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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03911713




Registration number
NCT03911713
Ethics application status
Date submitted
9/04/2019
Date registered
11/04/2019

Titles & IDs
Public title
A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Scientific title
A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Secondary ID [1] 0 0
2018-003970-28
Secondary ID [2] 0 0
VX18-561-101
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-561
Treatment: Drugs - IVA
Treatment: Drugs - Placebo
Treatment: Drugs - Placebo

Active comparator: Ivacaftor - Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.

Experimental: VX-561: 25 mg - Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.

Experimental: VX-561: 50 mg - Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.

Experimental: VX-561: 150 mg - Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.

Experimental: VX-561: 250 mg - Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.


Treatment: Drugs: VX-561
VX-561 tablets for oral administration.

Treatment: Drugs: IVA
150-mg film-coated tablet for oral administration.

Treatment: Drugs: Placebo
Placebo matched to IVA.

Treatment: Drugs: Placebo
Placebos matched to VX-561.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Timepoint [1] 0 0
From Baseline at Week 12
Secondary outcome [1] 0 0
Absolute Change in Sweat Chloride (SwCl)
Timepoint [1] 0 0
From Baseline at Week 12
Secondary outcome [2] 0 0
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
Timepoint [2] 0 0
At Week 4
Secondary outcome [3] 0 0
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [3] 0 0
Baseline up to Week 16

Eligibility
Key inclusion criteria
Key

* Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
* On ivacaftor therapy
* FEV1 value =40% and =100% of predicted mean for age, sex, and height

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of clinically significant cirrhosis with or without portal hypertension
* History of solid organ or hematological transplantation
* Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply

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 assessing the outcomes
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
17/04/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
20/08/2020
Sample size
Target
Accrual to date
Final
77
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [2] 0 0
Alfred Hospital - Melbourne, VIC
Recruitment hospital [3] 0 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [4] 0 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 0 0
- Chermside
Recruitment postcode(s) [2] 0 0
- Melbourne, VIC
Recruitment postcode(s) [3] 0 0
- South Brisbane
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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United States of America
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Arizona
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United States of America
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California
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Colorado
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Florida
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Indiana
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Kansas
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Kentucky
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Maryland
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Massachusetts
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Michigan
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Minnesota
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Missouri
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New Hampshire
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New York
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North Carolina
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Ohio
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Oklahoma
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Pennsylvania
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South Carolina
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Texas
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Utah
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United States of America
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Wisconsin
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Belgium
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Gent
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Germany
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Erlangen
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Germany
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Essen
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Germany
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Halle
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Germany
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Muenchen
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Germany
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Würzburg
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Ireland
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Dublin 12
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Ireland
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Dublin 9
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Ireland
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Dublin
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Ireland
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Limerick
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Netherlands
State/province [34] 0 0
Nijmegen
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United Kingdom
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Glasgow
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United Kingdom
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Leeds
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United Kingdom
State/province [37] 0 0
Liverpool

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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03911713