cystic fibrosis MedDRA version: 16.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Kalydeco
Product Name: ivacaftor
Product Code: VX-770
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IVACAFTOR
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Concentrat
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male or female with confirmed diagnosis of CF - Must have at least 1 allele of the R117H CFTR mutation - FEV1 40% to 90% (for subjects aged 12 years or older) or 40% to 105% (for subjects aged 6 to 11 years) predicted normal for age, sex, and height - 6 years of age or older - Minimum weight of 15 kg at screening - Females of childbearing potential must not be pregnant - Willing to comply with contraception requirements Are the trial subjects under 18? yes Number of subjects for this age range: 9 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 4 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Any 1 of the following CFTR gating mutations: G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D - History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the subject - An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before the first dose of study drug - History of solid organ or hematological transplantation - History of alcohol, medication or illicit drug abuse within 1 year before the first dose of study drug - Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days before screening - Use of any inhibitors or inducers of cytochrome P450 (CYP) 3A - Evidence of cataract or lens opacity at screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of ivacaftor in subjects with cystic fibrosis (CF) who have the R117H-CF transmembrane conductance regulator (CFTR) mutation;Secondary Objective: To evaluate the safety of ivacaftor in subjects with cystic fibrosis (CF) who have the R117H-CF transmembrane conductance regulator (CFTR) mutation;Primary end point(s): Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1);Timepoint(s) of evaluation of this end point: Through Week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Change from baseline in body mass index (BMI) - Change from baseline in sweat chloride - Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) - Time to first pulmonary exacerbation - Safety, as determined by adverse events, clinical laboratory values (serum chemistry, hematology, and coagulation), electrocardiograms (ECGs), ophthalmologic examinations, and vital signs;Timepoint(s) of evaluation of this end point: Through Week 24 | — |
Countries
United Kingdom, United States
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed