Phase 3
Completed N=107
A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
Source: ClinicalTrials.gov NCT03525548 ↗Enrolled (actual)
107
Serious AEs
2.8%
Results posted
Jan 2020
Primary outcomePrimary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) — 0.4; 10.4 percentage points — p=<0.0001
◆ Published Evidence
Highly cited
1,357citations · ~194 / year
Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial.
Summary
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F)
Linked Publications
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Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
0.4; 10.4 | <0.0001 sig |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) |
1.7; -43.4 | <0.0001 sig |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score |
-1.4; 16.0 | <0.0001 sig |
| SECONDARY Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
33; 32; 1; 2 | — |
| SECONDARY Observed Pre-Dose Concentration (Ctrough) of VX-445, TEZ, TEZ Metabolite (M1-TEZ), and IVA |
NA; 4.84; 1.71; 1.60; 1.48; 1.89 | — |
Eligibility Criteria
Key Inclusion Criteria
- Homozygous for the F508del mutation (F/F)
- Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height
Key Exclusion Criteria
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Solid organ or hematological transplantation
Other protocol defined Inclusion/Exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT03525548) and the linked publication. 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.