Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
- Registration Number
- NCT01946412
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- 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 available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 33
Not provided
(Observational Arm)
- Subjects receiving ivacaftor treatment will not be eligible for enrollment in the observational arm.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Ivacaftor 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.
- Primary Outcome Measures
Name Time Method Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) 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.
- Secondary Outcome Measures
Name Time Method Absolute Change From Baseline of Parent Study in Sweat Chloride at Week 24, 48, 72 and 84 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 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 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 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 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 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 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 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).