Phase 3
N=66
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT04183790 ↗Enrolled (actual)
66
Serious AEs
10.9%
Results posted
May 2025
Primary outcome: Primary: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 64; 7 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
- Feb 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
64; 7 | — |
| SECONDARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
9.6 | — |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) |
-57.9 | — |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score |
10.0 | — |
| SECONDARY Absolute Change in Body Mass Index (BMI) |
3.60 | — |
| SECONDARY Absolute Change in BMI-for-age Z-score |
0.39 | — |
| SECONDARY Number of Participants With Pulmonary Exacerbations (PEx) for 106/107 |
9 | — |
| SECONDARY Number of CF-related Hospitalizations for 106/107 |
5 | — |
| SECONDARY Absolute Change in Lung Clearance Index 2.5 (LCI 2.5) |
-2.33 | — |
| SECONDARY Absolute Change in Weight |
19.9 | — |
| SECONDARY Absolute Change in Weight-for-age Z-score |
0.38 | — |
| SECONDARY Absolute Change in Height |
23.1 | — |
| SECONDARY Absolute Change in Height-for-age Z-score |
0.04 | — |
Summary
The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
Eligibility Criteria
Key Inclusion Criteria
- Completed study drug treatment in parent study (VX18-445-106 Part B, NCT03691779), 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 parent study
Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT04183790). 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.