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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
Sex
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
1/09/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/07/2018
Sample size
Target
Accrual to date
Final
41
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
Trial website
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02730208