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Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation (KONNECTION)

This study has been completed.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01614470
First received: June 5, 2012
Last updated: October 23, 2014
Last verified: October 2014
Results First Received: October 23, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Cystic Fibrosis
Interventions: Drug: Ivacaftor
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
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Part 1: Ivacaftor First, Then Placebo Ivacaftor 150 milligram (mg) tablet orally twice daily for 8 weeks in treatment period 1 followed by placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
Part 1: Placebo First, Then Ivacaftor Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 1 followed by ivacaftor 150 mg tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
Part 2: Ivacaftor Ivacaftor 150 mg tablet orally twice daily for 16 weeks.

Participant Flow for 4 periods

Period 1:   Part 1: Treatment Period 1 (8 Weeks)
    Part 1: Ivacaftor First, Then Placebo     Part 1: Placebo First, Then Ivacaftor     Part 2: Ivacaftor  
STARTED     20     19     0  
COMPLETED     18     18     0  
NOT COMPLETED     2     1     0  
Lost to Follow-up                 1                 0                 0  
Need to extend washout period                 1                 1                 0  

Period 2:   Part 1: Washout Period (4 to 8 Weeks)
    Part 1: Ivacaftor First, Then Placebo     Part 1: Placebo First, Then Ivacaftor     Part 2: Ivacaftor  
STARTED     18     18     0  
COMPLETED     18     18     0  
NOT COMPLETED     0     0     0  

Period 3:   Part 1: Treatment Period 2 (8 Weeks)
    Part 1: Ivacaftor First, Then Placebo     Part 1: Placebo First, Then Ivacaftor     Part 2: Ivacaftor  
STARTED     18     18     0  
COMPLETED     18     18     0  
NOT COMPLETED     0     0     0  

Period 4:   Part 2: Open-label Period (16 Weeks)
    Part 1: Ivacaftor First, Then Placebo     Part 1: Placebo First, Then Ivacaftor     Part 2: Ivacaftor  
STARTED     0     0     36  
COMPLETED     0     0     36  
NOT COMPLETED     0     0     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.
Full Analysis Set (FAS) was defined as all randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo).

Reporting Groups
  Description
Part 1: Ivacaftor First, Then Placebo Ivacaftor 150 milligram (mg) tablet orally twice daily for 8 weeks in treatment period 1 followed by placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
Part 1: Placebo First, Then Ivacaftor Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 1 followed by ivacaftor 150 mg tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
Total Total of all reporting groups

Baseline Measures
    Part 1: Ivacaftor First, Then Placebo     Part 1: Placebo First, Then Ivacaftor     Total  
Number of Participants  
[units: participants]
  20     19     39  
Age  
[units: years]
Mean ± Standard Deviation
  23.8  ± 13.25     21.7  ± 12.92     22.8  ± 12.96  
Gender  
[units: participants]
     
Female     7     10     17  
Male     13     9     22  



  Outcome Measures
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1.  Primary:   Part 1: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8   [ Time Frame: Part 1: Baseline (pre-dose Day 1), Week 8 ]

2.  Primary:   Part 2: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through 24 Weeks of Treatment (Week 36 Visit)   [ Time Frame: Baseline (pre-dose Week 12), Week 36 ]

3.  Secondary:   Part 1: Change From Baseline in Body Mass Index (BMI) at Week 8   [ Time Frame: Part 1: Baseline (pre-dose Day 1), Week 8 ]

4.  Secondary:   Part 2: Change From Baseline in Body Mass Index (BMI) at 24 Weeks of Treatment (Week 36 Visit)   [ Time Frame: Baseline (pre-dose Week 12), Week 36 ]

5.  Secondary:   Part 1: Change From Baseline in Sweat Chloride Through Week 8   [ Time Frame: Part 1: Baseline (pre-dose Day 1), Week 8 ]

6.  Secondary:   Part 2: Change From Baseline in Sweat Chloride Through 24 Weeks of Treatment (Week 36 Visit)   [ Time Frame: Baseline (pre-dose Week 12), Week 36 ]

7.  Secondary:   Part 1: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8   [ Time Frame: Part 1: Baseline (pre-dose Day 1), Week 8 ]

8.  Secondary:   Part 2: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through 24 Weeks of Treatment (Week 36 Visit)   [ Time Frame: Baseline (pre-dose Week 12), Week 36 ]

9.  Secondary:   Part 1: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)   [ Time Frame: Part 1: From signing of informed consent up to Week 20 ]

10.  Secondary:   Part 2: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)   [ Time Frame: Part 2: Week 20 up to Week 40 ]


  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: NCT01614470     History of Changes
Other Study ID Numbers: VX12-770-111
Study First Received: June 5, 2012
Results First Received: October 23, 2014
Last Updated: October 23, 2014
Health Authority: United States: Food and Drug Administration
Belgium: Federal Agency for Medicinal Products and Health Products
France: Agence Nationale de Sécurité du Médicament et des produits de santé