A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del
- Registration Number
- NCT03625466
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 51
- Subjects with confirmed diagnosis of CF.
- Homozygous for F508del (F/F).
- Subjects who weigh ≥8 kg without shoes and wearing light clothing at the Screening Visit.
Key
- Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.
- Solid organ or hematological transplantation.
- History of any illness or comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might confound the results of the study.
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Part 1: Placebo Placebo Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks. Part 1: LUM/IVA LUM/IVA Participants weighing less than (\<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Participants weighing greater than or equals to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks. Part 2: Overall LUM/IVA LUM/IVA Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \>=6 years of age at week 48) in open-label period for 48 weeks.
- Primary Outcome Measures
Name Time Method Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48 From Baseline at Week 48 MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = \<50% of lobe involved and 2 = \>=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement.
- Secondary Outcome Measures
Name Time Method Part 1: Absolute Change in Lung Clearance Index2.5 (LCI2.5) Through Week 48 From Baseline Through Week 48 LCI2.5 represents the number of lung turnovers required to reduce the end-tidal inert gas concentration to 1/40th of its starting value.
Part 1: Absolute Change in Weight-for-age Z-score at Week 48 From Baseline at Week 48 The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.
Part 1: Absolute Change in Stature-for-age Z-score at Week 48 From Baseline at Week 48 The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.
Part 1: Absolute Change in Body Mass Index (BMI)-For-age Z-score at Week 48 From Baseline at Week 48 BMI was defined as weight in kilogram (kg) divided by squared height in meters (m\^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.
Trial Locations
- Locations (5)
Heidelberg Cystic Fibrosis Center
🇩🇪Heidelberg, Germany
Hannover Medical School
🇩🇪Hannover, Germany
Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin
🇩🇪Lubeck, Germany
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin
🇩🇪Giessen, Germany