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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

October 9, 2012
April 3, 2017
February 2013
April 2016   (Final data collection date for primary outcome measure)
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) ]
AE: any untoward medical occurrence in a participants during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. TEAEs were defined as adverse events with start date or increased severity on and after the first dose of study drug through Week 108.
Safety [ Time Frame: Up to 104 weeks ]
Subjective reporting of adverse events, clinical laboratory values (serum chemistry and hematology), electrocardiograms (ECGs), ophthalmologic examinations, and vital signs.
Complete list of historical versions of study NCT01707290 on ClinicalTrials.gov Archive Site
  • 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) ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 6 to 17 years and for female participants aged 6 to 15 years. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
  • 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) ]
    BMI was defined as weight in kg divided by height in m^2. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
  • 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) ]
    Sweat samples were collected using an approved collection device. Baseline was defined as the most recent measurement before intake of the first dose of study drug (Ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
  • 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) ]
    The CFQ-R is a validated participant reported outcome measuring health related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health related quality of life. Baseline was defined as the most recent measurement before intake of the first dose of study drug (ivacaftor) in Study 112. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
  • Number of Pulmonary Exacerbations Events [ Time Frame: Through Week 104 (Study 112) ]
    Pulmonary exacerbation events include those events which require treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. The number of events were reported. Results were planned to be reported for Ivacaftor arm and were stratified by parent study 110, 111 and 113.
  • Number of Participants With Serious Adverse Events (SAEs) in Observational Arm [ Time Frame: up to 2 years (Study 112) ]
    SAE was defined as a medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event.
  • Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: Through Week 104 ]
  • Change from baseline in body mass index (BMI) [ Time Frame: At Week 104 ]
  • Change from baseline in sweat chloride [ Time Frame: Through Week 104 ]
  • Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ R) [ Time Frame: Through Week 104 ]
  • Pulmonary exacerbation [ Time Frame: Up to 104 weeks ]
Not Provided
Not Provided
 
Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
A Phase 3, Two-Arm, Rollover Study to Evaluate the Safety of Long Term Ivacaftor Treatment in Subjects 6 Years of Age and Older With Cystic Fibrosis and a Non-G551D CFTR Mutation
The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801).

Ivacaftor is the first Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator to show an improvement in CFTR function and clinical benefit in participants with CF. Results from Phase 3 studies (NCT00909532 [Study 102] and NCT00909727 [Study 103]) showed that ivacaftor is effective in the treatment of participants with CF who have the G551D-CFTR mutation, as evidenced by sustained improvements in CFTR channel function (measured by reduction in sweat chloride concentration) and corresponding substantial, durable improvements in lung function, pulmonary exacerbations, respiratory symptoms, and weight gain. Ivacaftor was also well tolerated, as evidenced by the rates and reasons for premature discontinuation and results of safety assessments.

Ivacaftor (Trade Name Kalydeco; 150 mg tablets) was initially approved in the United States for the treatment of CF in participants 6 years of age and older who have a G551D mutation in the CFTR gene.

Interventional
Phase 3
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: No masking
Primary Purpose: Treatment
Cystic Fibrosis
Drug: Ivacaftor
150 mg tablet, oral use, every 12 hours (q12h)
Other Names:
  • Kalydeco
  • VX-770
  • Experimental: Ivacaftor
    Participants who received Ivacaftor 150 milligram (mg) tablet and/or Placebo matched to Ivacaftor tablet orally, 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 q12h in this VX12-770-112 (Study 112; NCT01707290) up to 104 weeks.
    Intervention: Drug: Ivacaftor
  • No Intervention: Observational
    Participants who received Ivacaftor 150 mg tablet and/or Placebo matched to Ivacaftor tablet, orally, 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.
Moss RB, Flume PA, Elborn JS, Cooke J, Rowe SM, McColley SA, Rubenstein RC, Higgins M; VX11-770-110 (KONDUCT) Study Group.. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. Lancet Respir Med. 2015 Jul;3(7):524-33. doi: 10.1016/S2213-2600(15)00201-5. Epub 2015 Jun 9.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
125
April 2016
April 2016   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Participants from Study 110 or Study 111 entering the ivacaftor arm must have completed the assigned study drug treatment duration in the previous study.
  • Participants from Study 113 entering the ivacaftor arm must have completed all study related treatments through the Follow-up Visit and met the Study 113 responder criteria during the previous study.
  • Participants entering the observational arm must have completed at least 4 weeks of study drug treatment in their previous study (Study 110 or Study 111), must have completed the previous study but do not wish to enroll in the ivacaftor arm, or must have completed the previous study but do not meet the inclusion criteria of the ivacaftor arm.
  • Participants of childbearing potential entering the ivacaftor arm must not be pregnant.
  • Participants entering the ivacaftor arm must be willing to comply with contraception requirements.

Exclusion Criteria (Ivacaftor Arm Only):

  • History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the Participant.
  • Use of moderate or strong inhibitors or inducers of cytochrome P450 (CYP) 3A.
  • Evidence of cataract or lens opacity at or before the Day 1 Visit.
Sexes Eligible for Study: All
6 Years and older   (Child, Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
United States,   Belgium,   France,   United Kingdom
Ireland
 
NCT01707290
VX12-770-112
2012-000389-39 ( EudraCT Number )
No
Not Provided
Not Provided
Not Provided
Vertex Pharmaceuticals Incorporated
Vertex Pharmaceuticals Incorporated
Cystic Fibrosis Foundation Therapeutics
Principal Investigator: Joseph Pilewski, MD Children's Hospital of Pittsburgh
Vertex Pharmaceuticals Incorporated
April 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP