- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01946412
Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
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
Study Overview
Study Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
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British Columbia
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Vancouver, British Columbia, Canada
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Edinburgh, United Kingdom
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Liverpool, United Kingdom
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London, United Kingdom
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Alabama
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Birmingham, Alabama, United States
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Colorado
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Denver, Colorado, United States
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Georgia
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Atlanta, Georgia, United States
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Indiana
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Indianapolis, Indiana, United States
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Massachusetts
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Boston, Massachusetts, United States
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Michigan
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Grand Rapids, Michigan, United States
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Minnesota
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Minneapolis, Minnesota, United States
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Missouri
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Kansas city, Missouri, United States
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Nebraska
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Omaha, Nebraska, United States
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Utah
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Salt Lake City, Utah, United States
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Virginia
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Richmond, Virginia, United States
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Washington
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Seattle, Washington, United States
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria (Ivacaftor Arm):
- Completed the last study visit of the treatment period of the previous study (NCT01705145)
- 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
- 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):
- Subjects who prematurely discontinued from the previous study
- 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
- 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
- Subjects receiving commercially-available ivacaftor treatment
- 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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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No Intervention: Observational Arm
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Experimental: Ivacaftor
Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the Week 84 Visit. The ivacaftor dose will be:
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Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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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)
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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.
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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)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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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)
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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).
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Baseline (study 108), Week 24, 48, 72 and 84 (study 109)
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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)
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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).
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Baseline (study 109), Week 24, 48, 72 and 84 (study 109)
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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)
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Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145)
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Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
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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)
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Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).
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Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
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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)
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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).
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Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
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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)
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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).
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Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
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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)
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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).
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Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
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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)
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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).
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Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
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Collaborators and Investigators
Collaborators
Investigators
- Study Director: Adebayo Lawal, M.D., Vertex Pharmaceuticals Incorporated
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- VX11-770-109
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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