Cystic Fibrosis
Conditions
Brief summary
This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF).
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
* Currently participating in study VX17-659-105 (NCT03447262)
Exclusion criteria
* History of drug intolerance in study VX17-659-105 that would pose an additional risk to the participant in the opinion of the investigator * Current participation in an investigational drug trial (other than study VX17-659-105) 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 Baseline through Week 100 |
Countries
Australia, Canada, Denmark, Germany, Ireland, Israel, Poland, Spain, Switzerland, United Kingdom, United States
Participant flow
Recruitment details
The study was conducted as a single part study (Part A) in countries including United States (US) with commercially-available ELX/TEZ/IVA. The regional protocol for countries without commercially-available ELX/TEZ/IVA was amended so that participants in these countries had the opportunity to participate for up to an additional 48 weeks in Part B.
Pre-assignment details
A total 458 participants were enrolled from the parent study VX17-659-105 (NCT03447262). One participant was enrolled but did not receive any dose in this study.
Participants by arm
| Arm | Count |
|---|---|
| ELX/TEZ/IVA Part A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks.
Part B: Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. | 457 |
| Total | 457 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 2 |
| Overall Study | Commercial drug is available for participants | 13 |
| Overall Study | Lost to Follow-up | 6 |
| Overall Study | Never Dosed | 1 |
| Overall Study | Other | 12 |
| Overall Study | Other non-compliance | 2 |
| Overall Study | Physician Decision | 2 |
| Overall Study | Withdrawal of Consent (not due to AE) | 8 |
Baseline characteristics
| Characteristic | ELX/TEZ/IVA |
|---|---|
| Age, Continuous | 28.5 years STANDARD_DEVIATION 9.8 |
| Race/Ethnicity, Customized American Indian or Alaska Native | 0 Participants |
| Race/Ethnicity, Customized Asian | 0 Participants |
| Race/Ethnicity, Customized Black or African American | 3 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 14 participants |
| Race/Ethnicity, Customized Multiple | 4 Participants |
| Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race/Ethnicity, Customized Not collected per local regulations | 5 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 437 participants |
| Race/Ethnicity, Customized White | 445 Participants |
| Sex: Female, Male Female | 202 Participants |
| Sex: Female, Male Male | 255 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 457 | 0 / 66 |
| other Total, other adverse events | 404 / 457 | 45 / 66 |
| serious Total, serious adverse events | 75 / 457 | 4 / 66 |
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 Baseline through Week 100
Population: Safety set for included all participants who received at least 1 dose of study drug.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| 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 | 435 participants |
| 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 | 75 participants |