Cystic Fibrosis
Conditions
Brief summary
This study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.
Interventions
DRUGELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration.
DRUGIVA
Tablet 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 (VX18-445-109); 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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 52 |
| Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 86 |
Countries
Australia, Belgium, Germany, United Kingdom
Participant flow
Pre-assignment details
The study was conducted in 2 parts, Part A and Part B. Participants from Part A also participated in Part B. The Participant flow was planned to be presented for the overall treatment arm.
Participants by arm
| Arm | Count |
|---|---|
| ELX/TEZ/IVA Part A: Participants received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks. | 172 |
| Total | 172 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Part A (48 Weeks) | Adverse Event | 2 |
| Part A (48 Weeks) | Commercial drug is available for participant | 2 |
| Part A (48 Weeks) | Other | 5 |
| Part A (48 Weeks) | Withdrawal of consent (not due to AE) | 4 |
| Part B (86 Weeks) | Commercial drug is available for participant | 46 |
| Part B (86 Weeks) | Other | 4 |
Baseline characteristics
| Characteristic | ELX/TEZ/IVA |
|---|---|
| Age, Continuous Part A | 27.9 years STANDARD_DEVIATION 11.4 |
| Age, Continuous Part B | 25.6 years STANDARD_DEVIATION 12 |
| Race/Ethnicity, Customized Part A American Indian or Alaska Native | 0 Participants |
| Race/Ethnicity, Customized Part A Asian | 2 Participants |
| Race/Ethnicity, Customized Part A Black or African American | 0 Participants |
| Race/Ethnicity, Customized Part A Hispanic or Latino | 3 Participants |
| Race/Ethnicity, Customized Part A Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race/Ethnicity, Customized Part A Not collected per local regulations | 2 Participants |
| Race/Ethnicity, Customized Part A Not Hispanic or Latino | 167 Participants |
| Race/Ethnicity, Customized Part A White | 169 Participants |
| Race/Ethnicity, Customized Part A White, Asian | 1 Participants |
| Race/Ethnicity, Customized Part B American Indian or Alaska Native | 0 Participants |
| Race/Ethnicity, Customized Part B Asian | 0 Participants |
| Race/Ethnicity, Customized Part B Black or African American | 0 Participants |
| Race/Ethnicity, Customized Part B Hispanic or Latino | 1 Participants |
| Race/Ethnicity, Customized Part B Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race/Ethnicity, Customized Part B Not collected per local regulations | 0 Participants |
| Race/Ethnicity, Customized Part B Not Hispanic or Latino | 49 Participants |
| Race/Ethnicity, Customized Part B White | 50 Participants |
| Race/Ethnicity, Customized Part B White, Asian | 0 Participants |
| Sex: Female, Male Part A Female | 87 Participants |
| Sex: Female, Male Part A Male | 85 Participants |
| Sex: Female, Male Part B Female | 26 Participants |
| Sex: Female, Male Part B Male | 24 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 172 | 0 / 50 |
| other Total, other adverse events | 127 / 172 | 47 / 50 |
| serious Total, serious adverse events | 26 / 172 | 8 / 50 |
Outcome results
Primary
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 up to Week 52
Population: Safety set included all participants who received at least 1 dose of study drug.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part A: ELX/TEZ/IVA | Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Participants with TEAEs | 160 Participants |
| Part A: ELX/TEZ/IVA | Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Participants with SAEs | 26 Participants |
Primary
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 up to Week 86
Population: Safety Set included all participants who received at least 1 dose of study drug.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part A: ELX/TEZ/IVA | Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Participants with TEAEs | 50 Participants |
| Part A: ELX/TEZ/IVA | Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Participants with SAEs | 8 Participants |