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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03525574




Registration number
NCT03525574
Ethics application status
Date submitted
2/05/2018
Date registered
15/05/2018
Date last updated
27/05/2019

Titles & IDs
Public title
A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
Scientific title
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
Secondary ID [1] 0 0
2018-000185-11
Secondary ID [2] 0 0
VX17-445-105
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
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
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

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - VX-445
Treatment: Drugs - TEZ
Treatment: Drugs - IVA
Treatment: Drugs - IVA

Experimental: Open-label Triple Combination - Subjects will receive 200 mg VX-445/ 100 mg TEZ/ 150 mg IVA as FDC tablets in the morning and 150 mg IVA as mono tablet in the evening.
Parent studies are Phase 3 Vertex studies investigating VX-445 in combination with TEZ and IVA. This includes Studies VX17-445-102 and VX17-445-103.


Treatment: Drugs: VX-445
Fixed-dose combination (FDC) tablets (VX-445/TEZ/IVA)

Treatment: Drugs: TEZ
FDC tablets (VX-445/TEZ/IVA)

Treatment: Drugs: IVA
FDC tablets (VX-445/TEZ/IVA)

Treatment: Drugs: IVA
IVA tablet

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

Outcomes
Primary outcome [1] 0 0
Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs)
Timepoint [1] 0 0
from baseline through safety follow-up (up to 100 weeks)
Secondary outcome [1] 0 0
Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)
Timepoint [1] 0 0
from baseline through last dose of study drug (up to 96 weeks)
Secondary outcome [2] 0 0
Absolute change in sweat chloride (SwCl)
Timepoint [2] 0 0
from baseline through last dose of study drug (up to 96 weeks)
Secondary outcome [3] 0 0
Number of pulmonary exacerbations (PEx)
Timepoint [3] 0 0
from baseline through last dose of study drug (up to 96 weeks)
Secondary outcome [4] 0 0
Time to first PEx
Timepoint [4] 0 0
from baseline through last dose of study drug (up to 96 weeks)
Secondary outcome [5] 0 0
Absolute change in body mass index (BMI)
Timepoint [5] 0 0
from baseline through last dose of study drug (up to 96 weeks)
Secondary outcome [6] 0 0
Absolute change in BMI z-score
Timepoint [6] 0 0
from baseline through last dose of study drug (up to 96 weeks)
Secondary outcome [7] 0 0
Absolute change in body weight
Timepoint [7] 0 0
from baseline through last dose of study drug (up to 96 weeks)
Secondary outcome [8] 0 0
Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score
Timepoint [8] 0 0
from baseline through last dose of study drug (up to 96 weeks)

Eligibility
Key inclusion criteria
- Completed study drug treatment in a parent study; or had study drug interruption(s) in
a parent study but completed study visits up to the last scheduled visit of the
Treatment Period in the parent study.
Minimum age
12 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- History of drug intolerance in a parent study that would pose an additional risk to
the subject in the opinion of the investigator.

- Current participation in an investigational drug trial (other than a parent study)

Other protocol defined Inclusion/Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
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
Women & Children's Hospital - North Adelaide
Recruitment hospital [2] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [3] 0 0
Mater Misericordiae Ltd - South Brisbane
Recruitment hospital [4] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [5] 0 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 0 0
- North Adelaide
Recruitment postcode(s) [2] 0 0
- Parkville
Recruitment postcode(s) [3] 0 0
- South Brisbane
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment outside Australia
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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 VX-445 in triple
combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis
(CF) who are homozygous or heterozygous for the F508del mutation
Trial website
https://clinicaltrials.gov/show/NCT03525574
Trial related presentations / publications
Public notes

Contacts
Principal investigator
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Email 0 0
Contact person for public queries
Name 0 0
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Summary results
Other publications