Phase 3
N=251
Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT04058366 ↗Enrolled (actual)
251
Serious AEs
12.2%
Results posted
Jan 2024
Primary outcome: Primary: Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 241; 38 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- ELX/TEZ/IVA (Drug); IVA (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Primary completion
- Dec 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
241; 38 | — |
| SECONDARY Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
4.1; 3.7 | — |
| SECONDARY Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) |
-23.0; -22.6 | — |
| SECONDARY Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI) |
1.15; 0.83 | — |
| SECONDARY Part A: Absolute Change From Parent Study Baseline in BMI Z-score |
0.11; 0.40 | — |
| SECONDARY Part A: Absolute Change From Parent Study Baseline in Body Weight |
3.6; 2.9 | — |
| SECONDARY Part A: Absolute Change From Parent Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score |
7.2; 8.1 | — |
Summary
This study evaluated the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).
Eligibility Criteria
Key Inclusion Criteria
- Completed study drug treatment in parent study (VX18-445-104); 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 (NCT04058366). 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.