Phase 3
Completed N=2
An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations
Source: ClinicalTrials.gov NCT02934698 ↗Enrolled (actual)
2
Serious AEs
0.0%
Results posted
Aug 2018
Primary outcomePrimary: Forced Expiratory Volume — 4 percentage of predicted
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Forced Expiratory Volume |
4 | — |
| SECONDARY Sputum Results |
2 | — |
| SECONDARY Sweat Chloride |
-5.7 | — |
Eligibility Criteria
Inclusion Criteria
- Subjects are >18 years of age and able to provide informed consent.
- Subjects reside in the US and are willing to be treated with ivacaftor.
- Subjects have the splicing mutation of interest.
- Subjects are willing and able to perform requirements of the study.
Exclusion Criteria
- There are no relevant exclusion criteria for this n-of-2 study.
Data sourced from ClinicalTrials.gov (NCT02934698). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.