Phase 3
Completed N=66
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older
Source: ClinicalTrials.gov NCT04183790 ↗Enrolled (actual)
66
Serious AEs
10.9%
Results posted
May 2025
Primary outcomePrimary: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 64; 7 Participants
◆ Published Evidence
Established
71citations · ~12 / year
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
Summary
The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
Linked Publications (4)
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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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Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged ⩾6 Years with Cystic Fibrosis and at Least One <i>F508del</i> Allele: A Phase 3, Open-Label Clinical 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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Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children ≥6 Years with Cystic Fibrosis and at Least One <i>F508del</i> Allele: A 192-Week, Phase 3, Open-Label Extension Study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
64; 7 | — |
| SECONDARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
9.6 | — |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) |
-57.9 | — |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score |
10.0 | — |
| SECONDARY Absolute Change in Body Mass Index (BMI) |
3.60 | — |
| SECONDARY Absolute Change in BMI-for-age Z-score |
0.39 | — |
| SECONDARY Number of Participants With Pulmonary Exacerbations (PEx) for 106/107 |
9 | — |
| SECONDARY Number of CF-related Hospitalizations for 106/107 |
5 | — |
| SECONDARY Absolute Change in Lung Clearance Index 2.5 (LCI 2.5) |
-2.33 | — |
| SECONDARY Absolute Change in Weight |
19.9 | — |
| SECONDARY Absolute Change in Weight-for-age Z-score |
0.38 | — |
| SECONDARY Absolute Change in Height |
23.1 | — |
| SECONDARY Absolute Change in Height-for-age Z-score |
0.04 | — |
Eligibility Criteria
Key Inclusion Criteria
- Completed study drug treatment in parent study (VX18-445-106 Part B, NCT03691779), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study
Key Exclusion Criteria
- History of study drug intolerance in parent study
Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT04183790) 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.