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

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ELX/TEZ/IVA
IVA
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Royal Adelaide Hospital
  • The Prince Charles Hospital
  • Alfred Hospital
  • Institute for Respiratory Health
  • Telethon Kids Institute
  • The Royal Children's Hospital
  • Mater Adult Hospital
  • Queensland Children's Hospital
  • Cliniques Universitaires de Bruxelles Hopital Erasme
  • Universitair Ziekenhuis Brussel - Campus Jette
  • Universitair Ziekenhuis Gent
  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
  • University of Calgary Medical Clinic of the Foothills Medical Centre
  • University of Alberta Hospital, Edmonton Clinic
  • Queen Elizabeth II Health Sciences Center
  • Hospital Universitari Vall d Hebron
  • Hospital Universitario 12 de Octubre
  • Hospital Universitario Ramon y Cajal
  • Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ELX/TEZ/IVA

Arm Description

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.

Outcomes

Primary Outcome Measures

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).

Secondary Outcome Measures

Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12

Full Information

First Posted
July 9, 2021
Last Updated
September 9, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04969224
Brief Title
A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)
Official Title
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)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
October 12, 2021 (Actual)
Primary Completion Date
July 26, 2022 (Actual)
Study Completion Date
July 26, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

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

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
82 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ELX/TEZ/IVA
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
ELX/TEZ/IVA
Other Intervention Name(s)
VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Intervention Description
Fixed dose combination (FDC) tablet for oral administration.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
Tablet for oral administration.
Primary Outcome Measure Information:
Title
Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12
Description
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).
Time Frame
Baseline, Week 8 through Week 12
Secondary Outcome Measure Information:
Title
Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12
Time Frame
Baseline, Week 8 through Week 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Royal Adelaide Hospital
City
Adelaide
Country
Australia
Facility Name
The Prince Charles Hospital
City
Chermside
Country
Australia
Facility Name
Alfred Hospital
City
Melbourne, VIC
Country
Australia
Facility Name
Institute for Respiratory Health
City
Nedlands
Country
Australia
Facility Name
Telethon Kids Institute
City
Nedlands
Country
Australia
Facility Name
The Royal Children's Hospital
City
Parkville, VIC
Country
Australia
Facility Name
Mater Adult Hospital
City
South Brisbane
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
Cliniques Universitaires de Bruxelles Hopital Erasme
City
Brussels
Country
Belgium
Facility Name
Universitair Ziekenhuis Brussel - Campus Jette
City
Brussels
Country
Belgium
Facility Name
Universitair Ziekenhuis Gent
City
Gent
Country
Belgium
Facility Name
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
City
Leuven
Country
Belgium
Facility Name
University of Calgary Medical Clinic of the Foothills Medical Centre
City
Calgary
Country
Canada
Facility Name
University of Alberta Hospital, Edmonton Clinic
City
Edmonton
Country
Canada
Facility Name
Queen Elizabeth II Health Sciences Center
City
Halifax
Country
Canada
Facility Name
Hospital Universitari Vall d Hebron
City
Barcelona
Country
Spain
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
Country
Spain
Facility Name
Hospital Universitario Ramon y Cajal
City
Madrid
Country
Spain
Facility Name
Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
City
Sabadell
Country
Spain

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing 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

Learn more about this trial

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

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