Phase 3
Completed N=307
Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation
Source: ClinicalTrials.gov NCT05274269 ↗Enrolled (actual)
307
Serious AEs
10.8%
Results posted
Aug 2024
Primary outcomePrimary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) — -0.4; 8.9 percent predicted FEV1 — p=< 0.0001
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
This study evaluated the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
-0.4; 8.9 | < 0.0001 sig |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) |
0.5; -27.8 | < 0.0001 sig |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score |
-2.0; 17.5 | < 0.0001 sig |
| SECONDARY Absolute Change in Body Mass Index (BMI) |
0.35; 0.81 | < 0.0001 sig |
| SECONDARY Absolute Change in Weight |
1.2; 2.4 | < 0.0001 sig |
| SECONDARY Number of Pulmonary Exacerbations (PEx) |
40; 21 | — |
| SECONDARY Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
97; 193; 15; 18 | — |
Eligibility Criteria
Key Inclusion Criteria
- Participant has a qualifying ELX/TEZ/IVA-responsive CFTR mutation and does not have an exclusionary CFTR mutation
- Forced expiratory volume in 1 second (FEV1) value >=40% and <=100% of predicted mean for age, sex, and height
Key Exclusion Criteria
- History of solid organ or hematological transplantation
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT05274269). 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.