Evaluation of Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older

Study Overview
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Brief Summary
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This study will evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF).

Official Title
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A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: ELX/TEZ/IVA
* Drug: IVA


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Triple Combination Arm<br>Subjects will receive ELX/TEZ/IVA TC in the morning and IVA as mono tablet in the evening. | Drug: ELX/TEZ/IVA<br>* Fixed-dose combination (FDC) tablet for oral administration<br>* Other names: elexacaftor/tezacaftor/ivacaftor;Drug: IVA<br>* Mono tablet for oral administration.<br>* Other names: ivacaftor;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability as assessed by adverse events (AEs) and serious adverse events (SAEs) |  | From Baseline up to Week 196 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) |  | From Baseline up to Week 192 | 
| Absolute change in sweat chloride (SwCl) |  | From Baseline up to Week 192 | 
| Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score |  | From Baseline up to Week 192 | 
| Absolute change in body mass index (BMI) |  | From Baseline up to Week 192 | 
| Absolute change in BMI-for-age z-score |  | From Baseline up to Week 192 | 
| Number of pulmonary exacerbations (PEx) |  | From Baseline up to Week 192 | 
| Number of CF-related hospitalizations |  | From Baseline up to Week 192 | 
| Absolute change in lung clearance index 2.5 (LCI 2.5) |  | From Baseline up to Week 192 | 
| Absolute change in weight |  | From Baseline up to Week 192 | 
| Absolute change in weight-for-age z-score |  | From Baseline up to Week 192 | 
| Absolute change in height |  | From Baseline up to Week 192 | 
| Absolute change in height-for-age z-score |  | From Baseline up to Week 192 |