Phase 3
Completed N=258
A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
Source: ClinicalTrials.gov NCT04058353 ↗Enrolled (actual)
258
Serious AEs
6.2%
Results posted
Jul 2021
Primary outcomePrimary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for ELX/TEZ/IVA Group — 3.7 percentage points
◆ Published Evidence
Highly cited
274citations · ~55 / year
Triple Therapy for Cystic Fibrosis <i>Phe508del</i>-Gating and -Residual Function Genotypes.
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 heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes).
Linked Publications
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Triple Therapy for Cystic Fibrosis <i>Phe508del</i>-Gating and -Residual Function Genotypes.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for ELX/TEZ/IVA Group |
3.7 | — |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) for ELX/TEZ/IVA Group |
-22.3 | — |
| SECONDARY Absolute Change in ppFEV1 for the ELX/TEZ/IVA Group Compared to the Control Group |
0.2; 3.7 | <0.0001 sig |
| SECONDARY Absolute Change in SwCl for ELX/TEZ/IVA Group Compared to the Control Group |
0.7; -22.3 | <0.0001 sig |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for ELX/TEZ/IVA Group |
10.3 | — |
| SECONDARY Absolute Change in CFQ-R Respiratory Domain Score for ELX/TEZ/IVA Group Compared to the Control Group |
1.6; 10.3 | <0.0001 sig |
| SECONDARY Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
83; 88; 11; 5 | — |
Eligibility Criteria
Key Inclusion Criteria
- Subject has a confirmed diagnosis of CF and is heterozygous for F508del and either a gating or residual function mutation (F/G and F/RF genotypes)
- 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 (NCT04058353) 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.