Cystic Fibrosis
Conditions
Brief summary
This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator (CFTR) gene mutation.
Interventions
DRUGELX/TEZ/IVA
Fixed-dose combination (FDC) tablets for oral administration.
DRUGIVA
Tablets 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
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: Part A: Completed study drug treatment in parent study 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 Part B: Completed study drug treatment in Part A or had study drug interruption(s) in Part A but completed study visits up to the last scheduled visit of the treatment period of Part A Key
Exclusion criteria
History of study drug intolerance in the parent study Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 196 |
Secondary
| Measure | Time frame |
|---|---|
| Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1) | From Baseline up to Week 96 |
| Part A: Absolute Change in Sweat Chloride (SwCl) | From Baseline up to Week 96 |
| Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score | From Baseline up to Week 96 |
| Part A: Absolute Change in Body Mass Index (BMI) | From Baseline up to Week 96 |
| Part A: Absolute Change in Weight | From Baseline up to Week 96 |
| Part A: Number of Pulmonary Exacerbations (PEx) | From Baseline up to Week 96 |
Countries
Austria, Belgium, Canada, Czechia, France, Germany, Hungary, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, Switzerland
Outcome results
None listed