Cystic Fibrosis MedDRA version: 14.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male or female subjects with confirmed diagnosis of CF - Must have the G551D CFTR mutation in at least 1 allele (any known or unknown mutations allowed in second allele) - FEV1 >90% of predicted normal for age, gender, and height at screening - LCI threshold at screening greater than the established ULN of 7.4 - 6 years of age or older on the date of signed informed consent form (ICF), and where appropriate, date of assent - Weight greater than or equal to 15 kg without shoes at screening - Able to swallow tablets Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 6 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: - Abnormal renal function at screening, defined as creatinine clearance <75 mL/min/1.73 m2 using the Counahan-Barratt equation (for subjects 6 to 17 years of age) or <50 mL/min using the Cockcroft-Gault equation (for subjects 18 years of age or older) - History of solid organ or hematological transplantation - Colonization with organisms associated with a more rapid decline in pulmonary status (e.g., B cenocepacia, B dolosa, and M abcessus) at screening - Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 30 days prior to screening. - Use of inhaled hypertonic saline treatment. (Subjects who have stopped inhaled hypertonic saline treatment will be eligible to participate, but they must have withheld treatment for 48 hours prior to the screening visit and have undergone a washout period of at least 2 weeks prior to the Period 1 Day 1 visit) - Concomitant use of any inhibitors or inducers of cytochrome P450 (CYP) 3A, including consumption of certain herbal medications (e.g., St. John’s Wort), and grapefruit/grapefruit juice. Subjects must stop consuming these items 14 days prior to Period 1 Day 1.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the effect of VX-770 on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D CFTR mutation on at least 1 allele;Secondary Objective: - To evaluate the safety of VX-770 in subjects aged 6 years and older with CF who have the G551D CFTR mutation on at least 1 allele - To evaluate the efficacy of VX-770 in subjects aged 6 years and older with CF who have the G551D CFTR mutation on at least 1 allele ;Primary end point(s): Absolute change from baseline in Lung Clearance Index (LCI);Timepoint(s) of evaluation of this end point: 4 Weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety as determined by adverse events, clinical laboratory values (chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), and vital signs Efficacy as determined by: • Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1) • Change from baseline in sweat chloride • Change from baseline in CF Questionnaire-Revised (CFQ-R) ;Timepoint(s) of evaluation of this end point: Safety: 16 weeks; Efficacy: 4 Weeks | — |
Countries
Canada, United Kingdom, United States
Contacts
Public ContactMark De Rosch, PhD
Vertex Pharmaceuticals Incorporated
Outcome results
None listed