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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00909727
First Posted: May 28, 2009
Last Update Posted: August 21, 2012
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Cystic Fibrosis Foundation Therapeutics
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
Results First Submitted: February 27, 2012  
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

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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.

Reporting Groups
  Description
Placebo Oral tablet every 12 hours (q12h) for up to 48 weeks
150 mg Ivacaftor q12h Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks

Participant Flow:   Overall Study
    Placebo   150 mg Ivacaftor q12h
STARTED   26 [1]   26 [2] 
Completed Treatment Period, Week 24   23   26 
COMPLETED   22 [3]   26 [3] 
NOT COMPLETED   4   0 
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)



  Baseline Characteristics
  Hide Baseline Characteristics

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.
No text entered.

Reporting Groups
  Description
Placebo Oral tablet every 12 hours (q12h) for up to 48 weeks
150 mg Ivacaftor q12h Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks
Total Total of all reporting groups

Baseline Measures
   Placebo   150 mg Ivacaftor q12h   Total 
Overall Participants Analyzed 
[Units: Participants]
 26   26   52 
Age 
[Units: Years]
Mean (Standard Deviation)
 8.9  (1.86)   8.9  (2.00)   8.9  (1.91) 
Age, Customized 
[Units: Participants]
     
6 to 8 Years   13   12   25 
9 to 11 Years   12   11   23 
> 11 Years   1   3   4 
Gender 
[Units: Participants]
     
Female   10   17   27 
Male   16   9   25 
Race/Ethnicity, Customized 
[Units: Participants]
     
White   23   22   45 
Other   1   2   3 
Not Allowed to Ask Per Local Regulations   2   2   4 
Race/Ethnicity, Customized 
[Units: 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 
[Units: Participants]
     
North America   15   12   27 
Europe   5   6   11 
Australia   6   8   14 
Weight 
[Units: Kilograms]
Mean (Standard Deviation)
 30.0  (7.16)   31.8  (9.95)   30.9  (8.63) 
Body Mass Index 
[Units: Kilograms per square meter]
Mean (Standard Deviation)
 16.8  (1.75)   17.1  (2.61)   17.0  (2.21) 
Sweat Chloride 
[Units: Millimoles per liter]
Mean (Standard Deviation)
 104.8  (8.87)   104.3  (14.54)   104.6  (11.92) 
Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) [1] 
[Units: Participants]
     
< 70%   8   4   12 
≥ 70% to ≤ 90%   6   12   18 
> 90%   12   10   22 
[1] Percent predicted for age, gender, and height
Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) [1] 
[Units: Percentage]
Mean (Standard Deviation)
 83.7  (20.37)   84.7  (15.83)   84.2  (18.07) 
[1] Percent predicted for age, gender, and height


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24   [ Time Frame: baseline through 24 weeks ]

2.  Secondary:   Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 48   [ Time Frame: baseline through 48 weeks ]

3.  Secondary:   Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Through Week 24 and Week 48 (Respiratory Domain Score, Children)   [ Time Frame: baseline through 24 weeks and 48 weeks ]

4.  Secondary:   Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48   [ Time Frame: baseline through 24 weeks and 48 weeks ]

5.  Secondary:   Absolute Change From Baseline in Weight at Week 24 and Week 48   [ Time Frame: baseline to 24 weeks and 48 weeks ]


  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 Other disclosure agreement that restricts the right of the PI 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
e-mail: medicalinfo@vrtx.com


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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