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 105% (inclusive) - 6 to 11 years of age (inclusive) - Weight 15 kg without shoes at Screening - 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 meet the contraception requirements Are the trial subjects under 18? yes Number of subjects for this age range: 52 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - History of any illness or condition that, in the opinion of the investigator 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 6 to 11 years of age with CF who have the G551D 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 6 to 11 years of age 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 6 to 11 years of age with CF who have the G551D CFTR mutation on at least 1 allele ;Timepoint(s) of evaluation of this end point: PART B: Week 24;Primary end point(s): Absolute change from baseline in percent predicted forced expiratory volume in 1 second (%predicted FEV1) through Week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Part A: Pharmacokinetic (PK) parameters of VX-770 (and metabolites M1 and M6, if possible) Part B 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 PART A AND PART B: 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, France, Germany, Ireland, United Kingdom, United States
Contacts
Public ContactMark De Rosch, PhD
Vertex Pharmaceuticals Incorporated
Outcome results
None listed