Trial Review

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/ct2/show/NCT04043806




Registration number
NCT04043806
Ethics application status
Date submitted
31/07/2019
Date registered
2/08/2019
Date last updated
6/07/2023

Titles & IDs
Public title
A Study Evaluating the Long-term Safety of VX-445 Combination Therapy
Scientific title
A Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis
Secondary ID [1] 0 0
2018-004652-38
Secondary ID [2] 0 0
VX18-445-113
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(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - ELX/TEZ/IVA
Treatment: Drugs - IVA

Experimental: ELX/TEZ/IVA - Part A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks.
Part B: Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 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
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
From Baseline through Week 100

Eligibility
Key inclusion criteria
- Currently participating in study VX17-659-105 (NCT03447262)
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 study VX17-659-105 that would pose an additional risk
to the participant in the opinion of the investigator

- Current participation in an investigational drug trial (other than study VX17-659-105)

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
9/08/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
14/12/2022
Sample size
Target
Accrual to date
Final
458
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
Perth Children's Hospital - Nedlands
Recruitment hospital [6] 0 0
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital - New Lambton
Recruitment hospital [7] 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
- New Lambton
Recruitment postcode(s) [6] 0 0
- South Brisbane
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
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Idaho
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Indiana
Country [9] 0 0
United States of America
State/province [9] 0 0
Iowa
Country [10] 0 0
United States of America
State/province [10] 0 0
Kentucky
Country [11] 0 0
United States of America
State/province [11] 0 0
Maryland
Country [12] 0 0
United States of America
State/province [12] 0 0
Massachusetts
Country [13] 0 0
United States of America
State/province [13] 0 0
Michigan
Country [14] 0 0
United States of America
State/province [14] 0 0
Minnesota
Country [15] 0 0
United States of America
State/province [15] 0 0
Mississippi
Country [16] 0 0
United States of America
State/province [16] 0 0
Missouri
Country [17] 0 0
United States of America
State/province [17] 0 0
New Hampshire
Country [18] 0 0
United States of America
State/province [18] 0 0
New Jersey
Country [19] 0 0
United States of America
State/province [19] 0 0
New York
Country [20] 0 0
United States of America
State/province [20] 0 0
North Carolina
Country [21] 0 0
United States of America
State/province [21] 0 0
Ohio
Country [22] 0 0
United States of America
State/province [22] 0 0
Oklahoma
Country [23] 0 0
United States of America
State/province [23] 0 0
Oregon
Country [24] 0 0
United States of America
State/province [24] 0 0
Pennsylvania
Country [25] 0 0
United States of America
State/province [25] 0 0
South Dakota
Country [26] 0 0
United States of America
State/province [26] 0 0
Tennessee
Country [27] 0 0
United States of America
State/province [27] 0 0
Texas
Country [28] 0 0
United States of America
State/province [28] 0 0
Utah
Country [29] 0 0
United States of America
State/province [29] 0 0
Washington
Country [30] 0 0
Canada
State/province [30] 0 0
Edmonton
Country [31] 0 0
Canada
State/province [31] 0 0
Halifax
Country [32] 0 0
Canada
State/province [32] 0 0
Toronto
Country [33] 0 0
Denmark
State/province [33] 0 0
Copenhagen
Country [34] 0 0
Germany
State/province [34] 0 0
Berlin
Country [35] 0 0
Germany
State/province [35] 0 0
Essen
Country [36] 0 0
Germany
State/province [36] 0 0
Frankfurt
Country [37] 0 0
Germany
State/province [37] 0 0
Hannover
Country [38] 0 0
Germany
State/province [38] 0 0
Jena
Country [39] 0 0
Germany
State/province [39] 0 0
Koeln
Country [40] 0 0
Germany
State/province [40] 0 0
Lübeck
Country [41] 0 0
Germany
State/province [41] 0 0
Muenchen
Country [42] 0 0
Germany
State/province [42] 0 0
München
Country [43] 0 0
Ireland
State/province [43] 0 0
Cork
Country [44] 0 0
Ireland
State/province [44] 0 0
Dublin
Country [45] 0 0
Ireland
State/province [45] 0 0
Galway
Country [46] 0 0
Ireland
State/province [46] 0 0
Limerick
Country [47] 0 0
Israel
State/province [47] 0 0
Haifa
Country [48] 0 0
Israel
State/province [48] 0 0
Jerusalem
Country [49] 0 0
Israel
State/province [49] 0 0
Petah Tikva
Country [50] 0 0
Israel
State/province [50] 0 0
Tel Hashomer
Country [51] 0 0
Poland
State/province [51] 0 0
Lomianki
Country [52] 0 0
Spain
State/province [52] 0 0
Barcelona
Country [53] 0 0
Spain
State/province [53] 0 0
Madrid
Country [54] 0 0
Spain
State/province [54] 0 0
Sabadell
Country [55] 0 0
Spain
State/province [55] 0 0
Sevilla
Country [56] 0 0
Spain
State/province [56] 0 0
Valencia
Country [57] 0 0
Switzerland
State/province [57] 0 0
Bern
Country [58] 0 0
Switzerland
State/province [58] 0 0
Zürich
Country [59] 0 0
United Kingdom
State/province [59] 0 0
Cambridge
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Glasgow
Country [61] 0 0
United Kingdom
State/province [61] 0 0
Leeds
Country [62] 0 0
United Kingdom
State/province [62] 0 0
Liverpool
Country [63] 0 0
United Kingdom
State/province [63] 0 0
London
Country [64] 0 0
United Kingdom
State/province [64] 0 0
Manchester
Country [65] 0 0
United Kingdom
State/province [65] 0 0
Newcastle Upon Tyne
Country [66] 0 0
United Kingdom
State/province [66] 0 0
Nottingham
Country [67] 0 0
United Kingdom
State/province [67] 0 0
Penarth

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

Ethics approval
Ethics application status

Summary
Brief summary
This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor
(TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic
fibrosis (CF).
Trial website
https://clinicaltrials.gov/ct2/show/NCT04043806
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/ct2/show/NCT04043806