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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
121
Fixed-dose combination tablet for oral administration qd in the morning.
Tablet for oral administration qd in the evening.
Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning.
Absolute Change in Lung Clearance Index 2.5 (LCI2.5)
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.
Time frame: From Baseline Through Week 24
Absolute Change in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
Time frame: From Baseline Through Week 24
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 28
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Placebo matched to IVA for oral administration qd in the evening.
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
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, France
CHU Lyon - Hopital Femme Mere-Enfant
Bron, France
Hopital Necker, Enfants Malades
Paris, France
...and 24 more locations