Phase 3
Completed N=67
A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
Source: ClinicalTrials.gov NCT03559062 ↗Enrolled (actual)
67
Serious AEs
0.0%
Results posted
Feb 2020
Primary outcomePrimary: Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8 — -0.51 lung clearance index — p=<0.0001
◆ 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 will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).
Linked Publications (2)
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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 Lung Clearance Index 2.5 (LCI2.5) Through Week 8 |
-0.51 | <0.0001 sig |
| SECONDARY Absolute Change in Sweat Chloride At Week 8 |
-12.3 | <0.0001 sig |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8 |
2.3 | 0.0546 |
| SECONDARY Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit |
8; 41; 2; 0; 0; 0 | — |
Eligibility Criteria
Key Inclusion Criteria
- Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
- Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
- Participants with a screening LCI2.5 result ≥7.5.
- Participants who are able to swallow tablets.
Key Exclusion Criteria
- Clinically significant cirrhosis with or without portal hypertension.
- Colonization 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 (NCT03559062) 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.