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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/NCT03911713




Registration number
NCT03911713
Ethics application status
Date submitted
9/04/2019
Date registered
11/04/2019
Date last updated
25/01/2022

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
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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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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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Wisconsin
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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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Muenchen
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Würzburg
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Dublin 12
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Dublin 9
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Dublin
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Ireland
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Limerick
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Netherlands
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Nijmegen
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Glasgow
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Leeds
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United Kingdom
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Liverpool

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

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and
pharmacokinetic (PK) effect of VX-561.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03911713
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
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Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
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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/NCT03911713