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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02392234




Registration number
NCT02392234
Ethics application status
Date submitted
12/03/2015
Date registered
18/03/2015
Date last updated
12/06/2018

Titles & IDs
Public title
A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation
Scientific title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation, and a Second Allele With a CFTR Mutation Predicted to Have Residual Function
Secondary ID [1] 0 0
2014-004788-18
Secondary ID [2] 0 0
VX14-661-108
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-661/Ivacaftor
Treatment: Drugs - Ivacaftor
Treatment: Drugs - Placebo matched to VX-661/ ivacaftor
Treatment: Drugs - Placebo matched to Ivacaftor

Experimental: VX-661/Ivacaftor combination -

Experimental: Ivacaftor monotherapy -

Placebo comparator: Placebo -


Treatment: Drugs: VX-661/Ivacaftor
Fixed dose combination tablet, oral use

Treatment: Drugs: Ivacaftor
Tablet, oral use

Treatment: Drugs: Placebo matched to VX-661/ ivacaftor
Fixed dose combination tablet, oral use

Treatment: Drugs: Placebo matched to Ivacaftor
Tablet, oral use
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8
Timepoint [1] 0 0
Baseline, Week 4 and Week 8 of each treatment period
Secondary outcome [1] 0 0
Absolute Change From Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Average of Week 4 and Week 8
Timepoint [1] 0 0
Baseline, Week 4 and Week 8 of each treatment period
Secondary outcome [2] 0 0
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [2] 0 0
Day 1 up to Week 28
Secondary outcome [3] 0 0
Relative Change From Study Baseline in ppFEV1 at Average of Week 4 and Week 8
Timepoint [3] 0 0
Baseline, Week 4 and Week 8 of each treatment period
Secondary outcome [4] 0 0
Absolute Change From Study Baseline in Sweat Chloride at Average of Week 4 and Week 8
Timepoint [4] 0 0
Baseline, Week 4 and Week 8 of each treatment period
Secondary outcome [5] 0 0
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661), IVA and IVA Metabolite (M1 IVA) After Administration of VX-661/IVA Combination Therapy
Timepoint [5] 0 0
Pre-morning dose on Week 8 of each treatment period
Secondary outcome [6] 0 0
Ctrough of IVA and IVA Metabolite (M1 IVA) After Administration of IVA Monotherapy
Timepoint [6] 0 0
Pre-morning dose on Week 8 of each treatment period

Eligibility
Key inclusion criteria
* Heterozygous for F508del-CFTR and a second allele with a CFTR mutation predicted to have residual function
* Forced Expiratory Volume in 1 Second (FEV1) greater than or equal to (=) 40 percent (%) and less than or equal to (=) 90% of predicted normal for age, sex, and height during screening
* Sweat chloride value =60 millimole per liter (mmol/L) during screening OR as documented in the participant's medical record
* 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 participant
* An acute upper or lower respiratory infection, pulmonary exacerbation
* History of solid organ or hematological transplantation
* Ongoing or prior participation in an investigational drug study (including studies investigating VX-661, lumacaftor [VX-809], and/or ivacaftor) within 30 days of screening
* Pregnant and nursing females
* Sexually active participants of reproductive potential who are not willing to follow the contraception requirements

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
Crossover
Other design features
Phase
Phase 3
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
1/03/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/02/2017
Sample size
Target
Accrual to date
Final
248
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Adelaide
Recruitment hospital [2] 0 0
- Melbourne
Recruitment hospital [3] 0 0
- South Brisbane
Recruitment hospital [4] 0 0
- Westmead
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Melbourne
Recruitment postcode(s) [3] 0 0
- South Brisbane
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment outside Australia
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United States of America
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Alabama
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Arizona
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California
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Colorado
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Illinois
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Massachusetts
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Michigan
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Toronto
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Vancouver
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Herault
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France
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Roma
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Strathclyde
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West Yorkshire

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/NCT02392234