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Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

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ClinicalTrials.gov Identifier: NCT01946412
Recruitment Status : Completed
First Posted : September 19, 2013
Results First Posted : February 1, 2017
Last Update Posted : February 1, 2017
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE September 16, 2013
First Posted Date  ICMJE September 19, 2013
Results First Submitted Date  ICMJE December 6, 2016
Results First Posted Date  ICMJE February 1, 2017
Last Update Posted Date February 1, 2017
Study Start Date  ICMJE December 2013
Actual Primary Completion Date December 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 6, 2016)
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Day 1 up to Week 97 (for participants who completed study drug dosing); Day 1 up to 24 weeks after the last dose (up to Week 108, for participants who prematurely discontinued study drug dosing) ]
AE: any untoward medical occurrence in a participant 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 the 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, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation was considered treatment-emergent.
Original Primary Outcome Measures  ICMJE
 (submitted: September 16, 2013)
Safety, as determined by adverse events, clinical laboratory values (serum chemistry and hematology), standard 12-lead electrocardiograms (ECGs), vital signs, and ophthalmologic examinations [ Time Frame: Baseline through week 88 ]
Change History Complete list of historical versions of study NCT01946412 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: December 6, 2016)
  • Absolute Change From Baseline of Parent Study in Sweat Chloride at Week 24, 48, 72 and 84 [ Time Frame: Baseline (study 108), Week 24, 48, 72 and 84 (study 109) ]
    Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145).
  • Absolute Change From Baseline of Study 109 in Sweat Chloride at Week 24, 48, 72 and 84 [ Time Frame: Baseline (study 109), Week 24, 48, 72 and 84 (study 109) ]
    Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of >=15 microliter was required for determination of sweat chloride. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).
  • Absolute Change From Baseline of Parent Study in Weight at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145)
  • Absolute Change From Baseline of Study 109 in Weight at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).
  • Absolute Change From Baseline of Parent Study in Stature at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145).
  • Absolute Change From Baseline of Study 109 in Stature at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).
  • Absolute Change From Baseline of Parent Study in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145).
  • Absolute Change From Baseline of Study 109 in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84 [ Time Frame: Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) ]
    BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).
Original Secondary Outcome Measures  ICMJE
 (submitted: September 16, 2013)
  • Absolute change from baseline of the previous study in weight [ Time Frame: Through week 88 ]
  • Absolute change from baseline in weight [ Time Frame: Baseline through week 88 ]
  • Absolute change from baseline of the previous study in stature [ Time Frame: Through week 88 ]
  • Absolute change from baseline in stature [ Time Frame: Baseline through week 88 ]
  • Absolute change from baseline of the previous study in body mass index (BMI) [ Time Frame: Through week 88 ]
  • Absolute change from baseline in BMI [ Time Frame: Baseline through week 88 ]
  • Absolute change from baseline of the previous study in sweat chloride [ Time Frame: Through week 88 ]
  • Absolute change from baseline in sweat chloride [ Time Frame: Baseline through week 88 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
Official Title  ICMJE A Phase 3, 2-Arm, Roll-Over Study to Evaluate the Long-term Safety and Pharmacodynamics of Ivacaftor Treatment in Pediatric Subjects With Cystic Fibrosis and a CFTR Gating Mutation
Brief Summary The purpose of this study is to provide information regarding the long-term safety and pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of age with Cystic Fibrosis (CF) who have a CFTR gating mutation in at least 1 allele and will further explore the efficacy of long-term ivacaftor treatment in this population of patients with CF.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE Drug: Ivacaftor
Other Names:
  • VX-770
  • Kalydeco
Study Arms  ICMJE
  • No Intervention: Observational Arm
  • Experimental: Ivacaftor

    Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the Week 84 Visit. The ivacaftor dose will be:

    • 50 mg q12h for subjects 2 to <6 years of age and <14 kg,
    • 75 mg q12h for subjects 2 to <6 years of age and ≥ 14 kg, or
    • 150 mg q12h for subjects ≥ 6 years of age.
    Intervention: Drug: Ivacaftor
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 4, 2013)
33
Original Enrollment  ICMJE Not Provided
Actual Study Completion Date  ICMJE December 2015
Actual Primary Completion Date December 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria (Ivacaftor Arm):

  1. Completed the last study visit of the treatment period of the previous study (NCT01705145)
  2. Hematology, serum chemistry, and vital signs results on Day 1 with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator
  3. As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions and the parent or legal guardian should be able to ensure that the subject assents to participation in the study to the degree the subject can assent, and that the subject will comply with and is likely to complete the study as planned

Inclusion Criteria (Observational Arm):

1. Subjects who completed their assigned study drug treatment in the previous study (NCT01705145) and elected not to enroll in the ivacaftor arm and subjects who prematurely discontinued treatment in the previous study and received at least 1 dose of study drug treatment in the previous study will be eligible for enrollment in the observational arm.

Exclusion Criteria (Ivacaftor Arm):

  1. Subjects who prematurely discontinued from the previous study
  2. History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject
  3. Subjects with a history of study treatment intolerance as observed in their previous study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
  4. Subjects receiving commercially-available ivacaftor treatment
  5. Subject was unable to complete an adequate slit-lamp examination at the last ophthalmologic examination in the previous study

Exclusion Criteria: (Observational Arm)

1. Subjects receiving ivacaftor treatment will not be eligible for enrollment in the observational arm.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01946412
Other Study ID Numbers  ICMJE VX11-770-109
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Vertex Pharmaceuticals Incorporated
Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Collaborators  ICMJE Cystic Fibrosis Foundation
Investigators  ICMJE
Study Director: Adebayo Lawal, M.D. Vertex Pharmaceuticals Incorporated
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date December 2016

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