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

This study has been completed.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT00909532
First received: May 26, 2009
Last updated: January 14, 2013
Last verified: January 2013
Results First Received: February 27, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, 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
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
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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.

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     78 [1]   83 [2]
Completed Treatment Period, Week 24     71     80  
COMPLETED     68 [3]   77 [3]
NOT COMPLETED     10     6  
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)



  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  
Number of Participants  
[units: participants]
  78     83     161  
Age  
[units: participants]
     
<=18 years     17     19     36  
Between 18 and 65 years     61     64     125  
>=65 years     0     0     0  
Age  
[units: years]
Mean ± Standard Deviation
  24.7  ± 9.21     26.2  ± 9.85     25.5  ± 9.54  
Gender  
[units: participants]
     
Female     40     44     84  
Male     38     39     77  
Ethnicity (NIH/OMB)  
[units: participants]
     
Hispanic or Latino     0     0     0  
Not Hispanic or Latino     77     81     158  
Unknown or Not Reported     1     2     3  
Race/Ethnicity, Customized  
[units: participants]
     
White     77     81     158  
Not Allowed to Ask Per Local Regulations     1     2     3  
Region of Enrollment  
[units: 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]
[units: percentage]
Mean ± Standard Deviation
  63.7  ± 16.83     63.5  ± 16.14     63.6  ± 16.43  
Percent Predicted FEV1, Categorical [1]
[units: participants]
     
< 70% predicted FEV1     45     49     94  
≥ 70% predicted FEV1     33     34     67  
Weight  
[units: kilograms]
Mean ± Standard Deviation
  61.2  ± 13.93     61.7  ± 14.26     61.5  ± 14.06  
Body Mass Index  
[units: kilograms per square meter]
Mean ± Standard Deviation
  21.9  ± 3.49     21.7  ± 3.65     21.8  ± 3.56  
Sweat Chloride  
[units: millimoles per liter]
Mean ± Standard Deviation
  100.1  ± 10.63     100.4  ± 10.00     100.2  ± 10.28  
[1] Percent predicted for age, gender, and height.



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Absolute Mean 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 Mean Change From Baseline in Percent Predicted FEV1 Through Week 48   [ Time Frame: baseline through 48 weeks ]

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

6.  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


  More Information
  Hide More Information

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.


Limitations and Caveats
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
No text entered.  


Results Point of Contact:  
Name/Title: Medical Monitor
Organization: Vertex
phone: 617-444-6777
e-mail: medicalinfo@vrtx.com


Publications:

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT00909532     History of Changes
Other Study ID Numbers: VX08-770-102
Study First Received: May 26, 2009
Results First Received: February 27, 2012
Last Updated: January 14, 2013
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Ireland: Irish Medicines Board
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medical Devices
Czech Republic: State Institute for Drug Control
Australia: Department of Health and Ageing Therapeutic Goods Administration
Canada: Health Canada