Cystic Fibrosis
Conditions
Brief summary
This is a Phase 3, multicenter, open-label and roll-over study in participants who are 12 to \<24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.
Interventions
DRUGLUM/IVA
LUM/IVA granules 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 Months to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Participants From Study VX16-809-122 Part B (Study 122) * Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B * Participants Not From Study 122 * Subjects will be 1 to less than 2 years of age * Homozygous for the F508del mutation (F/F) Key
Exclusion criteria
* Any clinically significant laboratory abnormalities that would interfere with the study assessments or pose an undue risk for the subject * Solid organ or hematological transplantation 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 120 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Absolute Change in Sweat Chloride (SwCl) | From Baseline at Week 96 | Sweat samples were collected using an approved collection device. |
Countries
Canada, United States
Participant flow
Recruitment details
The study was conducted in participants with cystic fibrosis (CF) aged 12 months through less than (\<) 24 months of age at treatment initiation who were homozygous for F508del and participants who completed the 24-week treatment period along with the safety follow-up in study VX16-809-122 (NCT03601637) Part B.
Participants by arm
| Arm | Count |
|---|---|
| LUM/IVA Participants weighing 7 to \<9 kg received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants \>=14 kg received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 weeks. | 52 |
| Total | 52 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 2 |
| Overall Study | Commercial drug is available for participants | 7 |
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Withdrawal of consent (not due to AE) | 4 |
Baseline characteristics
| Characteristic | LUM/IVA |
|---|---|
| Age, Continuous | 18.8 months STANDARD_DEVIATION 3.32 |
| Race/Ethnicity, Customized American Indian or Alaska Native | 1 Participants |
| Race/Ethnicity, Customized Asian | 0 Participants |
| Race/Ethnicity, Customized Black or African American | 0 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 1 Participants |
| Race/Ethnicity, Customized More than one race | 2 Participants |
| Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race/Ethnicity, Customized Not collected per local regulations | 8 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 43 Participants |
| Race/Ethnicity, Customized Other | 1 Participants |
| Race/Ethnicity, Customized White | 40 Participants |
| Sex: Female, Male Female | 24 Participants |
| Sex: Female, Male Male | 28 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 52 |
| other Total, other adverse events | 50 / 52 |
| serious Total, serious adverse events | 12 / 52 |
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 120
Population: Safety set included all participants who are exposed to any amount of study drug in this study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| LUM/IVA | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Participants with TEAEs | 52 Participants |
| LUM/IVA | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Participants with SAEs | 12 Participants |
Secondary
Absolute Change in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
Time frame: From Baseline at Week 96
Population: The Full Analysis Set (FAS) included all participants who were enrolled and dosed in Study 124. Here Overall Number of Participants Analyzed signifies those participants who were evaluated for this specific outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| LUM/IVA | Absolute Change in Sweat Chloride (SwCl) | -21.0 millimole per liter (mmol/L) | Standard Deviation 14.9 |