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

A Phase 3b Open-label Study Evaluating the Effects of Elexacaftor/Tezacaftor/Ivacaftor on Cough and Physical Activity in Cystic Fibrosis Subjects 12 Years of Age and Older Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

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

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: IVA; Drug: ELX/TEZ/IVA

• Study Type: Interventional
• Enrollment (Actual): 82
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Adelaide, Australia
    • Royal Adelaide Hospital
  • Chermside, Australia
    • The Prince Charles Hospital
  • Melbourne, VIC, Australia
    • Alfred Hospital
  • Nedlands, Australia
    • Institute for Respiratory Health
  • Nedlands, Australia
    • Telethon Kids Institute
  • Parkville, VIC, Australia
    • The Royal Children's Hospital
  • South Brisbane, Australia
    • Queensland Children's Hospital
  • South Brisbane, Australia
    • Mater Adult Hospital
• Belgium
  • Brussels, Belgium
    • Universitair Ziekenhuis Brussel - Campus Jette
  • Brussels, Belgium
    • Cliniques universitaires de Bruxelles Hopital Erasme
  • Ghent, Belgium
    • Universitair Ziekenhuis Gent
  • Leuven, Belgium
    • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
• Canada
  • Calgary, Canada
    • University of Calgary Medical Clinic of the Foothills Medical Centre
  • Edmonton, Canada
    • University of Alberta Hospital, Edmonton Clinic
  • Halifax, Canada
    • Queen Elizabeth II Health Sciences Center
• Spain
  • Barcelona, Spain
    • Hospital Universitari Vall d Hebron
  • Madrid, Spain
    • Hospital Universitario 12 de Octubre
  • Madrid, Spain
    • Hospital Universitario Ramón y Cajal
  • Sabadell, Spain
    • Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Key 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Drug: IVA; Tablet for oral administration.; Other Names: VX-770; ivacaftor; Drug: ELX/TEZ/IVA; Fixed dose combination (FDC) tablet for oral administration.; Other Names: VX-445/VX-661/VX-770; elexacaftor/tezacaftor/ivacaftor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of 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).	Baseline, Week 8 through Week 12

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Vertex Pharmaceuticals Incorporated

Study record dates

Study Major Dates

• Study Start (Actual): October 12, 2021
• Primary Completion (Actual): July 26, 2022
• Study Completion (Actual): July 26, 2022

Study Registration Dates

• First Submitted: July 9, 2021
• First Submitted That Met QC Criteria: July 9, 2021
• First Posted (Actual): July 20, 2021

Study Record Updates

• Last Update Posted (Estimated): September 24, 2025
• Last Update Submitted That Met QC Criteria: September 22, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Respiratory Tract Diseases; Digestive System Diseases; Lung Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cystic Fibrosis; Molecular Mechanisms of Pharmacological Action; Membrane Transport Modulators; Chloride Channel Agonists; elexacaftor, ivacaftor, tezacaftor drug combination; ivacaftor
• Other Study ID Numbers: VX20-445-126; 2021-001628-16 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes