The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02730208




Registration number
NCT02730208
Ethics application status
Date submitted
31/03/2016
Date registered
6/04/2016
Date last updated
23/10/2019

Titles & IDs
Public title
A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Scientific title
A Phase 2, Randomized, Placebo-Controlled, Double-blind Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Secondary ID [1] 0 0
VX15-661-112
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 - Tezacaftor/Ivacaftor
Treatment: Drugs - Ivacaftor
Treatment: Drugs - Placebo
Treatment: Drugs - Placebo

Experimental: TEZ/IVA - Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.

Placebo Comparator: Placebo - Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.


Treatment: Drugs: Tezacaftor/Ivacaftor
TEZ 100 mg/IVA 150 mg fixed-dose combination tablet.

Treatment: Drugs: Ivacaftor
IVA 150 mg tablet.

Treatment: Drugs: Placebo
Placebo matched to TEZ/IVA fixed-dose combination tablet.

Treatment: Drugs: Placebo
Placebo matched to IVA tablet.

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

Outcomes
Primary outcome [1] 0 0
Absolute Change in Total Brody/CF-CT Score - The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease.
Timepoint [1] 0 0
From Baseline at Week 72
Secondary outcome [1] 0 0
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
Day 1 up to Week 76

Eligibility
Key inclusion criteria
- Homozygous for the F508del CFTR mutation

- Confirmed diagnosis of CF

- Percent predicted forced expiratory volume (ppFEV1) =70% of predicted normal for age,
sex, and height during screening.

- Stable CF disease as judged by the investigator
Minimum age
12 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- History of any comorbidity that, in the opinion of the investigator, might confound
the results of the study or pose an additional risk in administering study drug to the
subject.

- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
therapy (including antibiotics) for pulmonary disease within 28 days before Day 1
(first dose of study drug)

- Pregnant or nursing females.

- Sexually active subjects of reproductive potential who are not willing to follow the
contraception requirements.

- Any contraindication to undergoing chest imaging, as per the site's institutional
guidelines

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
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
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
- Chermside
Recruitment hospital [2] 0 0
- Melbourne
Recruitment hospital [3] 0 0
- Nedlands
Recruitment hospital [4] 0 0
- New Lambton Heights
Recruitment hospital [5] 0 0
- Parkville SIC
Recruitment hospital [6] 0 0
- Randwick
Recruitment hospital [7] 0 0
- South Brisbane
Recruitment hospital [8] 0 0
- Subiaco
Recruitment hospital [9] 0 0
- Westmead
Recruitment postcode(s) [1] 0 0
- Chermside
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment postcode(s) [3] 0 0
- Nedlands
Recruitment postcode(s) [4] 0 0
- New Lambton Heights
Recruitment postcode(s) [5] 0 0
- Parkville SIC
Recruitment postcode(s) [6] 0 0
- Randwick
Recruitment postcode(s) [7] 0 0
- South Brisbane
Recruitment postcode(s) [8] 0 0
- Subiaco
Recruitment postcode(s) [9] 0 0
- Westmead

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

Ethics approval
Ethics application status

Summary
Brief summary
The primary purpose of study is to evaluate the treatment effect of tezacaftor in combination
with ivacaftor (TEZ/IVA) on chest imaging endpoints using low-dose computed tomography (LDCT)
at Week 72, and to evaluate the safety of TEZ/IVA through Week 72.
Trial website
https://clinicaltrials.gov/show/NCT02730208
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
Other publications