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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03537651




Registration number
NCT03537651
Ethics application status
Date submitted
15/05/2018
Date registered
25/05/2018
Date last updated
11/06/2020

Titles & IDs
Public title
A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Subjects 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
Treatment: Drugs - IVA

Experimental: TEZ + IVA - Subjects <30 kg will receive 50 mg TEZ/ 75 mg IVA as a FDC tablet in the morning and 75 mg IVA as a mono tablet in the evening.
Subjects >=30 kg will receive 100 mg TEZ/ 150 mg IVA FDC tablet in the morning and 150 mg IVA as a mono tablet in the evening.


Treatment: Drugs: TEZ
Fixed-dose Combination (FDC) tablet (TEZ/IVA)

Treatment: Drugs: IVA
FDC tablet (TEZ/IVA)

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability of long-term TEZ/IVA treatment based on adverse events (AEs) and serious adverse events (SAEs)
Timepoint [1] 0 0
from baseline through safety follow-up (up to 28 days after Last Dose at Week 96)
Secondary outcome [1] 0 0
Absolute change in lung clearance index2.5 (LCI2.5)
Timepoint [1] 0 0
from baseline through 96 weeks
Secondary outcome [2] 0 0
Absolute change in sweat chloride
Timepoint [2] 0 0
from baseline through 96 weeks
Secondary outcome [3] 0 0
Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score
Timepoint [3] 0 0
from baseline through 96 weeks
Secondary outcome [4] 0 0
Absolute change in body mass index (BMI)
Timepoint [4] 0 0
from baseline through 96 weeks

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
Gender
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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United States of America
State/province [2] 0 0
Alaska
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United States of America
State/province [3] 0 0
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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Ohio
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Pennsylvania
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South Carolina
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South Dakota
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Texas
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Virginia
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Washington
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United States of America
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Wisconsin
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Belgium
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Brussels
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Belgium
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Leuven
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Canada
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British Columbia
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Canada
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Ontario
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Canada
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Quebec
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Denmark
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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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Germany
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Hannover
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Germany
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Jena
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Germany
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Koeln
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Germany
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Tuebingen
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Ireland
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Dublin
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Ireland
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Limerick
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Poland
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Lomianki
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Switzerland
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Bern
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Switzerland
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Zürich
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United Kingdom
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Leeds
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United Kingdom
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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 will evaluate the long-term safety and tolerability of tezacaftor in combination
with ivacaftor (TEZ/IVA) in subjects with cystic fibrosis (CF) aged 6 years and older,
homozygous or heterozygous for the F508del mutation.
Trial website
https://clinicaltrials.gov/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

Summary results
Other publications