Phase 3
N=120
A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT04545515 ↗Enrolled (actual)
120
Serious AEs
10.8%
Results posted
May 2024
Primary outcome: Primary: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 118; 13 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- ELX/TEZ/IVA (Drug); IVA (Drug)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- All
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Primary completion
- Mar 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
118; 13 | — |
| SECONDARY Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) |
-57.3; -57.5 | — |
| SECONDARY Absolute Change From Parent Study Baseline in Lung Clearance Index 2.5 (LCI2.5) |
-1.74; -2.35 | — |
Summary
The study evaluates the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in participants with CF who are 6 years of age and older with F/MF genotypes.
Eligibility Criteria
Key Inclusion Criteria
- Completed study drug treatment in parent study (VX19-445-116, NCT04353817), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the treatment period in the parent study
Key Exclusion Criteria
- History of study drug intolerance in the parent study
Other protocol defined Inclusion/Exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT04545515). 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.