Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).

Official Title
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A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year of Age and Older

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: VNZ/TEZ/D-IVA


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222) Key Exclusion Criteria: Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug History of solid organ, hematological transplantation, or cancer History of drug intolerance in the parent study Other protocol defined Inclusion/Exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 1 Year

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: VNZ/TEZ/D-IVA<br>Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study. | Drug: VNZ/TEZ/D-IVA<br>* Fixed-dose combination tablets for oral administration.<br>* Other names: vanzacaftor/tezacaftor/deutivacaftor;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) |  | From Day 1 up to Week 100 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| All Cohorts: Absolute Change in Sweat Chloride (SwCl) |  | From Baseline Through Week 96 | 
| Cohort 1: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) |  | From Baseline Through Week 100 | 
| All Cohorts: Number of Pulmonary Exacerbation (PEx) Related Hospitalizations |  | From Baseline Through Week 100 | 
| All Cohorts: Number of CF- Related Hospitalizations |  | From Baseline Through Week 100 | 
| Cohort 1: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score |  | From Baseline Through Week 100 | 
| Cohorts 1 and 2: Absolute Change in Body Mass Index (BMI) |  | From Baseline Through Week 100 | 
| Cohorts 1 and 2: Absolute Change in BMI-for-age Z-score |  | From Baseline Through Week 100 | 
| Cohort 3: Absolute Change in Weight-for-length |  | From Baseline Through Week 100 | 
| Cohort 3: Absolute Change in Weight-for-length Z-score |  | From Baseline Through Week 100 | 
| All Cohorts: Absolute Change in Weight |  | From Baseline Through Week 100 | 
| All Cohorts: Change in Weight-for-age Z-score |  | From Baseline Through Week 100 | 
| Cohorts 1 and 2: Absolute Change in Height |  | From Baseline Through Week 100 | 
| Cohorts 1 and 2: Absolute Change in Height-for-age Z-score |  | From Baseline Through Week 100 | 
| Cohort 3: Absolute Change in Length |  | From Baseline Through Week 100 | 
| Cohort 3: Absolute Change in Length-for-age Z-score |  | From Baseline Through Week 100 |