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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/NCT03537651




Registration number
NCT03537651
Ethics application status
Date submitted
15/05/2018
Date registered
25/05/2018
Date last updated
19/04/2024

Titles & IDs
Public title
A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation
Scientific title
A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation
Secondary ID [1] 0 0
2017-002968-40
Secondary ID [2] 0 0
VX17-661-116
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 - TEZ/IVA
Treatment: Drugs - IVA

Experimental: TEZ/IVA - Part A: Participants weighing less than (<)40 kilograms (kg) at Day 1 received tezacaftor (TEZ) 50 milligrams (mg) once daily (qd)/ivacaftor (IVA) 75 mg every 12 hours (q12h) and the participants weighing greater than or equals to (>=) 40 kg at Day 1 received TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 96 weeks. Doses were adjusted upward for changes in body weight and/or age.
Part B: Participants weighing <30 kg at Day 1 received TEZ 50 mg qd/IVA 75 mg q12h and the participants weighing >=30 kg at Day 1 received TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 192 weeks. Doses were adjusted upward for changes in body weight and/or age.


Treatment: Drugs: TEZ/IVA
Fixed-dose combination 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
Day 1 up to Week 100
Secondary outcome [1] 0 0
Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group)
Timepoint [1] 0 0
From Parent Study 115 Baseline at Week 96 (Study 116)
Secondary outcome [2] 0 0
Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS
Timepoint [2] 0 0
From Parent Study 113B Baseline at Week 96 (Study 116)
Secondary outcome [3] 0 0
Part A: Absolute Change in Sweat Chloride (SwCl) for 115/116 FAS (TEZ/IVA Group)
Timepoint [3] 0 0
From Parent Study 115 Baseline at Week 96 (Study 116)
Secondary outcome [4] 0 0
Part A: Absolute Change in SwCl for 113B/116 FAS
Timepoint [4] 0 0
From Parent Study 113B Baseline at Week 96 (Study 116)
Secondary outcome [5] 0 0
Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group)
Timepoint [5] 0 0
From Parent Study 115 Baseline at Week 96 (Study 116)
Secondary outcome [6] 0 0
Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS
Timepoint [6] 0 0
From Parent Study 113B Baseline at Week 96 (Study 116)
Secondary outcome [7] 0 0
Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group)
Timepoint [7] 0 0
From Parent Study 115 Baseline at Week 96 (Study 116)
Secondary outcome [8] 0 0
Part A: Absolute Change in BMI for 113B/116 FAS
Timepoint [8] 0 0
From Parent Study 113B Baseline at Week 96 (Study 116)
Secondary outcome [9] 0 0
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [9] 0 0
Day 1 up to Week 192

Eligibility
Key inclusion criteria
- Completed the Week 24 Visit in Study 113 Part B or the Week 8 Visit in Study 115

- Eligible CFTR Mutation
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Pregnant and nursing females

- History of poor compliance with study drug and/or procedures in a previous study as
deemed by the investigator

- Ongoing participation in another study with investigational drug

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
25/04/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
29/09/2023
Sample size
Target
Accrual to date
Final
130
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Perth Children's Hospital - Nedlands
Recruitment hospital [2] 0 0
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital - New Lambton
Recruitment hospital [3] 0 0
Lady Cilento Children's Hospital - South Brisbane
Recruitment hospital [4] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
- Nedlands
Recruitment postcode(s) [2] 0 0
- New Lambton
Recruitment postcode(s) [3] 0 0
- South Brisbane
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment outside Australia
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United States of America
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Alabama
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Alaska
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Arkansas
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California
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Colorado
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Delaware
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Florida
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Georgia
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Idaho
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Indiana
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Massachusetts
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Minnesota
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Missouri
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New Hampshire
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New York
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North Carolina
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Virginia
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Washington
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Wisconsin
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Belgium
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Brussels
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Leuven
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British Columbia
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Ontario
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Quebec
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Copenhagen
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France
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Bordeaux cedex
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France
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Paris Cedex 15
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Germany
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Essen
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Germany
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Frankfurt
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Germany
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Giessen
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Hannover
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Jena
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Bern
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Zürich
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Leeds
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London
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United Kingdom
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Southampton

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

Ethics approval
Ethics application status

Summary
Brief summary
This study evaluates the long-term safety and tolerability of tezacaftor in combination with
ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older,
homozygous or heterozygous for the F508del mutation.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03537651
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
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Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries