Trial Outcomes & Findings for A Comparative Bioavailability Study of Lomitapide 20 mg Intact vs Sprinkled (NCT NCT02044419)
NCT ID: NCT02044419
Last Updated: 2018-02-13
Results Overview
Area under the concentration-time curve from hour 0 to the last measurable concentration of lomitapide and its metabolites (M1\& M3).
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
32 participants
Primary outcome timeframe
predose and 1, 2, 3, 4, 5, 6, 8, 10, 12, 18, 24, 48, 72, 96, 120, 144, and 168 hours post-dose
Results posted on
2018-02-13
Participant Flow
Participant milestones
| Measure |
Applesauce, Mashed Banana, Intact Capsule (ABC)
First Intervention: Contents of single 20 mg capsule of lomitapide sprinkled in applesauce Second Intervention: Contents of single 20 mg capsule of lomitapide sprinkled in mashed banana Third Intervention: Intact capsule of 20 mg lomitapide
|
Mashed Banana, Intact Capsule, Applesauce (BCA)
First Intervention: Contents of single 20 mg capsule of lomitapide sprinkled in mashed banana Second Intervention: Intact capsule of 20 mg lomitapide Third Intervention: Contents of single 20 mg capsule of lomitapide sprinkled in applesauce
|
Intact Capsule, Applesauce, Mashed Banana (CAB)
First Intervention: Contents of single 20 mg capsule of lomitapide sprinkled in applesauce Second Intervention: Contents of single 20 mg capsule of lomitapide sprinkled in mashed banana Third Intervention: Intact capsule of 20 mg lomitapide
|
|---|---|---|---|
|
First Intervention
STARTED
|
11
|
10
|
11
|
|
First Intervention
COMPLETED
|
11
|
9
|
10
|
|
First Intervention
NOT COMPLETED
|
0
|
1
|
1
|
|
Second Intervention
STARTED
|
11
|
9
|
10
|
|
Second Intervention
COMPLETED
|
11
|
9
|
10
|
|
Second Intervention
NOT COMPLETED
|
0
|
0
|
0
|
|
Third Intervention
STARTED
|
11
|
9
|
10
|
|
Third Intervention
COMPLETED
|
11
|
9
|
10
|
|
Third Intervention
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Comparative Bioavailability Study of Lomitapide 20 mg Intact vs Sprinkled
Baseline characteristics by cohort
| Measure |
Applesauce, Mashed Banana, Intact Capsule (ABC)
n=11 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in applesauce, mashed banana
Capsule taken intact
lomitapide
|
Mashed Banana, Intact Capsule, Applesauce (BCA)
n=10 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in applesauce\& mashed banana
Capsule taken intact
lomitapide
|
Intact Capsule, Applesauce, Mashed Banana (CAB)
n=11 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in applesauce\& mashed banana
Capsule taken intact
lomitapide
|
Total
n=32 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
32 Participants
n=7 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Age, Continuous
|
28.45 years
STANDARD_DEVIATION 5.837 • n=99 Participants
|
25.90 years
STANDARD_DEVIATION 5.131 • n=107 Participants
|
27.91 years
STANDARD_DEVIATION 3.27 • n=206 Participants
|
27.42 years
STANDARD_DEVIATION 4.746 • n=7 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
15 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
17 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
10 Participants
n=206 Participants
|
31 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
9 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
18 Participants
n=7 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
2 Participants
n=7 Participants
|
|
Region of Enrollment
United Kingdom
|
11 participants
n=99 Participants
|
10 participants
n=107 Participants
|
11 participants
n=206 Participants
|
32 participants
n=7 Participants
|
PRIMARY outcome
Timeframe: predose and 1, 2, 3, 4, 5, 6, 8, 10, 12, 18, 24, 48, 72, 96, 120, 144, and 168 hours post-dosePopulation: Pharmacokinetic Analysis Set included all subjects who received at least one single dose of lomitapide and who had evaluable PK data
Area under the concentration-time curve from hour 0 to the last measurable concentration of lomitapide and its metabolites (M1\& M3).
Outcome measures
| Measure |
Lomitapide Sprinkled in Applesauce (Treatment A)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in applesauce
|
Lomitapide Sprinkled in Mashed Banana (Treatment B)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in mashed banana
|
Intact Capsule of 20 mg Lomitapide (Treatment C)
n=30 Participants
Intact capsule of 20 mg lomitapide
|
|---|---|---|---|
|
AUC0-t
Lomitapide
|
38.3 ng*h/mL
Standard Deviation 15.2
|
40.2 ng*h/mL
Standard Deviation 13.4
|
43.6 ng*h/mL
Standard Deviation 17.7
|
|
AUC0-t
M1
|
70.4 ng*h/mL
Standard Deviation 20.2
|
72.9 ng*h/mL
Standard Deviation 23.1
|
75.2 ng*h/mL
Standard Deviation 22.8
|
|
AUC0-t
M3
|
443 ng*h/mL
Standard Deviation 308
|
450 ng*h/mL
Standard Deviation 292
|
460 ng*h/mL
Standard Deviation 325
|
PRIMARY outcome
Timeframe: predose and 1, 2, 3, 4, 5, 6, 8, 10, 12, 18, 24, 48, 72, 96, 120, 144, and 168 hours post-dosePopulation: Pharmacokinetic Analysis Set included all subjects who received at least one single dose of lomitapide and who had evaluable PK data
Maximum observed concentration of lomitapide and its metabolites (M1\& M3).
Outcome measures
| Measure |
Lomitapide Sprinkled in Applesauce (Treatment A)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in applesauce
|
Lomitapide Sprinkled in Mashed Banana (Treatment B)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in mashed banana
|
Intact Capsule of 20 mg Lomitapide (Treatment C)
n=30 Participants
Intact capsule of 20 mg lomitapide
|
|---|---|---|---|
|
Cmax
Lomitapide
|
0.986 ng/mL
Standard Deviation 0.536
|
1.03 ng/mL
Standard Deviation 0.463
|
1.13 ng/mL
Standard Deviation 0.591
|
|
Cmax
M1
|
2.52 ng/mL
Standard Deviation 0.748
|
2.65 ng/mL
Standard Deviation 0.813
|
2.74 ng/mL
Standard Deviation 0.796
|
|
Cmax
M3
|
30.2 ng/mL
Standard Deviation 15.6
|
31.0 ng/mL
Standard Deviation 13.3
|
28.6 ng/mL
Standard Deviation 13.7
|
PRIMARY outcome
Timeframe: predose and 1, 2, 3, 4, 5, 6, 8, 10, 12, 18, 24, 48, 72, 96, 120, 144, and 168 hours post-dosePopulation: Pharmacokinetic Analysis Set included all subjects who received at least one single dose of lomitapide and who had evaluable PK data
Time to reach maximum plasma concentration of lomitapide and its metabolites (M1\& M3).
Outcome measures
| Measure |
Lomitapide Sprinkled in Applesauce (Treatment A)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in applesauce
|
Lomitapide Sprinkled in Mashed Banana (Treatment B)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in mashed banana
|
Intact Capsule of 20 mg Lomitapide (Treatment C)
n=30 Participants
Intact capsule of 20 mg lomitapide
|
|---|---|---|---|
|
Tmax
M3
|
2.03 hours
Interval 1.0 to 4.0
|
3.00 hours
Interval 1.0 to 5.0
|
3.00 hours
Interval 1.0 to 4.0
|
|
Tmax
Lomitapide
|
4.00 hours
Interval 1.0 to 12.0
|
5.00 hours
Interval 1.0 to 12.0
|
5.00 hours
Interval 2.0 to 24.0
|
|
Tmax
M1
|
5.00 hours
Interval 2.0 to 8.0
|
5.00 hours
Interval 2.0 to 6.0
|
5.00 hours
Interval 3.0 to 8.0
|
PRIMARY outcome
Timeframe: predose and 1, 2, 3, 4, 5, 6, 8, 10, 12, 18, 24, 48, 72, 96, 120, 144, and 168 hours post-dosePopulation: Pharmacokinetic Analysis Set included all subjects who received at least one single dose of lomitapide and who had evaluable PK data
Terminal elimination half-life of lomitapide and its metabolites (M1\& M3).
Outcome measures
| Measure |
Lomitapide Sprinkled in Applesauce (Treatment A)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in applesauce
|
Lomitapide Sprinkled in Mashed Banana (Treatment B)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in mashed banana
|
Intact Capsule of 20 mg Lomitapide (Treatment C)
n=30 Participants
Intact capsule of 20 mg lomitapide
|
|---|---|---|---|
|
t1/2
Lomitapide
|
52.4 h
Standard Deviation 13.3
|
47.7 h
Standard Deviation 12.2
|
48.8 h
Standard Deviation 10.7
|
|
t1/2
M1
|
38.7 h
Standard Deviation 7.56
|
37.0 h
Standard Deviation 6.78
|
39.1 h
Standard Deviation 6.32
|
|
t1/2
M3
|
46.0 h
Standard Deviation 13.1
|
45.7 h
Standard Deviation 11.5
|
44.0 h
Standard Deviation 11.6
|
PRIMARY outcome
Timeframe: predose and 1, 2, 3, 4, 5, 6, 8, 10, 12, 18, 24, 48, 72, 96, 120, 144, and 168 hours post-dosePopulation: Pharmacokinetic Analysis Set included all subjects who received at least one single dose of lomitapide and who had evaluable PK data
Area under the plasma concentration vs time curve from zero to infinity of lomitapide and its metabolites (M1\& M3).
Outcome measures
| Measure |
Lomitapide Sprinkled in Applesauce (Treatment A)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in applesauce
|
Lomitapide Sprinkled in Mashed Banana (Treatment B)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in mashed banana
|
Intact Capsule of 20 mg Lomitapide (Treatment C)
n=30 Participants
Intact capsule of 20 mg lomitapide
|
|---|---|---|---|
|
AUC0-∞
Lomitapide
|
42.5 ng*h/mL
Standard Deviation 17.7
|
43.8 ng*h/mL
Standard Deviation 15.3
|
47.7 ng*h/mL
Standard Deviation 20.6
|
|
AUC0-∞
M1
|
72.8 ng*h/mL
Standard Deviation 20.8
|
75.2 ng*h/mL
Standard Deviation 23.7
|
77.8 ng*h/mL
Standard Deviation 23.9
|
|
AUC0-∞
M3
|
457 ng*h/mL
Standard Deviation 317
|
464 ng*h/mL
Standard Deviation 300
|
474 ng*h/mL
Standard Deviation 333
|
PRIMARY outcome
Timeframe: predose and 1, 2, 3, 4, 5, 6, 8, 10, 12, 18, 24, 48, 72, 96, 120, 144, and 168 hours post-dosePopulation: Pharmacokinetic Analysis Set included all subjects who received at least one single dose of lomitapide and who had evaluable PK data
Elimination rate constant estimated from individual linear regression of the terminal part of the log concentration vs time curve of lomitapide and its metabolites (M1\& M3).
Outcome measures
| Measure |
Lomitapide Sprinkled in Applesauce (Treatment A)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in applesauce
|
Lomitapide Sprinkled in Mashed Banana (Treatment B)
n=30 Participants
Contents of single 20 mg capsule of lomitapide sprinkled in mashed banana
|
Intact Capsule of 20 mg Lomitapide (Treatment C)
n=30 Participants
Intact capsule of 20 mg lomitapide
|
|---|---|---|---|
|
λz
Lomitapide
|
0.0140 1/h
Standard Deviation 0.00341
|
0.0153 1/h
Standard Deviation 0.00343
|
0.0149 1/h
Standard Deviation 0.00320
|
|
λz
M1
|
0.0185 1/h
Standard Deviation 0.00344
|
0.0195 1/h
Standard Deviation 0.00457
|
0.0182 1/h
Standard Deviation 0.00309
|
|
λz
M3
|
0.0161 1/h
Standard Deviation 0.00408
|
0.0162 1/h
Standard Deviation 0.00428
|
0.0167 1/h
Standard Deviation 0.00405
|
Adverse Events
Lomitapide Sprinkled in Applesauce (Treatment A)
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Lomitapide Sprinkled in Mashed Banana (Treatment B)
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Intact Capsule of 20 mg Lomitapide (Treatment C)
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Lomitapide Sprinkled in Applesauce (Treatment A)
n=30 participants at risk
Contents of single 20 mg capsule of lomitapide sprinkled in applesauce
|
Lomitapide Sprinkled in Mashed Banana (Treatment B)
n=31 participants at risk
Contents of single 20 mg capsule of lomitapide sprinkled in mashed banana
|
Intact Capsule of 20 mg Lomitapide (Treatment C)
n=31 participants at risk
Intact capsule of 20 mg lomitapide
|
|---|---|---|---|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/30
|
0.00%
0/31
|
3.2%
1/31 • Number of events 1
|
|
Nervous system disorders
Headache
|
3.3%
1/30 • Number of events 1
|
0.00%
0/31
|
0.00%
0/31
|
|
Renal and urinary disorders
Polyuria
|
3.3%
1/30 • Number of events 1
|
0.00%
0/31
|
0.00%
0/31
|
|
Vascular disorders
Presyncope
|
3.3%
1/30 • Number of events 1
|
0.00%
0/31
|
0.00%
0/31
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/30
|
3.2%
1/31 • Number of events 1
|
0.00%
0/31
|
|
Gastrointestinal disorders
Vomiting
|
3.3%
1/30 • Number of events 1
|
0.00%
0/31
|
3.2%
1/31 • Number of events 1
|
|
Gastrointestinal disorders
Dry mouth
|
0.00%
0/30
|
3.2%
1/31 • Number of events 1
|
0.00%
0/31
|
|
Gastrointestinal disorders
Glossodynia
|
0.00%
0/30
|
0.00%
0/31
|
3.2%
1/31 • Number of events 1
|
|
Gastrointestinal disorders
Oral pain
|
0.00%
0/30
|
0.00%
0/31
|
3.2%
1/31 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Nasopharyngitis
|
3.3%
1/30 • Number of events 1
|
0.00%
0/31
|
3.2%
1/31 • Number of events 1
|
|
Eye disorders
Conjunctival haemorrhage
|
0.00%
0/30
|
3.2%
1/31 • Number of events 1
|
0.00%
0/31
|
|
Eye disorders
Eye swelling
|
0.00%
0/30
|
3.2%
1/31 • Number of events 1
|
0.00%
0/31
|
|
General disorders
Catheter site pain
|
0.00%
0/30
|
3.2%
1/31 • Number of events 1
|
0.00%
0/31
|
|
Infections and infestations
Herpes simplex
|
0.00%
0/30
|
0.00%
0/31
|
3.2%
1/31 • Number of events 1
|
|
Vascular disorders
Epistaxis
|
0.00%
0/30
|
0.00%
0/31
|
3.2%
1/31 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/30
|
3.2%
1/31 • Number of events 1
|
3.2%
1/31 • Number of events 1
|
Additional Information
Alison Long, MD - VP Clinical
Aegerion Pharmaceuticals, Inc.
Phone: 857-242-5142
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Described in contract.
- Publication restrictions are in place
Restriction type: OTHER