Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
Allocation: NON_RANDOMIZED | Intervention model: PARALLEL
Enrollment: 33
Healthy volunteers: no

Brief summary:
The purpose of this study is to provide information regarding the long-term safety and pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of age with Cystic Fibrosis (CF) who have a CFTR gating mutation in at least 1 allele and will further explore the efficacy of long-term ivacaftor treatment in this population of patients with CF.

Detailed description:

Arms:
arm 1: None arm 2: Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the Week 84 Visit. The ivacaftor dose will be:

* 50 mg q12h for subjects 2 to \<6 years of age and \<14 kg,
* 75 mg q12h for subjects 2 to \<6 years of age and ≥ 14 kg, or
* 150 mg q12h for subjects ≥ 6 years of age.

Interventions: intervention 1: Ivacaftor
Intervention descriptions: intervention 1: None

Protocol document: