None listed
Conditions
Brief summary
Examine the effect of twice daily administration of 150mg ivacaftor on exocrine pancreatic function of patients with cystic fibrosis and at least one copy of the G551D CFTR mutation
Interventions
Kalydeco (ivacaftor) 150mg administered orally twice daily for 34 weeks. Tablet counts will be used to assess adherence to study medicine dosage.
Sponsors
Sydney Children's Hospitals Network
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
6 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
Participants 6 years and older with cystic fibrosis and at least one copy of the G551D CFTR mutation, requiring pancreatic enzyme replacement therapy and who are naive for ivacaftor
Exclusion criteria
1. known hypersensitivity to ivacaftor, its metabolites, or formulation excipients 2. galactose intolerance, Lapp lactase deficiency or glucose-galactose malabsorption 3. have undergone organ transplantation 4.Use of any inhibitors (itraconazole, posaconazole, voriconazole, ketoconazole, telithromycin, clarithromycin) or inducers of (rifampicin, rifabutin, phenobarbital, carbamazepine, phenytoin, St Johns Wort) CYP450 3A, within 14 days prior to the first dose of ivacaftor or during the study period 5. hepatic insufficiency defined as Child-Pugh Class C impairment (a score of 10) 6. severe renal impairment, as defined by creatinine clearance less than or equal to 30mL/min or end stage renal disease 7. Pregnant, planning a pregnancy during the course of the study , or breast-feeding
Outcome results
None listed