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Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation (KONTINUE)

This study has been completed.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation Therapeutics
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01707290
First received: October 9, 2012
Last updated: April 3, 2017
Last verified: April 2017
Results First Received: April 3, 2017  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Cystic Fibrosis
Intervention: Drug: Ivacaftor

  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
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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).

Reporting Groups
  Description
Ivacaftor Arm 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.
Observational Arm 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.

Participant Flow:   Overall Study
    Ivacaftor Arm   Observational Arm
STARTED   121   4 
COMPLETED   35   3 
NOT COMPLETED   86   1 
Adverse event (AE)                1                0 
Physician Decision                1                0 
Lost to Follow-up                3                0 
Withdrawal of Consent (Not Due to AE)                4                0 
Commercial drug available                60                1 
Other unspecified                17                0 



  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.
No text entered.

Reporting Groups
  Description
Ivacaftor Arm 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.
Observational Arm 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 Total of all reporting groups

Baseline Measures
   Ivacaftor Arm   Observational Arm   Total 
Overall Participants Analyzed 
[Units: Participants]
 121   4   125 
Age, Customized 
[Units: Participants]
     
6 to 11 years   21   1   22 
12 to 17 years   11   1   12 
18 years and over   89   2   91 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      61  50.4%      3  75.0%      64  51.2% 
Male      60  49.6%      1  25.0%      61  48.8% 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor Arm   [ Time Frame: Day 1 up to Week 108 (Study 112) ]

2.  Secondary:   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   [ Time Frame: Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112) ]

3.  Secondary:   Absolute Change From Baseline in Body Mass Index (BMI) at Week 2,12, 24, 36, 48, 60, 72, 84, 96 and 104   [ Time Frame: Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112) ]

4.  Secondary:   Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104   [ Time Frame: Baseline, Week 2, 24, 48 and 104 (Study 112) ]

5.  Secondary:   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   [ Time Frame: Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112) ]

6.  Secondary:   Number of Pulmonary Exacerbations Events   [ Time Frame: Through Week 104 (Study 112) ]

7.  Secondary:   Number of Participants With Serious Adverse Events (SAEs) in Observational Arm   [ Time Frame: up to 2 years (Study 112) ]


  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


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

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01707290     History of Changes
Other Study ID Numbers: VX12-770-112
2012-000389-39 ( EudraCT Number )
Study First Received: October 9, 2012
Results First Received: April 3, 2017
Last Updated: April 3, 2017