Study of VX-809 in Cystic Fibrosis Subjects With the ?F508-CFTR Gene Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00865904
• Date of registration: 18/03/2009
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study of VX-809 in Cystic Fibrosis Subjects With the ?F508-CFTR Gene Mutation
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics, and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the ?F508-CFTR Gene Mutation
• Date of first enrolment: March 2009
• Target sample size: 93
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00865904
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Belgium, Canada, Germany, Netherlands, United States

Contacts

• Name: Medical Monitor
• Affiliation: Vertex Pharmaceuticals Incorporated

Key inclusion & exclusion criteria

Inclusion Criteria:

- Confirmed diagnosis of CF with ?F508-CFTR mutation in both alleles

- Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent
(%) of predicted normal for age, gender, and height

- Weight >=40 kilograms (kg) and body mass index greater than or equal to 18.5 kilogram
per square meter (kg/m^2)

- Screening laboratory values, tests, and physical examination within acceptable ranges

- Negative pregnancy test (for women of child-bearing potential)

- Able and willing to follow contraceptive requirements

- Willing to remain on a stable medication regimen for the duration of study
participation

Exclusion Criteria:

- History of any illness, or any ongoing acute illness, that could impact the safety of
the study participant or may confound results of study

- Pulmonary exacerbation or changes in therapy for pulmonary disease within 14 days
before receiving the first dose of study drug

- Impaired hepatic or renal function

- History of organ or hematological transplant



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: VX-809

Drug: Placebo

Primary Outcome(s)

Safety and Tolerability Based on Adverse Events (AEs) [Time Frame: Up to 14 days after last dose (last dose = Day 28)]

Secondary Outcome(s)

Change From Baseline in Forced Expiratory Flow Over the Middle Half of the FVC (FEF25-75) at Day 28 [Time Frame: Baseline, Day 28]

Change From Baseline in Nasal Potential Difference (NPD) of Zero Chloride Plus Isoproterenol Response at Day 28 [Time Frame: Baseline, Day 28]

Change From Baseline in Percent Predicted FEV1 at Day 28 [Time Frame: Baseline, Day 28]

Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24) of VX-809 [Time Frame: Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post dose)]

Change From Baseline in Forced Vital Capacity (FVC) at Day 28 [Time Frame: Baseline, Day 28]

Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Domain Scores at Day 28 [Time Frame: Baseline, Day 28]

Maximum Plasma Concentration (Cmax) of VX-809 [Time Frame: Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, 24, and 30-60 hours post dose)]

Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Day 28 [Time Frame: Baseline, Day 28]

Change From Baseline in Sweat Chloride at Day 28 [Time Frame: Baseline, Day 28]

Secondary ID(s)

VX08-809-101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 28/08/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00865904