Cystic Fibrosis
Conditions
Brief summary
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).
Interventions
DRUGVNZ/TEZ/D-IVA
Fixed-dose combination tablets or granules for oral administration.
Sponsors
Vertex Pharmaceuticals Incorporated
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: Parts A and B: • Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222) Part B: -Meets at least 1 of the following criteria: * Completed study drug treatment in Part A * Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A 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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) | From Baseline up to Week 100 |
| Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) | From Baseline up to Week 196 |
Secondary
| Measure | Time frame |
|---|---|
| Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl) | From Baseline Through Week 96 |
| Part B: Absolute Change in Sweat Chloride (SwCl) | From Baseline Through Week 192 |
| Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) | From Baseline Through Week 100 |
| Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) | From Baseline Through Week 196 |
| Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx) | From Baseline Through Week 100 |
| Part B: Number of Pulmonary Exacerbation (PEx) | From Baseline Through Week 196 |
| Part A (All Cohorts): Number of CF- Related Hospitalizations | From Baseline Through Week 100 |
| Part B: Number of CF- Related Hospitalizations | From Baseline Through Week 196 |
| Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score | From Baseline Through Week 100 |
| Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score | From Baseline Through Week 196 |
| Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI) | From Baseline Through Week 100 |
| Part B: Absolute Change in Body Mass Index (BMI) | From Baseline Through Week 196 |
| Part A (Cohorts 1 and 2): Absolute Change in BMI-for-age Z-score | From Baseline Through Week 100 |
| Part B: Absolute Change in BMI-for-age Z-score | From Baseline Through Week 196 |
| Part A (Cohort 3): Absolute Change in Weight-for-length | From Baseline Through Week 100 |
| Part B: Absolute Change in Weight-for-length | From Baseline Through Week 196 |
| Part A (Cohort 3): Absolute Change in Weight-for-length Z-score | From Baseline Through Week 100 |
| Part B: Absolute Change in Weight-for-length Z-score | From Baseline Through Week 196 |
| Part A (All Cohorts): Absolute Change in Weight | From Baseline Through Week 100 |
| Part B: Absolute Change in Weight | From Baseline Through Week 196 |
| Part A (All Cohorts): Change in Weight-for-age Z-score | From Baseline Through Week 100 |
| Part B: Change in Weight-for-age Z-score | From Baseline Through Week 196 |
| Part A (Cohorts 1 and 2): Absolute Change in Height | From Baseline Through Week 100 |
| Part B: Absolute Change in Height | From Baseline Through Week 196 |
| Part A (Cohorts 1 and 2): Absolute Change in Height-for-age Z-score | From Baseline Through Week 100 |
| Part B: Absolute Change in Height-for-age Z-score | From Baseline Through Week 196 |
| Part A (Cohort 3): Absolute Change in Length | From Baseline Through Week 100 |
| Part B: Absolute Change in Length | From Baseline Through Week 196 |
| Part A (Cohort 3): Absolute Change in Length-for-age Z-score | From Baseline Through Week 100 |
| Part B: Absolute Change in Length-for-age Z-score | From Baseline Through Week 196 |
Countries
Australia, Canada, France, Germany, Netherlands, New Zealand, Sweden, Switzerland, United Kingdom, United States
Outcome results
None listed