At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 2 enrolled
Drug / intervention
Ivacaftordrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations
In Brief
A Phase 3 clinical trial evaluating Ivacaftor for Cystic Fibrosis. Completed, enrolled 2 participants.
Detailed Summary
This postmarketing N of 2 study is designed to evaluate the efficacy and safety of open-label ivacaftor treatment in two sisters with cystic fibrosis and pancreatic sufficiency.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsCystic Fibrosis
Countries--
CollaboratorsVertex Pharmaceuticals Incorporated
Timeline
Phase 3CompletedFinished
2017201820192020202120222023202420252026
First PostedOct 2016
Enrollment StartNov 2016
Primary CompletionMay 2018
TodayJul 2026
First PostedOct 17, 2016
Enrollment StartNov 1, 2016
Primary CompletionMay 1, 2018
TodayJul 2, 2026
Enrollment to primary: 1.5 yearsPosted 9.7 years ago
Interventions
Ivacaftordrug
Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.