A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)

Phase 3

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: ELX/TEZ/IVA; Drug: IVA

• Registration Number: NCT04969224
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

This study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-07-09
• Study First Submitted QC: 2021-07-09
• Study First Posted: 2021-07-20
• Study Start: 2021-10-12
• Primary Completion: 2022-07-26
• Study Completion: 2022-07-26
• Results First Submitted: 2023-07-25
• Status Verified: 2023-09
• Results First Submitted QC: 2023-09-09
• Last Update Submitted: 2023-09-09
• Results First Posted: 2023-10-03
• Last Update Posted: 2023-10-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 82

Inclusion Criteria

• Forced expiratory volume in 1 second (FEV1) value ≥30% and ≤90% predicted
• Heterozygous for CF transmembrane conductance regulator gene (CFTR) F508del mutation and a minimal function mutation (F/MF genotypes)

Key

Exclusion Criteria

• Clinically significant liver cirrhosis
• Solid organ or hematological transplantation
• Non-ambulatory status
• Lung infection with organisms associated with a more rapid decline in pulmonary status

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 received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period approximately 13 weeks.
ELX/TEZ/IVA	ELX/TEZ/IVA	Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period approximately 13 weeks.

Primary Outcome Measures

Name	Time	Method
Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12	Baseline, Week 8 through Week 12	Percent reduction in cough frequency was analyzed with a mixed effects model for repeated measures (MMRM), with change from baseline at each post-baseline visit on the natural log scale as the dependent variable. The percent reduction was estimated as 100% × (1-exponential form of LS mean change estimate from the MMRM).

Secondary Outcome Measures

Name	Time	Method
Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12	Baseline, Week 8 through Week 12

Trial Locations

Locations (19)

Cliniques Universitaires de Bruxelles Hopital Erasme; 🇧🇪 Brussels, Belgium

Universitair Ziekenhuis Brussel - Campus Jette; 🇧🇪 Brussels, Belgium

Universitair Ziekenhuis Gent; 🇧🇪 Gent, Belgium

University of Calgary Medical Clinic of the Foothills Medical Centre; 🇨🇦 Calgary, Canada

University of Alberta Hospital, Edmonton Clinic; 🇨🇦 Edmonton, Canada

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg; 🇧🇪 Leuven, Belgium

Queen Elizabeth II Health Sciences Center; 🇨🇦 Halifax, Canada

The Prince Charles Hospital; 🇦🇺 Chermside, Australia

Alfred Hospital; 🇦🇺 Melbourne, VIC, Australia

Institute for Respiratory Health; 🇦🇺 Nedlands, Australia

Telethon Kids Institute; 🇦🇺 Nedlands, Australia

Royal Adelaide Hospital; 🇦🇺 Adelaide, Australia

Mater Adult Hospital; 🇦🇺 South Brisbane, Australia

Queensland Children's Hospital; 🇦🇺 South Brisbane, Australia

The Royal Children's Hospital; 🇦🇺 Parkville, VIC, Australia

Hospital Universitari Vall d Hebron; 🇪🇸 Barcelona, Spain

Hospital Universitario Ramon y Cajal; 🇪🇸 Madrid, Spain

Hospital Universitario 12 de Octubre; 🇪🇸 Madrid, Spain

Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari; 🇪🇸 Sabadell, Spain