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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/NCT05444257




Registration number
NCT05444257
Ethics application status
Date submitted
29/06/2022
Date registered
5/07/2022
Date last updated
22/12/2023

Titles & IDs
Public title
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
Scientific title
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
Secondary ID [1] 0 0
2021-000713-17
Secondary ID [2] 0 0
VX20-121-104
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 - VX-121/TEZ/D-IVA

Experimental: VX-121/TEZ/D-IVA - Participants will receive VX-121/TEZ/D-IVA once daily.


Treatment: Drugs: VX-121/TEZ/D-IVA
Fixed-dose combination 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 Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
From Day 1 up to Week 100
Secondary outcome [1] 0 0
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Timepoint [1] 0 0
From Baseline up to Week 96
Secondary outcome [2] 0 0
Absolute Change From Baseline in Sweat Chloride (SwCl)
Timepoint [2] 0 0
From Baseline up to Week 96
Secondary outcome [3] 0 0
Number of Pulmonary Exacerbations (PEx)
Timepoint [3] 0 0
From Baseline up to Week 96

Eligibility
Key inclusion criteria
Key

- Completed study drug treatment in a parent study VX20-121-102 (NCT05033080) and
VX20-121-103 (NCT05076149); or had study drug interruption(s) in a parent study but
did not permanently discontinue study drug, and 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

- Pregnant or breast-feeding females

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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/11/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2025
Actual
Sample size
Target
Accrual to date
Final
822
Recruitment in Australia
Recruitment state(s)
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Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [3] 0 0
Alfred Hospital - Melbourne
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
Recruitment hospital [7] 0 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [8] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [9] 0 0
Westmead Hospital - Westmead
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- Camperdown
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- Chermside
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- Melbourne
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- Nedlands
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- Parkville
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- South Brisbane
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- Westmead
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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 long-term safety, tolerability, and efficacy of
VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05444257
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
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Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05444257