NCT04969224

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
82

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Oct 2021

Shorter than P25 for phase_3

Geographic Reach
4 countries

19 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 9, 2021

Completed
11 days until next milestone

First Posted

Study publicly available on registry

July 20, 2021

Completed
3 months until next milestone

Study Start

First participant enrolled

October 12, 2021

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 26, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 26, 2022

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

October 3, 2023

Completed
Last Updated

September 24, 2025

Status Verified

September 1, 2025

Enrollment Period

10 months

First QC Date

July 9, 2021

Results QC Date

July 25, 2023

Last Update Submit

September 22, 2025

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (1)

  • 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

Study Arms (1)

ELX/TEZ/IVA

EXPERIMENTAL

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: ELX/TEZ/IVADrug: IVA

Interventions

ELX/TEZ/IVADRUG

Fixed dose combination (FDC) tablet for oral administration.

Also known as: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
ELX/TEZ/IVA
IVADRUG

Tablet for oral administration.

Also known as: VX-770, ivacaftor
ELX/TEZ/IVA

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (19)

Royal Adelaide Hospital

Adelaide, Australia

Location

The Prince Charles Hospital

Chermside, Australia

Location

Alfred Hospital

Melbourne, VIC, Australia

Location

Institute for Respiratory Health

Nedlands, Australia

Location

Telethon Kids Institute

Nedlands, Australia

Location

The Royal Children's Hospital

Parkville, VIC, Australia

Location

Mater Adult Hospital

South Brisbane, Australia

Location

Queensland Children's Hospital

South Brisbane, Australia

Location

Cliniques Universitaires de Bruxelles Hopital Erasme

Brussels, Belgium

Location

Universitair Ziekenhuis Brussel - Campus Jette

Brussels, Belgium

Location

Universitair Ziekenhuis Gent

Ghent, Belgium

Location

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

Leuven, Belgium

Location

University of Calgary Medical Clinic of the Foothills Medical Centre

Calgary, Canada

Location

University of Alberta Hospital, Edmonton Clinic

Edmonton, Canada

Location

Queen Elizabeth II Health Sciences Center

Halifax, Canada

Location

Hospital Universitari Vall d Hebron

Barcelona, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, Spain

Location

Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari

Sabadell, Spain

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

elexacaftor, ivacaftor, tezacaftor drug combinationivacaftor

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Results Point of Contact

Title
Medical Monitor
Organization
Vertex Pharmaceuticals Incorporated
medicalinfo@vrtx.com
617-341-6777

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 9, 2021

First Posted

July 20, 2021

Study Start

October 12, 2021

Primary Completion

July 26, 2022

Study Completion

July 26, 2022

Last Updated

September 24, 2025

Results First Posted

October 3, 2023

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will not share

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

Locations

CA(3)BE(4)ES(4)AU(8)