Phase 3
N=510
A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT03525574 ↗Enrolled (actual)
510
Serious AEs
33.9%
Results posted
May 2024
Primary outcome: Primary: Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 504; 175 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
- Jan 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
504; 175 | — |
| SECONDARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set |
15.3; 13.8 | — |
| SECONDARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set |
10.9; 10.7 | — |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set |
-47.0; -45.3 | — |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set |
-48.2; -48.2 | — |
| SECONDARY Number of Pulmonary Exacerbations (PEx) for 102/105 Efficacy Set |
174 | — |
| SECONDARY Number of Pulmonary Exacerbations (PEx) for 103/105 Efficacy Set |
43 | — |
| SECONDARY Time to First PEx for 102/105 Efficacy Set |
NA | — |
| SECONDARY Time to First PEx for 103/105 Efficacy Set |
NA | — |
| SECONDARY Absolute Change in Body Mass Index (BMI) for 102/105 Efficacy Set |
1.81; 1.74 | — |
| SECONDARY Absolute Change in Body Mass Index (BMI) for 103/105 Efficacy Set |
1.72; 1.85 | — |
| SECONDARY Absolute Change in BMI Z-score for 102/105 Efficacy Set |
0.24; 0.18 | — |
| SECONDARY Absolute Change in BMI Z-score for 103/105 Efficacy Set |
0.36; 0.24 | — |
| SECONDARY Absolute Change in Body Weight for 102/105 Efficacy Set |
6.6; 6.0 | — |
| SECONDARY Absolute Change in Body Weight for 103/105 Efficacy Set |
6.1; 6.3 | — |
| SECONDARY Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 102/105 Efficacy Set |
15.3; 18.3 | — |
| SECONDARY Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/105 Efficacy Set |
14.8; 17.6 | — |
Summary
The study evaluates the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation
Eligibility Criteria
Inclusion Criteria
- Completed study drug treatment in a parent study i.e.VX17-445-102 (NCT03525444) and VX17-445-103 (NCT03525548); or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study.
Exclusion Criteria
- History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
- Current participation in an investigational drug trial (other than a parent study)
Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT03525574). 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.