Cystic Fibrosis
Conditions
Brief summary
This study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.
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: * Forced expiratory volume in 1 second (FEV1) value ≥30% and ≤90% predicted * Heterozygous for CF transmembrane conductance regulator gene (CFTR) F508del mutation and a minimal function mutation (F/MF genotypes) Key
Exclusion criteria
* Clinically significant liver cirrhosis * Solid organ or hematological transplantation * Non-ambulatory status * Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12 | Baseline, Week 8 through Week 12 | Percent reduction in cough frequency was analyzed with a mixed effects model for repeated measures (MMRM), with change from baseline at each post-baseline visit on the natural log scale as the dependent variable. The percent reduction was estimated as 100% × (1-exponential form of LS mean change estimate from the MMRM). |
Secondary
| Measure | Time frame |
|---|---|
| Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12 | Baseline, Week 8 through Week 12 |
Countries
Australia, Belgium, Canada, Spain
Participant flow
Pre-assignment details
This study was conducted in participants with cystic fibrosis, aged 12 years and older, who are heterozygous for the F508del mutation and the minimal function mutation (F/MF) genotypes.
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 approximately 13 weeks. | 81 |
| Total | 81 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Enrolled but never dosed | 1 |
Baseline characteristics
| Characteristic | ELX/TEZ/IVA |
|---|---|
| Age, Continuous | 25.7 years STANDARD_DEVIATION 9.6 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 3 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 78 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 81 Participants |
| Sex: Female, Male Female | 37 Participants |
| Sex: Female, Male Male | 44 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 81 |
| other Total, other adverse events | 56 / 81 |
| serious Total, serious adverse events | 2 / 81 |
Outcome results
Primary
Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12
Percent reduction in cough frequency was analyzed with a mixed effects model for repeated measures (MMRM), with change from baseline at each post-baseline visit on the natural log scale as the dependent variable. The percent reduction was estimated as 100% × (1-exponential form of LS mean change estimate from the MMRM).
Time frame: Baseline, Week 8 through Week 12
Population: The Full Analysis Set (FAS) will include all enrolled participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| ELX/TEZ/IVA | Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12 | 91.7 percent reduction |
Secondary
Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12
Time frame: Baseline, Week 8 through Week 12
Population: FAS. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| ELX/TEZ/IVA | Absolute Change From Baseline in Total Step Count Per Day to the Average of Week 8 Through Week 12 | 637.56 step count per day |