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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/NCT03447262




Registration number
NCT03447262
Ethics application status
Date submitted
21/02/2018
Date registered
27/02/2018
Date last updated
25/01/2022

Titles & IDs
Public title
A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
Scientific title
A Phase 3, Open-label Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
Secondary ID [1] 0 0
2017-004134-29
Secondary ID [2] 0 0
VX17-659-105
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
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 - VX-659/TEZ/IVA
Treatment: Drugs - IVA

Experimental: VX-659/TEZ/IVA TC - Participants from parent studies VX17-659-102 (NCT03447249) or VX17-659-103 (NCT03460990) were administered VX-659 240 milligrams (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the TC treatment period for up to 96 weeks in the current study VX17-659-105.


Treatment: Drugs: VX-659/TEZ/IVA
Fixed-dose combination tablets for oral administration qd in the morning.

Treatment: Drugs: IVA
IVA tablet qd in the evening.
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 Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
From Day 1 up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100)
Secondary outcome [1] 0 0
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102
Timepoint [1] 0 0
From Baseline at Week 72 (Study 659-105)
Secondary outcome [2] 0 0
Absolute Change in ppFEV1 for Participants From the Parent Study 659-103
Timepoint [2] 0 0
From Baseline at Week 72 (Study 659-105)
Secondary outcome [3] 0 0
Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102
Timepoint [3] 0 0
From Baseline at Week 24 (Study 659-105)
Secondary outcome [4] 0 0
Absolute Change in SwCl for Participants From the Parent Study 659-103
Timepoint [4] 0 0
From Baseline at Week 24 (Study 659-105)
Secondary outcome [5] 0 0
Number of Pulmonary Exacerbations (PEx) for Participants From the Parent Study 659-102
Timepoint [5] 0 0
From Baseline up to Week 96 (Study 659-105)
Secondary outcome [6] 0 0
Number of PEx for Participants From the Parent Study 659-103
Timepoint [6] 0 0
From Baseline up to Week 96 (Study 659-105)
Secondary outcome [7] 0 0
Number of Participants With at Least One PEx for Participants From the Parent Study 659-102
Timepoint [7] 0 0
From Baseline up to Week 96 (Study 659-105)
Secondary outcome [8] 0 0
Number of Participants With at Least One PEx for Participants From the Parent Study 659-103
Timepoint [8] 0 0
From Baseline up to Week 96 (Study 659-105)
Secondary outcome [9] 0 0
Absolute Change in Body Mass Index (BMI) for Participants From the Parent Study 659-102
Timepoint [9] 0 0
From Baseline at Week 72 (Study 659-105)
Secondary outcome [10] 0 0
Absolute Change in BMI for Participants From the Parent Study 659-103
Timepoint [10] 0 0
From Baseline at Week 72 (Study 659-105)
Secondary outcome [11] 0 0
Absolute Change in BMI Z-score for Participants From the Parent Study 659-102 (Participants <=20 Years Old at Parent Study Baseline)
Timepoint [11] 0 0
From Baseline at Week 60 (Study 659-105)
Secondary outcome [12] 0 0
Absolute Change in BMI Z-score for Participants From The Parent Study 659-103 (Participants <=20 Years Old at Parent Study Baseline)
Timepoint [12] 0 0
From Baseline at Week 60 (Study 659-105)
Secondary outcome [13] 0 0
Absolute Change in Body Weight for Participants From the Parent Study 659-102
Timepoint [13] 0 0
From Baseline at Week 72 (Study 659-105)
Secondary outcome [14] 0 0
Absolute Change in Body Weight for Participants From the Parent Study 659-103
Timepoint [14] 0 0
From Baseline at Week 72 (Study 659-105)
Secondary outcome [15] 0 0
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for Participants From the Parent Study 659-102
Timepoint [15] 0 0
From Baseline at Week 72 (Study 659-105)
Secondary outcome [16] 0 0
Absolute Change in CFQ-R Respiratory Domain Score for Participants From the Parent Study 659-103
Timepoint [16] 0 0
From Baseline at Week 72 (Study 659-105)

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
Sex
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
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
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/07/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
9/09/2020
Sample size
Target
Accrual to date
Final
484
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
Recruitment hospital [4] 0 0
Institute for Respiratory Health, Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [5] 0 0
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital - New Lambton
Recruitment hospital [6] 0 0
Telethon Kids Institute - Perth
Recruitment hospital [7] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [8] 0 0
Lady Cilento 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
Recruitment postcode(s) [4] 0 0
- Nedlands
Recruitment postcode(s) [5] 0 0
- New Lambton
Recruitment postcode(s) [6] 0 0
- Perth
Recruitment postcode(s) [7] 0 0
- Randwick
Recruitment postcode(s) [8] 0 0
- South Brisbane
Recruitment outside Australia
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Alabama
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Lubeck
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Israel
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Israel
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Tel HaShomer
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Manchester
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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 will evaluate the long-term safety and tolerability of VX-659 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/ct2/show/NCT03447262
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
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/ct2/show/NCT03447262