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

Phase 3

Completed

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.

Recruitment & Eligibility

Inclusion Criteria

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

Exclusion Criteria

Other protocol defined Inclusion/Exclusion criteria may apply

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.

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
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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Telethon Kids Institute
🇦🇺Nedlands, Australia