Trial Review

Did you know?

The ANZCTR now automatically displays published trial results and simplifies the addition of trial documents such as unpublished protocols and statistical analysis plans.

These enhancements will offer a more comprehensive view of trials, regardless of whether their results are positive, negative, or inconclusive.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05444257




Registration number
NCT05444257
Ethics application status
Date submitted
29/06/2022
Date registered
5/07/2022
Date last updated
13/08/2024

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/TEZ/D-IVA 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 - Part A: Participants will receive VX-121/TEZ/D-IVA once daily for 96 weeks. Part B: Participants will receive VX-121/TEZ/D-IVA once daily for an additional 48 weeks.


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 Baseline up to Week 148
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 144
Secondary outcome [2] 0 0
Absolute Change From Baseline in Sweat Chloride (SwCl)
Timepoint [2] 0 0
From Baseline up to Week 144
Secondary outcome [3] 0 0
Number of Pulmonary Exacerbations (PEx)
Timepoint [3] 0 0
From Baseline up to Week 144

Eligibility
Key inclusion criteria
Key

* Part A: 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
* Part B: Completed study drug treatment in Part A; or had study drug interruption(s) in Part A, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of Part A

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 at the time of enrollment in Part A

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
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
1/10/2026
Actual
Sample size
Target
Accrual to date
Final
822
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
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
Recruitment postcode(s) [1] 0 0
- Camperdown
Recruitment postcode(s) [2] 0 0
- Chermside
Recruitment postcode(s) [3] 0 0
- Melbourne
Recruitment postcode(s) [4] 0 0
- Nedlands
Recruitment postcode(s) [5] 0 0
- Parkville
Recruitment postcode(s) [6] 0 0
- South Brisbane
Recruitment postcode(s) [7] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
Arkansas
Country [4] 0 0
United States of America
State/province [4] 0 0
California
Country [5] 0 0
United States of America
State/province [5] 0 0
Colorado
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Georgia
Country [8] 0 0
United States of America
State/province [8] 0 0
Idaho
Country [9] 0 0
United States of America
State/province [9] 0 0
Illinois
Country [10] 0 0
United States of America
State/province [10] 0 0
Indiana
Country [11] 0 0
United States of America
State/province [11] 0 0
Kansas
Country [12] 0 0
United States of America
State/province [12] 0 0
Kentucky
Country [13] 0 0
United States of America
State/province [13] 0 0
Louisiana
Country [14] 0 0
United States of America
State/province [14] 0 0
Maine
Country [15] 0 0
United States of America
State/province [15] 0 0
Massachusetts
Country [16] 0 0
United States of America
State/province [16] 0 0
Michigan
Country [17] 0 0
United States of America
State/province [17] 0 0
Minnesota
Country [18] 0 0
United States of America
State/province [18] 0 0
Missouri
Country [19] 0 0
United States of America
State/province [19] 0 0
Montana
Country [20] 0 0
United States of America
State/province [20] 0 0
Nebraska
Country [21] 0 0
United States of America
State/province [21] 0 0
New Hampshire
Country [22] 0 0
United States of America
State/province [22] 0 0
New Jersey
Country [23] 0 0
United States of America
State/province [23] 0 0
New York
Country [24] 0 0
United States of America
State/province [24] 0 0
North Carolina
Country [25] 0 0
United States of America
State/province [25] 0 0
Ohio
Country [26] 0 0
United States of America
State/province [26] 0 0
Oklahoma
Country [27] 0 0
United States of America
State/province [27] 0 0
Oregon
Country [28] 0 0
United States of America
State/province [28] 0 0
Pennsylvania
Country [29] 0 0
United States of America
State/province [29] 0 0
South Carolina
Country [30] 0 0
United States of America
State/province [30] 0 0
South Dakota
Country [31] 0 0
United States of America
State/province [31] 0 0
Tennessee
Country [32] 0 0
United States of America
State/province [32] 0 0
Texas
Country [33] 0 0
United States of America
State/province [33] 0 0
Utah
Country [34] 0 0
United States of America
State/province [34] 0 0
Vermont
Country [35] 0 0
United States of America
State/province [35] 0 0
Virginia
Country [36] 0 0
United States of America
State/province [36] 0 0
Washington
Country [37] 0 0
United States of America
State/province [37] 0 0
West Virginia
Country [38] 0 0
United States of America
State/province [38] 0 0
Wisconsin
Country [39] 0 0
Austria
State/province [39] 0 0
Graz
Country [40] 0 0
Austria
State/province [40] 0 0
Innsbruck
Country [41] 0 0
Austria
State/province [41] 0 0
Salzburg
Country [42] 0 0
Austria
State/province [42] 0 0
Vienna
Country [43] 0 0
Belgium
State/province [43] 0 0
Brussels
Country [44] 0 0
Belgium
State/province [44] 0 0
Gent
Country [45] 0 0
Belgium
State/province [45] 0 0
Leuven
Country [46] 0 0
Belgium
State/province [46] 0 0
Woluwe-Saint-Lambert
Country [47] 0 0
Canada
State/province [47] 0 0
Calgary
Country [48] 0 0
Canada
State/province [48] 0 0
Edmonton
Country [49] 0 0
Canada
State/province [49] 0 0
London
Country [50] 0 0
Canada
State/province [50] 0 0
Montreal
Country [51] 0 0
Canada
State/province [51] 0 0
Quebec
Country [52] 0 0
Canada
State/province [52] 0 0
Vancouver
Country [53] 0 0
Czechia
State/province [53] 0 0
Brno
Country [54] 0 0
Czechia
State/province [54] 0 0
Praha 5
Country [55] 0 0
Denmark
State/province [55] 0 0
Aarhus
Country [56] 0 0
Denmark
State/province [56] 0 0
Copenhagen
Country [57] 0 0
France
State/province [57] 0 0
Bordeaux cedex
Country [58] 0 0
France
State/province [58] 0 0
Bron Cedex
Country [59] 0 0
France
State/province [59] 0 0
Creteil
Country [60] 0 0
France
State/province [60] 0 0
Lille
Country [61] 0 0
France
State/province [61] 0 0
Montpellier Cedex 5
Country [62] 0 0
France
State/province [62] 0 0
Nice
Country [63] 0 0
France
State/province [63] 0 0
Paris
Country [64] 0 0
France
State/province [64] 0 0
Pierre-Benite
Country [65] 0 0
France
State/province [65] 0 0
Roscoff cedex
Country [66] 0 0
France
State/province [66] 0 0
Rouen
Country [67] 0 0
France
State/province [67] 0 0
Strasbourg
Country [68] 0 0
France
State/province [68] 0 0
Suresnes
Country [69] 0 0
France
State/province [69] 0 0
Tours
Country [70] 0 0
Germany
State/province [70] 0 0
Berlin
Country [71] 0 0
Germany
State/province [71] 0 0
Erlangen
Country [72] 0 0
Germany
State/province [72] 0 0
Essen
Country [73] 0 0
Germany
State/province [73] 0 0
Frankfurt
Country [74] 0 0
Germany
State/province [74] 0 0
Gießen
Country [75] 0 0
Germany
State/province [75] 0 0
Hannover
Country [76] 0 0
Germany
State/province [76] 0 0
Jena
Country [77] 0 0
Germany
State/province [77] 0 0
Mainz
Country [78] 0 0
Germany
State/province [78] 0 0
Muenchen
Country [79] 0 0
Germany
State/province [79] 0 0
Potsdam
Country [80] 0 0
Germany
State/province [80] 0 0
Ulm
Country [81] 0 0
Germany
State/province [81] 0 0
Würzburg
Country [82] 0 0
Greece
State/province [82] 0 0
Thessaloniki
Country [83] 0 0
Hungary
State/province [83] 0 0
Budapest
Country [84] 0 0
Hungary
State/province [84] 0 0
Torokbalint
Country [85] 0 0
Ireland
State/province [85] 0 0
Cork
Country [86] 0 0
Ireland
State/province [86] 0 0
Dublin
Country [87] 0 0
Ireland
State/province [87] 0 0
Limerick
Country [88] 0 0
Israel
State/province [88] 0 0
Jerusalem
Country [89] 0 0
Israel
State/province [89] 0 0
Petach Tikvah
Country [90] 0 0
Israel
State/province [90] 0 0
Tel HaShomer
Country [91] 0 0
Italy
State/province [91] 0 0
Ancona
Country [92] 0 0
Italy
State/province [92] 0 0
Firenze
Country [93] 0 0
Italy
State/province [93] 0 0
Genova
Country [94] 0 0
Italy
State/province [94] 0 0
Milano
Country [95] 0 0
Italy
State/province [95] 0 0
Milan
Country [96] 0 0
Italy
State/province [96] 0 0
Orbassano
Country [97] 0 0
Italy
State/province [97] 0 0
Potenza
Country [98] 0 0
Italy
State/province [98] 0 0
Verona
Country [99] 0 0
Netherlands
State/province [99] 0 0
Amsterdam
Country [100] 0 0
Netherlands
State/province [100] 0 0
Den Haag
Country [101] 0 0
Netherlands
State/province [101] 0 0
Nijmegen
Country [102] 0 0
Netherlands
State/province [102] 0 0
Rotterdam
Country [103] 0 0
Netherlands
State/province [103] 0 0
Utrecht
Country [104] 0 0
New Zealand
State/province [104] 0 0
Christchurch
Country [105] 0 0
New Zealand
State/province [105] 0 0
Grafton
Country [106] 0 0
New Zealand
State/province [106] 0 0
Hamilton
Country [107] 0 0
New Zealand
State/province [107] 0 0
One Tree Hill
Country [108] 0 0
Norway
State/province [108] 0 0
Bergen
Country [109] 0 0
Norway
State/province [109] 0 0
Oslo
Country [110] 0 0
Poland
State/province [110] 0 0
Gdansk
Country [111] 0 0
Poland
State/province [111] 0 0
Lomianki
Country [112] 0 0
Portugal
State/province [112] 0 0
Lisbon
Country [113] 0 0
Portugal
State/province [113] 0 0
Porto
Country [114] 0 0
Spain
State/province [114] 0 0
Barcelona
Country [115] 0 0
Spain
State/province [115] 0 0
Madrid
Country [116] 0 0
Spain
State/province [116] 0 0
Murcia
Country [117] 0 0
Spain
State/province [117] 0 0
Sabadell
Country [118] 0 0
Spain
State/province [118] 0 0
Sevilla
Country [119] 0 0
Spain
State/province [119] 0 0
Valencia
Country [120] 0 0
Sweden
State/province [120] 0 0
Göteborg
Country [121] 0 0
Sweden
State/province [121] 0 0
Lund
Country [122] 0 0
Sweden
State/province [122] 0 0
Stockholm
Country [123] 0 0
Switzerland
State/province [123] 0 0
Zurich
Country [124] 0 0
United Kingdom
State/province [124] 0 0
Birmingham
Country [125] 0 0
United Kingdom
State/province [125] 0 0
Bristol
Country [126] 0 0
United Kingdom
State/province [126] 0 0
Cambridge
Country [127] 0 0
United Kingdom
State/province [127] 0 0
Edinburgh
Country [128] 0 0
United Kingdom
State/province [128] 0 0
Exeter
Country [129] 0 0
United Kingdom
State/province [129] 0 0
Glasgow
Country [130] 0 0
United Kingdom
State/province [130] 0 0
Leeds
Country [131] 0 0
United Kingdom
State/province [131] 0 0
Liverpool
Country [132] 0 0
United Kingdom
State/province [132] 0 0
London
Country [133] 0 0
United Kingdom
State/province [133] 0 0
Manchester
Country [134] 0 0
United Kingdom
State/province [134] 0 0
Newcastle Upon Tyne
Country [135] 0 0
United Kingdom
State/province [135] 0 0
Penarth
Country [136] 0 0
United Kingdom
State/province [136] 0 0
Southampton

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/study/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
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

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