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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
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
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Alabama
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California
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Connecticut
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Mississippi
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Missouri
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Oregon
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Edmonton
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Essen
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Jena
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Koeln
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Muenchen
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Limerick
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Haifa
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Jerusalem
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Petah Tikva
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Israel
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Tel Hashomer
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Barcelona
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Sabadell
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Newcastle Upon Tyne
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Nottingham
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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/study/NCT04043806
Trial related presentations / publications
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
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/NCT04043806