Trial Outcomes & Findings for A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del CFTR Mutation (NCT NCT02823470)
NCT ID: NCT02823470
Last Updated: 2018-09-27
Results Overview
Percentage adherence was reported in terms of median and full range due to small sample size.
Recruitment status
TERMINATED
Study phase
PHASE4
Target enrollment
24 participants
Primary outcome timeframe
Up to 35 weeks
Results posted on
2018-09-27
Participant Flow
Participant milestones
| Measure |
LUM/IVA Through Deactivated Smart Pill Bottle (Control)
Participants received lumacaftor (LUM) 400 milligram (mg) in combination with ivacaftor (IVA) 250 mg as oral tablet every 12 hours (q12h) for up to 35 weeks through a de-activated smart pill bottle (control).
|
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.
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
15
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
9
|
15
|
Reasons for withdrawal
| Measure |
LUM/IVA Through Deactivated Smart Pill Bottle (Control)
Participants received lumacaftor (LUM) 400 milligram (mg) in combination with ivacaftor (IVA) 250 mg as oral tablet every 12 hours (q12h) for up to 35 weeks through a de-activated smart pill bottle (control).
|
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.
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Study terminated by sponsor
|
8
|
15
|
Baseline Characteristics
A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del CFTR Mutation
Baseline characteristics by cohort
| Measure |
LUM/IVA Through Deactivated Smart Pill Bottle (Control)
n=9 Participants
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).
|
LUM/IVA Through Activated Smart Pill Bottle (Test)
n=15 Participants
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.
|
Total
n=24 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
20.8 Years
STANDARD_DEVIATION 4.0 • n=5 Participants
|
25.1 Years
STANDARD_DEVIATION 10.3 • n=7 Participants
|
23.5 Years
STANDARD_DEVIATION 8.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
24 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
24 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 35 weeksPopulation: Full analysis set (FAS) included randomized participants who received at least 1 dose of study drug.
Percentage adherence was reported in terms of median and full range due to small sample size.
Outcome measures
| Measure |
LUM/IVA Through Deactivated Smart Pill Bottle (Control)
n=9 Participants
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).
|
LUM/IVA Through Activated Smart Pill Bottle (Test)
n=15 Participants
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.
|
|---|---|---|
|
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment
|
85.49 percentage of adherence
Interval 32.0 to 96.4
|
92.07 percentage of adherence
Interval 74.2 to 99.3
|
SECONDARY outcome
Timeframe: Up to Week 12Population: 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.
Percentage adherence was reported in terms of median and full range due to small sample size.
Outcome measures
| Measure |
LUM/IVA Through Deactivated Smart Pill Bottle (Control)
n=6 Participants
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).
|
LUM/IVA Through Activated Smart Pill Bottle (Test)
n=14 Participants
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.
|
|---|---|---|
|
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks
|
88.69 percentage of adherence
Interval 37.5 to 94.0
|
97.32 percentage of adherence
Interval 74.4 to 100.0
|
SECONDARY outcome
Timeframe: Up to Week 12Population: 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.
Number of participants with \>=80% adherence to LUM/IVA treatment over 12 weeks were reported.
Outcome measures
| Measure |
LUM/IVA Through Deactivated Smart Pill Bottle (Control)
n=6 Participants
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).
|
LUM/IVA Through Activated Smart Pill Bottle (Test)
n=14 Participants
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.
|
|---|---|---|
|
Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence
|
4 Participants
|
13 Participants
|
SECONDARY outcome
Timeframe: Up to Week 12Population: 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.
Number of participants with \>=90% adherence to LUM/IVA treatment over 12 weeks were reported.
Outcome measures
| Measure |
LUM/IVA Through Deactivated Smart Pill Bottle (Control)
n=6 Participants
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).
|
LUM/IVA Through Activated Smart Pill Bottle (Test)
n=14 Participants
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.
|
|---|---|---|
|
Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence
|
2 Participants
|
12 Participants
|
Adverse Events
LUM/IVA Through Deactivated Smart Pill Bottle (Control)
Serious events: 1 serious events
Other events: 4 other events
Deaths: 0 deaths
LUM/IVA Through Activated Smart Pill Bottle (Test)
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
LUM/IVA Through Deactivated Smart Pill Bottle (Control)
n=9 participants at risk
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).
|
LUM/IVA Through Activated Smart Pill Bottle (Test)
n=15 participants at risk
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.
|
|---|---|---|
|
Hepatobiliary disorders
Cholecystitis
|
11.1%
1/9 • Baseline up to Week 35
|
0.00%
0/15 • Baseline up to Week 35
|
Other adverse events
| Measure |
LUM/IVA Through Deactivated Smart Pill Bottle (Control)
n=9 participants at risk
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).
|
LUM/IVA Through Activated Smart Pill Bottle (Test)
n=15 participants at risk
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.
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiration abnormal
|
11.1%
1/9 • Baseline up to Week 35
|
13.3%
2/15 • Baseline up to Week 35
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
11.1%
1/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/9 • Baseline up to Week 35
|
13.3%
2/15 • Baseline up to Week 35
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/9 • Baseline up to Week 35
|
13.3%
2/15 • Baseline up to Week 35
|
|
Gastrointestinal disorders
Flatulence
|
0.00%
0/9 • Baseline up to Week 35
|
13.3%
2/15 • Baseline up to Week 35
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
1/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Gastrointestinal disorders
Frequent bowel movements
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Infections and infestations
Upper respiratory tract infection
|
11.1%
1/9 • Baseline up to Week 35
|
20.0%
3/15 • Baseline up to Week 35
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
11.1%
1/9 • Baseline up to Week 35
|
0.00%
0/15 • Baseline up to Week 35
|
|
Infections and infestations
Upper respiratory tract infection bacterial
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Nervous system disorders
Headache
|
0.00%
0/9 • Baseline up to Week 35
|
13.3%
2/15 • Baseline up to Week 35
|
|
Nervous system disorders
Loss of consciousness
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Nervous system disorders
Syncope
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Metabolism and nutrition disorders
Abnormal loss of weight
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Congenital, familial and genetic disorders
Cystic fibrosis related diabetes
|
11.1%
1/9 • Baseline up to Week 35
|
0.00%
0/15 • Baseline up to Week 35
|
|
General disorders
Fatigue
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Injury, poisoning and procedural complications
Procedural pain
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
|
Psychiatric disorders
Depressed mood
|
0.00%
0/9 • Baseline up to Week 35
|
6.7%
1/15 • Baseline up to Week 35
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee PI is free to publish results of the study after (1) the first multi-center publication, (2) if the sponsor elects not to publish the results, or (3) 18 months after close of the study, whichever occurs first. Proposed publications are to be submitted to the sponsor for review and comment for a period of at least 45 days (which may be extended under certain circumstances related to protection of intellectual property); the sponsor cannot require changes to the proposed publications.
- Publication restrictions are in place
Restriction type: OTHER