Phase 3
Completed N=175
A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del
Source: ClinicalTrials.gov NCT04105972 ↗Enrolled (actual)
175
Serious AEs
10.9%
Results posted
Aug 2021
Primary outcomePrimary: Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score — 1.2; 17.1 units on a scale — p=< 0.0001
◆ Published Evidence
Highly cited
140citations · ~35 / year
Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial.
Summary
This study will evaluate the efficacy, safety, 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 homozygous for F508del.
Linked Publications (3)
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Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial.
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score |
1.2; 17.1 | < 0.0001 sig |
| SECONDARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
1.0; 11.2 | <0.0001 sig |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) |
-3.4; -46.2 | — |
| SECONDARY Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
81; 77; 14; 5 | — |
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 (NCT04105972) 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.