Cystic Fibrosis
Conditions
Brief summary
The study will evaluate the impact of smart adherence technology for monitoring on lumacaftor/ivacaftor (LUM/IVA) adherence rates among subjects 16 years of age and older with Cystic Fibrosis (CF) who are homozygous for the F508del Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mutation.
Interventions
DRUGLUM/IVA
LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
DEVICEactivated smart device
DEVICEde-activated smart device
Sponsors
Vertex Pharmaceuticals Incorporated
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF). * Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures. * Confirmed diagnosis of CF and homozygous for the F508del-CFTR mutation * Forced Expiratory Volume in one second/forced vital capacity (FEV1) ≥40% of predicted normal for age, sex, and height
Exclusion criteria
* Presence of moderate or severe hepatic impairment (Child-Pugh Class B or C) * Subjects currently receiving invasive mechanical ventilation * Known history of alcohol or drug abuse in the past year * Clinically significant abnormal laboratory values during screening * Pregnant or nursing females * Female subjects and female partners of male subjects who plan to become pregnant during Treatment Period or within 90 days following the last dose of study drug * History of solid organ or hematological transplantation * Ongoing or prior participation in an investigational drug study within 30 days of screening * Current use of commercial LUM/IVA combination therapy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment | Up to 35 weeks | Percentage adherence was reported in terms of median and full range due to small sample size. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks | Up to Week 12 | Percentage adherence was reported in terms of median and full range due to small sample size. |
| Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence | Up to Week 12 | Number of participants with \>=80% adherence to LUM/IVA treatment over 12 weeks were reported. |
| Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence | Up to Week 12 | Number of participants with \>=90% adherence to LUM/IVA treatment over 12 weeks were reported. |
Countries
Canada, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| LUM/IVA Through Deactivated Smart Pill Bottle (Control) Participants received LUM 400 mg in combination with IVA 250 mg as oral tablet q12h for up to 35 weeks through a de-activated smart pill bottle (control). | 9 |
| LUM/IVA Through Activated Smart Pill Bottle (Test) Participants received LUM 400 mg in combination with IVA 250 mg as oral tablet q12h for up to 35 weeks through an activated smart pill bottle. | 15 |
| Total | 24 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 0 |
| Overall Study | Study terminated by sponsor | 8 | 15 |
Baseline characteristics
| Characteristic | LUM/IVA Through Deactivated Smart Pill Bottle (Control) | LUM/IVA Through Activated Smart Pill Bottle (Test) | Total |
|---|---|---|---|
| Age, Continuous | 20.8 Years STANDARD_DEVIATION 4 | 25.1 Years STANDARD_DEVIATION 10.3 | 23.5 Years STANDARD_DEVIATION 8.6 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 9 Participants | 15 Participants | 24 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 9 Participants | 15 Participants | 24 Participants |
| Sex: Female, Male Female | 5 Participants | 6 Participants | 11 Participants |
| Sex: Female, Male Male | 4 Participants | 9 Participants | 13 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 9 | 0 / 15 |
| other Total, other adverse events | 4 / 9 | 9 / 15 |
| serious Total, serious adverse events | 1 / 9 | 0 / 15 |
Outcome results
Primary
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment
Percentage adherence was reported in terms of median and full range due to small sample size.
Time frame: Up to 35 weeks
Population: Full analysis set (FAS) included randomized participants who received at least 1 dose of study drug.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| LUM/IVA Through Deactivated Smart Pill Bottle (Control) | Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment | 85.49 percentage of adherence |
| LUM/IVA Through Activated Smart Pill Bottle (Test) | Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment | 92.07 percentage of adherence |
Secondary
Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence
Number of participants with \>=80% adherence to LUM/IVA treatment over 12 weeks were reported.
Time frame: Up to Week 12
Population: FAS included randomized participants who received at least 1 dose of study drug. Overall Number of Participants Analyzed signifies those participants who had at least 12 weeks of eligible smart pill bottle data.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| LUM/IVA Through Deactivated Smart Pill Bottle (Control) | Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence | 4 Participants |
| LUM/IVA Through Activated Smart Pill Bottle (Test) | Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence | 13 Participants |
Secondary
Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence
Number of participants with \>=90% adherence to LUM/IVA treatment over 12 weeks were reported.
Time frame: Up to Week 12
Population: FAS included randomized participants who received at least 1 dose of study drug. Overall Number of Participants Analyzed signifies those participants who had at least 12 weeks of eligible smart pill bottle data.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| LUM/IVA Through Deactivated Smart Pill Bottle (Control) | Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence | 2 Participants |
| LUM/IVA Through Activated Smart Pill Bottle (Test) | Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence | 12 Participants |
Secondary
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks
Percentage adherence was reported in terms of median and full range due to small sample size.
Time frame: Up to Week 12
Population: FAS included randomized participants who received at least 1 dose of study drug. Overall Number of Participants Analyzed signifies those participants who had at least 12 weeks of eligible smart pill bottle data.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| LUM/IVA Through Deactivated Smart Pill Bottle (Control) | Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks | 88.69 percentage of adherence |
| LUM/IVA Through Activated Smart Pill Bottle (Test) | Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks | 97.32 percentage of adherence |