viral infection | 2/121 (1.7%) | 0/0 (NaN) | ||
| Pharyngitis bacterial | 2/121 (1.7%) | 0/0 (NaN) | ||
| Chronic sinusitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Clostridium difficile colitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Croup infectious | 1/121 (0.8%) | 0/0 (NaN) | ||
| Diarrhoea infectious | 1/121 (0.8%) | 0/0 (NaN) | ||
| Folliculitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Ear lobe infection | 1/121 (0.8%) | 0/0 (NaN) | ||
| Haemophilus infection | 1/121 (0.8%) | 0/0 (NaN) | ||
| Furuncle | 1/121 (0.8%) | 0/0 (NaN) | ||
| Herpes zoster | 1/121 (0.8%) | 0/0 (NaN) | ||
| Infected dermal cyst | 1/121 (0.8%) | 0/0 (NaN) | ||
| Lung infection | 1/121 (0.8%) | 0/0 (NaN) | ||
| Laryngitis viral | 1/121 (0.8%) | 0/0 (NaN) | ||
| Molluscum contagiosum | 1/121 (0.8%) | 0/0 (NaN) | ||
| Myringitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Oral herpes | 1/121 (0.8%) | 0/0 (NaN) | ||
| Oesophagitis bacterial | 1/121 (0.8%) | 0/0 (NaN) | ||
| Respiratory tract infection bacterial | 1/121 (0.8%) | 0/0 (NaN) | ||
| Pneumonia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Otitis media | 1/121 (0.8%) | 0/0 (NaN) | ||
| Staphylococcal skin infection | 1/121 (0.8%) | 0/0 (NaN) | ||
| Staphylococcal infection | 1/121 (0.8%) | 0/0 (NaN) | ||
| Tooth infection | 1/121 (0.8%) | 0/0 (NaN) | ||
| Tooth abscess | 1/121 (0.8%) | 0/0 (NaN) | ||
| Vaginitis bacterial | 1/121 (0.8%) | 0/0 (NaN) | ||
| Viral pharyngitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Vulvovaginal candidiasis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Pharyngitis | 2/121 (1.7%) | 0/0 (NaN) | ||
| Nasal abscess | 1/121 (0.8%) | 0/0 (NaN) | ||
| Injury, poisoning and procedural complications | ||||
| Procedural pain | 3/121 (2.5%) | 0/0 (NaN) | ||
| Contusion | 3/121 (2.5%) | 0/0 (NaN) | ||
| Hand fracture | 2/121 (1.7%) | 0/0 (NaN) | ||
| Laceration | 2/121 (1.7%) | 0/0 (NaN) | ||
| Muscle strain | 2/121 (1.7%) | 0/0 (NaN) | ||
| Arthropod sting | 1/121 (0.8%) | 0/0 (NaN) | ||
| Arthropod bite | 1/121 (0.8%) | 0/0 (NaN) | ||
| Epicondylitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Concussion | 1/121 (0.8%) | 0/0 (NaN) | ||
| Humerus fracture | 1/121 (0.8%) | 0/0 (NaN) | ||
| Fall | 1/121 (0.8%) | 0/0 (NaN) | ||
| Ligament rupture | 1/121 (0.8%) | 0/0 (NaN) | ||
| Joint dislocation | 1/121 (0.8%) | 0/0 (NaN) | ||
| Skeletal injury | 1/121 (0.8%) | 0/0 (NaN) | ||
| Snake bite | 1/121 (0.8%) | 0/0 (NaN) | ||
| Ligament sprain | 1/121 (0.8%) | 0/0 (NaN) | ||
| Soft tissue injury | 1/121 (0.8%) | 0/0 (NaN) | ||
| Tooth fracture | 1/121 (0.8%) | 0/0 (NaN) | ||
| Investigations | ||||
| Bacterial test positive | 7/121 (5.8%) | 0/0 (NaN) | ||
| Forced expiratory volume decreased | 6/121 (5%) | 0/0 (NaN) | ||
| Weight decreased | 5/121 (4.1%) | 0/0 (NaN) | ||
| C-reactive protein increased | 4/121 (3.3%) | 0/0 (NaN) | ||
| Haemophilus test positive | 3/121 (2.5%) | 0/0 (NaN) | ||
| Alanine aminotransferase increased | 2/121 (1.7%) | 0/0 (NaN) | ||
| Liver function test abnormal | 3/121 (2.5%) | 0/0 (NaN) | ||
| Atypical mycobacterium test positive | 2/121 (1.7%) | 0/0 (NaN) | ||
| Blood pressure increased | 2/121 (1.7%) | 0/0 (NaN) | ||
| Hepatic enzyme increased | 2/121 (1.7%) | 0/0 (NaN) | ||
| Body temperature increased | 2/121 (1.7%) | 0/0 (NaN) | ||
| Pseudomonas test positive | 2/121 (1.7%) | 0/0 (NaN) | ||
| Vitamin D decreased | 2/121 (1.7%) | 0/0 (NaN) | ||
| Blood bilirubin increased | 1/121 (0.8%) | 0/0 (NaN) | ||
| Weight increased | 2/121 (1.7%) | 0/0 (NaN) | ||
| Blood calcium decreased | 1/121 (0.8%) | 0/0 (NaN) | ||
| Blood creatinine increased | 1/121 (0.8%) | 0/0 (NaN) | ||
| Blood urea increased | 1/121 (0.8%) | 0/0 (NaN) | ||
| Blood glucose increased | 1/121 (0.8%) | 0/0 (NaN) | ||
| Crystal urine present | 1/121 (0.8%) | 0/0 (NaN) | ||
| Electrocardiogram abnormal | 1/121 (0.8%) | 0/0 (NaN) | ||
| Electrocardiogram QT prolonged | 1/121 (0.8%) | 0/0 (NaN) | ||
| Flavobacterium test positive | 1/121 (0.8%) | 0/0 (NaN) | ||
| Influenza A virus test positive | 1/121 (0.8%) | 0/0 (NaN) | ||
| Streptococcus test positive | 1/121 (0.8%) | 0/0 (NaN) | ||
| Klebsiella test positive | 1/121 (0.8%) | 0/0 (NaN) | ||
| Urinary sediment present | 1/121 (0.8%) | 0/0 (NaN) | ||
| Ultrasound biliary tract abnormal | 1/121 (0.8%) | 0/0 (NaN) | ||
| Urine analysis abnormal | 1/121 (0.8%) | 0/0 (NaN) | ||
| Vitamin A decreased | 1/121 (0.8%) | 0/0 (NaN) | ||
| White blood cells urine positive | 1/121 (0.8%) | 0/0 (NaN) | ||
| Pulmonary function test decreased | 2/121 (1.7%) | 0/0 (NaN) | ||
| Metabolism and nutrition disorders | ||||
| Decreased appetite | 2/121 (1.7%) | 0/0 (NaN) | ||
| Vitamin D deficiency | 4/121 (3.3%) | 0/0 (NaN) | ||
| Diabetes mellitus | 1/121 (0.8%) | 0/0 (NaN) | ||
| Dehydration | 1/121 (0.8%) | 0/0 (NaN) | ||
| Dyslipidaemia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Fluid retention | 1/121 (0.8%) | 0/0 (NaN) | ||
| Impaired fasting glucose | 1/121 (0.8%) | 0/0 (NaN) | ||
| Increased appetite | 1/121 (0.8%) | 0/0 (NaN) | ||
| Hypokalaemia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Vitamin A deficiency | 1/121 (0.8%) | 0/0 (NaN) | ||
| Type 2 diabetes mellitus | 1/121 (0.8%) | 0/0 (NaN) | ||
| Musculoskeletal and connective tissue disorders | ||||
| Back pain | 6/121 (5%) | 0/0 (NaN) | ||
| Arthralgia | 9/121 (7.4%) | 0/0 (NaN) | ||
| Musculoskeletal pain | 5/121 (4.1%) | 0/0 (NaN) | ||
| Muscle spasms | 4/121 (3.3%) | 0/0 (NaN) | ||
| Osteopenia | 4/121 (3.3%) | 0/0 (NaN) | ||
| Pain in extremity | 4/121 (3.3%) | 0/0 (NaN) | ||
| Myalgia | 3/121 (2.5%) | 0/0 (NaN) | ||
| Arthritis | 2/121 (1.7%) | 0/0 (NaN) | ||
| Intervertebral disc protrusion | 2/121 (1.7%) | 0/0 (NaN) | ||
| Bone pain | 1/121 (0.8%) | 0/0 (NaN) | ||
| Clubbing | 1/121 (0.8%) | 0/0 (NaN) | ||
| Joint swelling | 1/121 (0.8%) | 0/0 (NaN) | ||
| Monarthritis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Metatarsalgia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Neck pain | 1/121 (0.8%) | 0/0 (NaN) | ||
| Musculoskeletal chest pain | 1/121 (0.8%) | 0/0 (NaN) | ||
| Pain in jaw | 1/121 (0.8%) | 0/0 (NaN) | ||
| Tendonitis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Rotator cuff syndrome | 1/121 (0.8%) | 0/0 (NaN) | ||
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ||||
| Skin papilloma | 1/121 (0.8%) | 0/0 (NaN) | ||
| Seborrhoeic keratosis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Thyroid neoplasm | 1/121 (0.8%) | 0/0 (NaN) | ||
| Nervous system disorders | ||||
| Sinus headache | 9/121 (7.4%) | 0/0 (NaN) | ||
| Headache | 23/121 (19%) | 0/0 (NaN) | ||
| Migraine | 3/121 (2.5%) | 0/0 (NaN) | ||
| Dizziness | 7/121 (5.8%) | 0/0 (NaN) | ||
| Burning sensation | 1/121 (0.8%) | 0/0 (NaN) | ||
| Carpal tunnel syndrome | 1/121 (0.8%) | 0/0 (NaN) | ||
| Hypoaesthesia | 2/121 (1.7%) | 0/0 (NaN) | ||
| Hypersomnia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Hyperaesthesia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Psychiatric disorders | ||||
| Insomnia | 4/121 (3.3%) | 0/0 (NaN) | ||
| Anxiety | 6/121 (5%) | 0/0 (NaN) | ||
| Abnormal dreams | 1/121 (0.8%) | 0/0 (NaN) | ||
| Depression | 3/121 (2.5%) | 0/0 (NaN) | ||
| Panic attack | 1/121 (0.8%) | 0/0 (NaN) | ||
| Tobacco abuse | 1/121 (0.8%) | 0/0 (NaN) | ||
| Drug dependence | 1/121 (0.8%) | 0/0 (NaN) | ||
| Renal and urinary disorders | ||||
| Nephrolithiasis | 2/121 (1.7%) | 0/0 (NaN) | ||
| Dysuria | 1/121 (0.8%) | 0/0 (NaN) | ||
| Reproductive system and breast disorders | ||||
| Amenorrhoea | 1/121 (0.8%) | 0/0 (NaN) | ||
| Dysmenorrhoea | 1/121 (0.8%) | 0/0 (NaN) | ||
| Breast pain | 1/121 (0.8%) | 0/0 (NaN) | ||
| Testicular torsion | 1/121 (0.8%) | 0/0 (NaN) | ||
| Menorrhagia | 1/121 (0.8%) | 0/0 (NaN) | ||
| Vulvovaginal discomfort | 1/121 (0.8%) | 0/0 (NaN) | ||
| Vulvovaginal pruritus | 1/121 (0.8%) | 0/0 (NaN) | ||
| Respiratory, thoracic and mediastinal disorders | ||||
| Cough | 41/121 (33.9%) | 0/0 (NaN) | ||
| Sinus congestion | 22/121 (18.2%) | 0/0 (NaN) | ||
| Sputum increased | 22/121 (18.2%) | 0/0 (NaN) | ||
| Oropharyngeal pain | 18/121 (14.9%) | 0/0 (NaN) | ||
| Nasal congestion | 17/121 (14%) | 0/0 (NaN) | ||
| Dyspnoea | 12/121 (9.9%) | 0/0 (NaN) | ||
| Haemoptysis | 12/121 (9.9%) | 0/0 (NaN) | ||
| Respiratory tract congestion | 9/121 (7.4%) | 0/0 (NaN) | ||
| Wheezing | 9/121 (7.4%) | 0/0 (NaN) | ||
| Rales | 7/121 (5.8%) | 0/0 (NaN) | ||
| Paranasal sinus hypersecretion | 5/121 (4.1%) | 0/0 (NaN) | ||
| Rhinorrhoea | 6/121 (5%) | 0/0 (NaN) | ||
| Pleuritic pain | 5/121 (4.1%) | 0/0 (NaN) | ||
| Respiration abnormal | 4/121 (3.3%) | 0/0 (NaN) | ||
| Nasal polyps | 3/121 (2.5%) | 0/0 (NaN) | ||
| Throat irritation | 3/121 (2.5%) | 0/0 (NaN) | ||
| Dysphonia | 2/121 (1.7%) | 0/0 (NaN) | ||
| Productive cough | 2/121 (1.7%) | 0/0 (NaN) | ||
| Nasal mucosal disorder | 2/121 (1.7%) | 0/0 (NaN) | ||
| Upper-airway cough syndrome | 2/121 (1.7%) | 0/0 (NaN) | ||
| Asthma | 1/121 (0.8%) | 0/0 (NaN) | ||
| Dyspnoea exertional | 1/121 (0.8%) | 0/0 (NaN) | ||
| Epistaxis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Increased viscosity of bronchial secretion | 1/121 (0.8%) | 0/0 (NaN) | ||
| Lung hyperinflation | 1/121 (0.8%) | 0/0 (NaN) | ||
| Nasal oedema | 1/121 (0.8%) | 0/0 (NaN) | ||
| Nasal inflammation | 1/121 (0.8%) | 0/0 (NaN) | ||
| Pleurisy | 1/121 (0.8%) | 0/0 (NaN) | ||
| Painful respiration | 1/121 (0.8%) | 0/0 (NaN) | ||
| Prolonged expiration | 1/121 (0.8%) | 0/0 (NaN) | ||
| Respiratory tract irritation | 1/121 (0.8%) | 0/0 (NaN) | ||
| Rhinitis allergic | 1/121 (0.8%) | 0/0 (NaN) | ||
| Sinus polyp | 1/121 (0.8%) | 0/0 (NaN) | ||
| Throat tightness | 1/121 (0.8%) | 0/0 (NaN) | ||
| Sneezing | 1/121 (0.8%) | 0/0 (NaN) | ||
| Sputum discoloured | 1/121 (0.8%) | 0/0 (NaN) | ||
| Skin and subcutaneous tissue disorders | ||||
| Eczema | 3/121 (2.5%) | 0/0 (NaN) | ||
| Rash | 6/121 (5%) | 0/0 (NaN) | ||
| Dermatitis contact | 2/121 (1.7%) | 0/0 (NaN) | ||
| Rash erythematous | 3/121 (2.5%) | 0/0 (NaN) | ||
| Eczema nummular | 1/121 (0.8%) | 0/0 (NaN) | ||
| Hyperhidrosis | 2/121 (1.7%) | 0/0 (NaN) | ||
| Erythema | 1/121 (0.8%) | 0/0 (NaN) | ||
| Ingrowing nail | 1/121 (0.8%) | 0/0 (NaN) | ||
| Night sweats | 1/121 (0.8%) | 0/0 (NaN) | ||
| Pruritus | 1/121 (0.8%) | 0/0 (NaN) | ||
| Psoriasis | 1/121 (0.8%) | 0/0 (NaN) | ||
| Rash generalised | 1/121 (0.8%) | 0/0 (NaN) | ||
| Rosacea | 1/121 (0.8%) | 0/0 (NaN) | ||
| Skin lesion | 1/121 (0.8%) | 0/0 (NaN) | ||
| Rash pruritic | 1/121 (0.8%) | 0/0 (NaN) | ||
| Subcutaneous nodule | 1/121 (0.8%) | 0/0 (NaN) | ||
| Acne | 3/121 (2.5%) | 0/0 (NaN) | ||
| Vascular disorders | ||||
| Hot flush | 1/121 (0.8%) | 0/0 (NaN) | ||
| Hypertension | 2/121 (1.7%) | 0/0 (NaN) | ||
| Orthostatic hypotension | 1/121 (0.8%) | 0/0 (NaN) | ||
| Pallor | 1/121 (0.8%) | 0/0 (NaN) | ||
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
PI is free to publish results of the study after (1) the first multi-center publication, (2) if the sponsor elects not to publish the results, or (3) 18 months after close of the study, whichever occurs first. Proposed publications are to be submitted to the sponsor for review and comment for a period of at least 45 days (which may be extended under certain circumstances related to protection of intellectual property); the sponsor cannot require changes to the proposed publications.
Results Point of Contact
| Name/Title | Medical Monitor |
|---|---|
| Organization | Vertex Pharmaceuticals Incorporated |
| Phone | 617-341-6777 |
| medicalinfo@vrtx.com |
Responsible Party:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01707290
Other Study ID Numbers:
- VX12-770-112
- 2012-000389-39
First Posted:
Oct 16, 2012
Last Update Posted:
May 12, 2017
Last Verified:
Apr 1, 2017