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 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 40% to 90% (inclusive) - 12 years of age or older - Females of child-bearing potential must have a negative serum pregnancy test at Screening - Subjects of child-bearing potential and who are sexually active must follow contraception requirements Are the trial subjects under 18? yes Number of subjects for this age range: 36 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 125 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: - History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject - An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks before Day 1 (first dose of study drug) - Abnormal liver function - Abnormal renal function - History of solid organ or hematological transplantation - History of alcohol, medication or illicit drug abuse within one year prior to Day 1 (first dose of study drug) - Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 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 undergone a wash-out period of 4 weeks prior to Day 1 [first dose of study drug]) - Concomitant use of any inhibitors or inducers of CYP 3A4
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of VX-770 after 24 weeks of treatment in subjects with cystic fibrosis (CF) who have the G551D cystic fibrosis transmembrane conductance regulator (CFTR) mutation on at least 1 allele;Secondary Objective: To evaluate the safety of VX-770 after 24 and 48 weeks of treatment in subjects with CF who have the G551D CFTR mutation on at least 1 allele To evaluate the efficacy of VX-770 after 48 weeks of treatment in subjects with CF who have the G551D CFTR mutation on at least 1 allele ;Primary end point(s): Absolute change from baseline in percent predicted forced expiratory volume in 1 second (%predicted FEV1) through Week 24;Timepoint(s) of evaluation of this end point: Week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy as determined by: • Absolute change from baseline in percent predicted FEV1 through Week 48 • Change from baseline in sweat chloride through Weeks 24 and 48 • Change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) through Weeks 24 and 48 • Rate of change in weight through Weeks 24 and 48 • Time to first pulmonary exacerbation through Weeks 24 and 48 Safety as determined by: Adverse events, clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), ambulatory ECGs, vital signs, and physical examinations;Timepoint(s) of evaluation of this end point: Week 24 and Week 48 | — |
Countries
Australia, Canada, Czech Republic, France, Germany, Ireland, United Kingdom, United States
Contacts
Public ContactMark De Rosch, PhD
Vertex Pharmaceuticals Incorporated
Outcome results
None listed