A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
- Conditions
- Cystic Fibrosis
- Interventions
- Registration Number
- NCT04353817
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 121
- Heterozygous for the F508del mutation (F/MF)
- Forced expiratory volume in 1 second (FEV1) value greater than equal to(≥) 70%
Key
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Solid organ or hematological transplantation
Other protocol defined Inclusion/Exclusion criteria may apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo (matched to IVA) Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks. ELX/TEZ/IVA IVA Participants weighing less than (\<) 30 kilograms (kg) at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) and participants weighing greater than equals to (\>=) 30 kg at screening received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. Placebo Placebo (matched to ELX/TEZ/IVA) Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks. ELX/TEZ/IVA ELX/TEZ/IVA Participants weighing less than (\<) 30 kilograms (kg) at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) and participants weighing greater than equals to (\>=) 30 kg at screening received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks.
- Primary Outcome Measures
Name Time Method Absolute Change in Lung Clearance Index 2.5 (LCI2.5) From Baseline Through Week 24 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.
- Secondary Outcome Measures
Name Time Method Absolute Change in Sweat Chloride (SwCl) From Baseline Through Week 24 Sweat samples were collected using an approved collection device.
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) Day 1 up to Week 28
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
🇨🇦Montréal, Canada
The Hospital for Sick Children
🇨🇦Toronto, Canada
British Columbia Children's Hospital
🇨🇦Vancouver, Canada
Juliane Marie Center, Rigshospitalet
🇩🇰Copenhagen, Denmark
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
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