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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/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
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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
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Secondary ID [1]
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2017-002968-40
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Secondary ID [2]
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VX17-661-116
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Cystic fibrosis
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Respiratory
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Other respiratory disorders / diseases
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Oral and Gastrointestinal
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Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
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Inflammatory and Immune System
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Connective tissue diseases
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Inflammatory and Immune System
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Other inflammatory or immune system disorders
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Intervention/exposure
Study type
Interventional
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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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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Assessment method [1]
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Timepoint [1]
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Day 1 up to Week 100
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Secondary outcome [1]
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Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group)
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Assessment method [1]
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The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
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Timepoint [1]
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From Parent Study 115 Baseline at Week 96 (Study 116)
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Secondary outcome [2]
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Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS
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Assessment method [2]
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The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
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Timepoint [2]
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From Parent Study 113B Baseline at Week 96 (Study 116)
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Secondary outcome [3]
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Part A: Absolute Change in Sweat Chloride (SwCl) for 115/116 FAS (TEZ/IVA Group)
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Assessment method [3]
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Sweat samples were collected using an approved collection device.
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Timepoint [3]
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From Parent Study 115 Baseline at Week 96 (Study 116)
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Secondary outcome [4]
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Part A: Absolute Change in SwCl for 113B/116 FAS
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Assessment method [4]
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Sweat samples were collected using an approved collection device.
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Timepoint [4]
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From Parent Study 113B Baseline at Week 96 (Study 116)
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Secondary outcome [5]
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Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group)
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Assessment method [5]
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
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Timepoint [5]
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From Parent Study 115 Baseline at Week 96 (Study 116)
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Secondary outcome [6]
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Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS
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Assessment method [6]
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
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Timepoint [6]
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From Parent Study 113B Baseline at Week 96 (Study 116)
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Secondary outcome [7]
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Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group)
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Assessment method [7]
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BMI was defined as weight in kilograms (kg) divided by squared height in meters (m\^2).
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Timepoint [7]
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From Parent Study 115 Baseline at Week 96 (Study 116)
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Secondary outcome [8]
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Part A: Absolute Change in BMI for 113B/116 FAS
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Assessment method [8]
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BMI was defined as weight in kg divided by m\^2.
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Timepoint [8]
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From Parent Study 113B Baseline at Week 96 (Study 116)
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Secondary outcome [9]
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Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [9]
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Timepoint [9]
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Day 1 up to Week 192
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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
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Minimum age
6
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
25/04/2018
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Date of last participant enrolment
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Actual
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Date of last data collection
Anticipated
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Actual
29/09/2023
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Sample size
Target
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Accrual to date
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Final
130
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Perth Children's Hospital - Nedlands
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Recruitment hospital [2]
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John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital - New Lambton
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Recruitment hospital [3]
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Lady Cilento Children's Hospital - South Brisbane
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Recruitment hospital [4]
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The Children's Hospital at Westmead - Westmead
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Recruitment postcode(s) [1]
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- Nedlands
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Recruitment postcode(s) [2]
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- New Lambton
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Recruitment postcode(s) [3]
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- South Brisbane
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Recruitment postcode(s) [4]
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- Westmead
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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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Southampton
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Vertex Pharmaceuticals Incorporated
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Address
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Ethics approval
Ethics application status
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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.
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Trial website
https://clinicaltrials.gov/study/NCT03537651
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Trial related presentations / publications
Sawicki GS, Chilvers M, McNamara J, Naehrlich L, Saunders C, Sermet-Gaudelus I, Wainwright CE, Ahluwalia N, Campbell D, Harris RS, Paz-Diaz H, Shih JL, Davies JC. A Phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children >/= 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant. J Cyst Fibros. 2022 Jul;21(4):675-683. doi: 10.1016/j.jcf.2022.02.003. Epub 2022 Feb 18. 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.
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Public notes
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Contacts
Principal investigator
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/51/NCT03537651/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/51/NCT03537651/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT03537651
Download to PDF