A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes
- Registration Number
- NCT04545515
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
The study evaluates the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in participants with CF who are 6 years of age and older with F/MF genotypes.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 120
- Completed study drug treatment in parent study (VX19-445-116, NCT04353817), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the treatment period in the parent study
Key
- History of study drug intolerance in the parent study
Other protocol defined Inclusion/Exclusion criteria may apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ELX/TEZ/IVA IVA Participants 6 to less than \<12 year of age and weighing \<30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg /IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 96 weeks. Doses were adjusted upward with subsequent changes in weight. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 96 weeks. ELX/TEZ/IVA ELX/TEZ/IVA Participants 6 to less than \<12 year of age and weighing \<30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg /IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 96 weeks. Doses were adjusted upward with subsequent changes in weight. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 96 weeks.
- Primary Outcome Measures
Name Time Method Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) From Baseline up to Week 100
- Secondary Outcome Measures
Name Time Method Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) From Parent Study Baseline to Week 96 Sweat samples were collected using an approved collection device.
Absolute Change From Parent Study Baseline in Lung Clearance Index 2.5 (LCI2.5) From Parent Study Baseline to Week 96 The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
Trial Locations
- Locations (34)
Telethon Kids Institute
🇦🇺Nedlands, Australia
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
The Children's Hospital at Westmead
🇦🇺Westmead, Australia
McGill University Health Centre, Glen Site, Montreal Children's Hospital
🇨🇦Montreal, Canada
British Columbia Children's Hospital
🇨🇦Vancouver, Canada
Groupe Hospitaler Pellegrin, CHU De Bordeaux
🇫🇷Bordeaux cedex, France
CHU Lyon - Hopital Femme Mere-Enfant
🇫🇷Bron Cedex, France
Hopital Necker, Enfants Malades
🇫🇷Paris Cedex 15, France
Centre de Perharidy
🇫🇷Roscoff cedex, France
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
🇩🇪Essen, Germany
Johann Wolfgang Goethe University
🇩🇪Frankfurt, Germany
Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
🇩🇪Gießen, Germany
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
Schneider Children's Medical Center of Israel
🇮🇱Petach Tikvah, Israel
Hospital Virgen de la Arrixaca
🇪🇸Murcia, Spain
Hospital Universitari Vall d Hebron
🇪🇸Barcelona, Spain
Kinderspital Zuerich
🇨🇭Zurich, Switzerland
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
🇬🇧Bristol, United Kingdom
Children's Hospital of Wales
🇬🇧Cardiff, United Kingdom
Alder Hey Children's NHS Foundation Trust
🇬🇧Liverpool, United Kingdom
Royal Hospital for Sick Children
🇬🇧Edinburgh, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
🇬🇧London, United Kingdom
Great Ormond Street Hospital for Sick Children
🇬🇧London, United Kingdom
Southampton General Hospital
🇬🇧Southampton, United Kingdom
Universitair Medisch Centrum Groningen
🇳🇱Groningen, Netherlands
Hadassah University Hospital Mount Scopus
🇮🇱Jerusalem, Israel
Erasmus Medical Center / Sophia Children's Hospital
🇳🇱Rotterdam, Netherlands
Inselspital - Universitaetsspital Bern
🇨🇭Bern, Switzerland
The Hospital for Sick Children
🇨🇦Toronto, Canada
Juliane Marie Center, Rigshospitalet
🇩🇰Copenhagen, Denmark
Hopital Robert Debre
🇫🇷Paris, France
Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
🇩🇪Heidelberg, Germany
Universitaetsklinkum Koeln, CF-Studienzentrum
🇩🇪Koeln, Germany