Cystic Fibrosis
Conditions
Brief summary
The purpose of this study is to evaluate the safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in participants with CF who are 12 years of age or older.
Interventions
DRUGELX/TEZ/IVA
Fixed-dose combination 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
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Completed study drug treatment in parent study (VX19-445-117 \[NCT04599465\] or VX20-445-126 \[NCT04969224\]), 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 drug intolerance in a parent study Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 36 |
Countries
Australia, Belgium, Canada, Czechia, Spain
Participant flow
Pre-assignment details
Participants from Parent Studies VX19-445-117 (NCT04599465) and VX20-445-126 (NCT04969224) were enrolled in this study. A total of 86 participants were enrolled in this study.
Participants by arm
| Arm | Count |
|---|---|
| ELX/TEZ/IVA Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 36 weeks. | 86 |
| Total | 86 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Commercial drug is available for participant | 76 |
| Overall Study | Participant enrolled in another qualified Vertex study | 10 |
Baseline characteristics
| Characteristic | ELX/TEZ/IVA |
|---|---|
| Age, Continuous | 25.3 years STANDARD_DEVIATION 9.9 |
| Race/Ethnicity, Customized American Indian or Alaska Native | 0 Participants |
| Race/Ethnicity, Customized Asian | 0 Participants |
| Race/Ethnicity, Customized Black or African American | 0 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 11 Participants |
| Race/Ethnicity, Customized Native Hawaiian or other Pacific Islander | 0 Participants |
| Race/Ethnicity, Customized Not collected per local regulations | 7 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 68 Participants |
| Race/Ethnicity, Customized Other | 0 Participants |
| Race/Ethnicity, Customized White | 79 Participants |
| Sex: Female, Male Female | 37 Participants |
| Sex: Female, Male Male | 49 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 86 |
| other Total, other adverse events | 33 / 86 |
| serious Total, serious adverse events | 4 / 86 |
Outcome results
Primary
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 36
Population: Safety set included for all participants who received at least 1 dose of study drug.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| ELX/TEZ/IVA | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Participants with TEAEs | 61 Participants |
| ELX/TEZ/IVA | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Participants with SAEs | 4 Participants |