Phase 3
Completed N=130
A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation
Source: ClinicalTrials.gov NCT03537651 ↗Enrolled (actual)
130
Serious AEs
20.3%
Results posted
Nov 2021
Primary outcomePrimary: Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 129; 31 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
This study evaluates the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del mutation.
Linked Publications (3)
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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).
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A Phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children ≥ 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
129; 31 | — |
| SECONDARY Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group) |
-0.95 | — |
| SECONDARY Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS |
-2.04 | — |
| SECONDARY Part A: Absolute Change in Sweat Chloride (SwCl) for 115/116 FAS (TEZ/IVA Group) |
-13.8 | — |
| SECONDARY Part A: Absolute Change in SwCl for 113B/116 FAS |
-16.2 | — |
| SECONDARY Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group) |
6.4 | — |
| SECONDARY Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS |
6.0 | — |
| SECONDARY Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group) |
1.25 | — |
| SECONDARY Part A: Absolute Change in BMI for 113B/116 FAS |
1.19 | — |
| SECONDARY Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
53; 8 | — |
Eligibility Criteria
Inclusion Criteria
- Completed the Week 24 Visit in Study 113 Part B or the Week 8 Visit in Study 115
- Eligible CFTR Mutation
Exclusion Criteria
- Pregnant and nursing females
- History of poor compliance with study drug and/or procedures in a previous study as deemed by the investigator
- Ongoing participation in another study with investigational drug
Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT03537651) 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.