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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04969224




Registration number
NCT04969224
Ethics application status
Date submitted
9/07/2021
Date registered
20/07/2021

Titles & IDs
Public title
A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)
Scientific 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)
Secondary ID [1] 0 0
2021-001628-16
Secondary ID [2] 0 0
VX20-445-126
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ELX/TEZ/IVA
Treatment: Drugs - IVA

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.


Treatment: Drugs: ELX/TEZ/IVA
Fixed dose combination (FDC) tablet for oral administration.

Treatment: Drugs: IVA
Tablet for oral administration.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12
Timepoint [1] 0 0
Baseline, Week 8 through Week 12
Secondary outcome [1] 0 0
Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12
Timepoint [1] 0 0
Baseline, Week 8 through Week 12

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/10/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/07/2022
Sample size
Target
Accrual to date
Final
82
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [3] 0 0
Alfred Hospital - Melbourne, VIC
Recruitment hospital [4] 0 0
Institute for Respiratory Health - Nedlands
Recruitment hospital [5] 0 0
Telethon Kids Institute - Nedlands
Recruitment hospital [6] 0 0
The Royal Children's Hospital - Parkville, VIC
Recruitment hospital [7] 0 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [8] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Chermside
Recruitment postcode(s) [3] 0 0
- Melbourne, VIC
Recruitment postcode(s) [4] 0 0
- Nedlands
Recruitment postcode(s) [5] 0 0
- Parkville, VIC
Recruitment postcode(s) [6] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Brussels
Country [2] 0 0
Belgium
State/province [2] 0 0
Gent
Country [3] 0 0
Belgium
State/province [3] 0 0
Leuven
Country [4] 0 0
Canada
State/province [4] 0 0
Calgary
Country [5] 0 0
Canada
State/province [5] 0 0
Edmonton
Country [6] 0 0
Canada
State/province [6] 0 0
Halifax
Country [7] 0 0
Spain
State/province [7] 0 0
Barcelona
Country [8] 0 0
Spain
State/province [8] 0 0
Madrid
Country [9] 0 0
Spain
State/province [9] 0 0
Sabadell

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Vertex Pharmaceuticals Incorporated
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
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


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04969224