Study of the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis and the G551D Mutation

This study has been completed.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01161537
First received: July 9, 2010
Last updated: July 1, 2014
Last verified: July 2014
Results First Received: February 27, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Single Blind (Subject);   Primary Purpose: Treatment
Condition: Cystic Fibrosis
Interventions: Drug: VX-770
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Study was initiated on October 10, 2010 after first eligible subject signed informed consent form and enrolled in study.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
All results were planned to be reported separately for Part A and Part B of the study.

Reporting Groups
  Description
VX-770

Part A: Subjects received placebo tablets matched to VX-770 150 milligram (mg) orally twice daily from Day 1 to 14 (Placebo run-in period), followed by VX-770 150 mg tablets orally twice daily from Day 15 to 42 (VX-770 treatment period), and then placebo tablets matched to VX-770 150 mg orally twice daily from Day 43 to 57 (Placebo washout period) during Part A of the study.

Part B: Subjects received VX-770 150 mg tablets orally twice daily for 48 weeks during Part B of the study. Part B included subjects from Part A and newly enrolled subjects.


Participant Flow for 2 periods

Period 1:   Part A
    VX-770  
STARTED     8  
COMPLETED     8  
NOT COMPLETED     0  

Period 2:   Part B
    VX-770  
STARTED     9 [1]
COMPLETED     7  
NOT COMPLETED     2  
Withdrawal by Subject                 2  
[1] 4 subjects from Part A continued in Part B of the study.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Full Analysis Set (FAS) = all enrolled subjects who received at least 1 dose of study drug (VX-770 or placebo in Part A and VX-770 in Part B).

Reporting Groups
  Description
VX-770

Part A: Subjects received placebo tablets matched to VX-770 150 mg orally twice daily from Day 1 to 14 (Placebo run-in period), followed by VX-770 150 mg tablets orally twice daily from Day 15 to 42 (VX-770 treatment period), and then placebo tablets matched to VX-770 150 mg orally twice daily from Day 43 to 57 (Placebo washout period) during Part A of the study.

Part B: Subjects received VX-770 150 mg tablets orally twice daily for 48 weeks during Part B of the study. Part B included subjects from Part A and newly enrolled subjects.


Baseline Measures
    VX-770  
Number of Participants  
[units: participants]
  13  
Age [1]
[units: years]
Mean ± Standard Deviation
 
Part A (n = 8)     18.9  ± 4.64  
Part B (n = 9)     24.4  ± 10.3  
Gender, Customized [2]
[units: participants]
 
Part A: Female (n = 8)     4  
Part A: Male (n = 8)     4  
Part B: Female (n = 9)     3  
Part B: Male (n = 9)     6  
Race/Ethnicity, Customized [3]
[units: participants]
 
Part A, Race: White (n = 8)     7  
Part A, Race: Black or African American (n = 8)     1  
Part A, Ethnicity: Not Hispanic or Latino (n = 8)     8  
Part B, Race: White (n = 9)     9  
Part B, Ethnicity: Not Hispanic or Latino (n = 9)     9  
Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) [4]
[units: participants]
 
Part A: <70% (n = 8)     2  
Part A: >=70%-<90% (n = 8)     1  
Part A: >=90% (n = 8)     5  
Part B: <70% (n = 9)     6  
Part B: >=70%-<90% (n = 9)     1  
Part B: >=90% (n = 9)     2  
Body Weight [5]
[units: kilogram (kg)]
Mean ± Standard Deviation
 
Part A (n = 8)     66.31  ± 14.393  
Part B (n = 9)     66.53  ± 13.693  
Height [5]
[units: centimeter (cm)]
Mean ± Standard Deviation
 
Part A (n = 8)     167.5  ± 9.66  
Part B (n = 9)     169.9  ± 11.82  
Body Mass Index (BMI) [6]
[units: kilogram per square meter (kg/m^2)]
Mean ± Standard Deviation
 
Part A (n = 8)     23.44  ± 3.570  
Part B (n = 9)     22.96  ± 4.066  
[1] Data was planned to be reported separately for Part A and Part B of the study. Here “n” signifies participants who were evaluable for specified part of the study.
[2] Data was planned to be reported separately for Part A and Part B of the study. Here “n” signifies subjects who were evaluable for specified part of the study. Subjects may be counted in more than one category.
[3] Data was planned to be reported separately for Part A and Part B of the study. Here “n” signifies subjects who were evaluable for specified part of the study. Subject may be counted in more than one category.
[4] FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Number of subjects in each percent predicted FEV1 category (less than [<] 70%, greater than or equal to [>=] 70%-<90%, and >=90%) are reported. Data was planned to be reported separately for Part A and Part B of the study. Here “n” signifies subjects who were evaluable for specified part of the study. Subjects may be reported in more than one category.
[5] Data was planned to be reported separately for Part A and Part B of the study. Here “n” signifies subjects who were evaluable for specified part of the study.
[6] BMI = (Weight [in kg]) divided by (Height [in meters])^2. Data was planned to be reported separately for Part A and Part B of the study. Here “n” signifies subjects who were evaluable for specified part of the study.



  Outcome Measures
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1.  Primary:   Part A: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging (3He-MRI) at Day 43   [ Time Frame: Part A: Baseline (pre-dose Day 15), Day 43 ]

2.  Primary:   Part B: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging (3He-MRI) at Week 48   [ Time Frame: Part B: Baseline (Day -1), Week 48 ]

3.  Secondary:   Part A: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs   [ Time Frame: Part A: Day 1 up to Day 57 ]

4.  Secondary:   Part A: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Day 43   [ Time Frame: Part A: Baseline (pre-dose Day 15), Day 43 ]

5.  Secondary:   Part A: Absolute Change From Baseline in Sweat Chloride at Day 43   [ Time Frame: Part A: Baseline (pre-dose Day 15), Day 43 ]

6.  Secondary:   Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At Day 43   [ Time Frame: Baseline (pre-dose Day 15), Day 43 ]

7.  Secondary:   Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs   [ Time Frame: Part B: Day 1 up to Week 48 ]

8.  Secondary:   Part B: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 48   [ Time Frame: Part B: Baseline (Day -1), Week 48 ]

9.  Secondary:   Part B: Absolute Change From Baseline in Sweat Chloride at Week 48   [ Time Frame: Part B: Baseline (Day -1), Week 48 ]

10.  Secondary:   Part B: Absolute Change From Baseline in in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At Week 48   [ Time Frame: Part B: Baseline (Day -1), Week 48 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Medical Monitor
Organization: Vertex Pharmaceuticals Incorporated
phone: 617-341-6777
e-mail: medicalinfo@vrtx.com


No publications provided


Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01161537     History of Changes
Other Study ID Numbers: VX10-770-107
Study First Received: July 9, 2010
Results First Received: February 27, 2014
Last Updated: July 1, 2014
Health Authority: United States: Food and Drug Administration