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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05111145




Registration number
NCT05111145
Ethics application status
Date submitted
27/10/2021
Date registered
8/11/2021
Date last updated
11/07/2023

Titles & IDs
Public title
A Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Participants With Cystic Fibrosis (CF)
Scientific title
A Phase 3b Open-label Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis
Secondary ID [1] 0 0
2020-004885-21
Secondary ID [2] 0 0
VX20-445-121
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 mg once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for up to 36 weeks.


Treatment: Drugs: ELX/TEZ/IVA
Fixed-dose combination tablets for oral administration.

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

Outcomes
Primary outcome [1] 0 0
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
Day 1 up to Week 36

Eligibility
Key inclusion criteria
Key

- Completed study drug treatment in parent study (VX19-445-117 [NCT04599465] or
VX20-445-126 [NCT04969224]), or had study drug interruption(s) in parent study but
completed study visits up to the last scheduled visit of the treatment period in the
parent study

Key
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- History of drug intolerance in a parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
14/01/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
20/12/2022
Sample size
Target
Accrual to date
Final
86
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [2] 0 0
Alfred Hospital - Melbourne
Recruitment hospital [3] 0 0
Institute for Respiratory Health - Nedlands
Recruitment hospital [4] 0 0
Telethon Kids Institute - Nedlands
Recruitment hospital [5] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [6] 0 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [7] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
- Chermside
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment postcode(s) [3] 0 0
- Nedlands
Recruitment postcode(s) [4] 0 0
- Parkville
Recruitment postcode(s) [5] 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
Czechia
State/province [7] 0 0
Brno
Country [8] 0 0
Czechia
State/province [8] 0 0
Praha 5
Country [9] 0 0
Spain
State/province [9] 0 0
Barcelona
Country [10] 0 0
Spain
State/province [10] 0 0
Madrid
Country [11] 0 0
Spain
State/province [11] 0 0
Murcia
Country [12] 0 0
Spain
State/province [12] 0 0
Sabadell
Country [13] 0 0
Spain
State/province [13] 0 0
Sevilla

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

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate the safety and tolerability of elexacaftor
(ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in participants with CF who are 12 years of age or
older.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05111145
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