Trial Outcomes & Findings for Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia (NCT NCT00943306)

NCT ID: NCT00943306

Last Updated: 2018-06-13

Results Overview

Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

19 participants

Primary outcome timeframe

Baseline and Week 126

Results posted on

2018-06-13

Participant Flow

Participant milestones

Participant milestones
Measure
Lomitapide
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Overall Study
STARTED
19
Overall Study
COMPLETED
16
Overall Study
NOT COMPLETED
3

Reasons for withdrawal

Reasons for withdrawal
Measure
Lomitapide
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Overall Study
Physician Decision
1
Overall Study
Death
1
Overall Study
Sponsor decision due to non-compliance
1

Baseline Characteristics

Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Lomitapide
n=19 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
19 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Age, Continuous
30.4 years
STANDARD_DEVIATION 11.74 • n=5 Participants
Sex: Female, Male
Female
9 Participants
n=5 Participants
Sex: Female, Male
Male
10 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
1 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
17 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
Region of Enrollment
Canada
2 participants
n=5 Participants
Region of Enrollment
United States
3 participants
n=5 Participants
Region of Enrollment
South Africa
9 participants
n=5 Participants
Region of Enrollment
Italy
5 participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline and Week 126

Population: Week 126 Completers Population

Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=17 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
-45.5 Percent Change
Standard Deviation 31.35

SECONDARY outcome

Timeframe: Baseline and Week 174

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=16 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
-51.0 Percent Change
Standard Deviation 16.03

SECONDARY outcome

Timeframe: Baseline and Week 222

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=15 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
-58.5 Percent Change
Standard Deviation 24.25

SECONDARY outcome

Timeframe: Baseline and Week 246

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=14 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
-60.1 Percent Change
Standard Deviation 18.51

SECONDARY outcome

Timeframe: Baseline and Week 270

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
-74.0 Percent Change
Standard Deviation 19.10

SECONDARY outcome

Timeframe: Baseline and Week 294

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=3 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
-51.1 Percent Change
Standard Deviation 10.11

SECONDARY outcome

Timeframe: Baseline and Week 126

Population: Week 126 Completers Population

Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=17 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Total Cholesterol
-43.2 Percent Change
Standard Deviation 25.35

SECONDARY outcome

Timeframe: Baseline and Week 174

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=16 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Total Cholesterol
-46.9 Percent Change
Standard Deviation 15.21

SECONDARY outcome

Timeframe: Baseline and Week 222

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=15 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Total Cholesterol
-51.0 Percent Change
Standard Deviation 21.34

SECONDARY outcome

Timeframe: Baseline and Week 246

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=14 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Total Cholesterol
-54.1 Percent Change
Standard Deviation 16.88

SECONDARY outcome

Timeframe: Baseline and Week 270

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Total Cholesterol
-65.2 Percent Change
Standard Deviation 15.97

SECONDARY outcome

Timeframe: Baseline and Week 294

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=3 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Total Cholesterol
-43.9 Percent Change
Standard Deviation 5.01

SECONDARY outcome

Timeframe: Baseline and Week 126

Population: Week 126 Completers Population

Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=17 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein B (Apo B)
-53.6 Percent Change
Standard Deviation 23.74

SECONDARY outcome

Timeframe: Baseline and Week 174

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=16 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein B (Apo B)
-59.4 Percent Change
Standard Deviation 12.60

SECONDARY outcome

Timeframe: Baseline and Week 222

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=15 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein B (Apo B)
-65.1 Percent Change
Standard Deviation 20.72

SECONDARY outcome

Timeframe: Baseline and Week 246

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=14 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein B (Apo B)
-65.9 Percent Change
Standard Deviation 15.76

SECONDARY outcome

Timeframe: Baseline and Week 270

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein B (Apo B)
-76.7 Percent Change
Standard Deviation 16.09

SECONDARY outcome

Timeframe: Baseline and Week 294

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=3 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein B (Apo B)
-60.9 Percent Change
Standard Deviation 12.17

SECONDARY outcome

Timeframe: Baseline and Week 126

Population: Week 126 Completers Population

Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=17 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Triglycerides
-37.5 Percent Change
Standard Deviation 42.52

SECONDARY outcome

Timeframe: Baseline and Week 174

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=16 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Triglycerides
-31.7 Percent Change
Standard Deviation 37.09

SECONDARY outcome

Timeframe: Baseline and Week 222

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=15 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Triglycerides
-27.6 Percent Change
Standard Deviation 48.88

SECONDARY outcome

Timeframe: Baseline and Week 246

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=14 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Triglycerides
-41.9 Percent Change
Standard Deviation 33.16

SECONDARY outcome

Timeframe: Baseline and Week 270

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Triglycerides
-48.5 Percent Change
Standard Deviation 34.24

SECONDARY outcome

Timeframe: Baseline and Week 294

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=3 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Triglycerides
29.2 Percent Change
Standard Deviation 58.74

SECONDARY outcome

Timeframe: Baseline and Week 126

Population: Week 126 Completers Population

Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=17 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
-47.1 Percent Change
Standard Deviation 27.83

SECONDARY outcome

Timeframe: Baseline and Week 174

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=16 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
-53.5 Percent Change
Standard Deviation 16.42

SECONDARY outcome

Timeframe: Baseline and Week 222

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=15 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
-57.0 Percent Change
Standard Deviation 24.07

SECONDARY outcome

Timeframe: Baseline and Week 246

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=14 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
-58.8 Percent Change
Standard Deviation 17.73

SECONDARY outcome

Timeframe: Baseline and Week 270

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
-71.5 Percent Change
Standard Deviation 17.65

SECONDARY outcome

Timeframe: Baseline and Week 294

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=3 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
-46.6 Percent Change
Standard Deviation 5.19

SECONDARY outcome

Timeframe: Baseline and Week 126

Population: Week 126 Completers Population

Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=17 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
-36.8 Percent Change
Standard Deviation 43.90

SECONDARY outcome

Timeframe: Baseline and Week 174

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=16 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
-31.5 Percent Change
Standard Deviation 36.32

SECONDARY outcome

Timeframe: Baseline and Week 222

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=15 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
-26.3 Percent Change
Standard Deviation 49.94

SECONDARY outcome

Timeframe: Baseline and Week 246

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=14 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
-41.4 Percent Change
Standard Deviation 34.20

SECONDARY outcome

Timeframe: Baseline and Week 270

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
-48.7 Percent Change
Standard Deviation 33.41

SECONDARY outcome

Timeframe: Baseline and Week 294

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=3 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
30.6 Percent Change
Standard Deviation 59.94

SECONDARY outcome

Timeframe: Baseline and Week 126

Population: Week 126 Completers Population

Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=16 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Lp(a)
5.5 Percent Change
Standard Deviation 43.62

SECONDARY outcome

Timeframe: Baseline and Week 174

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=15 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Lp(a)
10.2 Percent Change
Standard Deviation 60.64

SECONDARY outcome

Timeframe: Baseline and Week 222

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=14 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Lp(a)
-12.8 Percent Change
Standard Deviation 49.00

SECONDARY outcome

Timeframe: Baseline and Week 246

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=13 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Lp(a)
3.4 Percent Change
Standard Deviation 54.22

SECONDARY outcome

Timeframe: Baseline and Week 270

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Lp(a)
-6.6 Percent Change
Standard Deviation 48.99

SECONDARY outcome

Timeframe: Baseline and Week 294

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=3 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Lp(a)
-10.4 Percent Change
Standard Deviation 35.64

SECONDARY outcome

Timeframe: Baseline and Week 126

Population: Week 126 Completers Population

Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=17 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
-8.3 Percent Change
Standard Deviation 19.28

SECONDARY outcome

Timeframe: Baseline and Week 174

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=16 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
3.8 Percent Change
Standard Deviation 26.51

SECONDARY outcome

Timeframe: Baseline and Week 222

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=15 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
-2.7 Percent Change
Standard Deviation 21.22

SECONDARY outcome

Timeframe: Baseline and Week 246

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=14 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
-12.5 Percent Change
Standard Deviation 19.17

SECONDARY outcome

Timeframe: Baseline and Week 270

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
-10.3 Percent Change
Standard Deviation 27.68

SECONDARY outcome

Timeframe: Baseline and Week 294

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=3 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
-23.5 Percent Change
Standard Deviation 2.27

SECONDARY outcome

Timeframe: Baseline and Week 126

Population: Week 126 Completers Population

Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=17 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein AI (Apo AI)
-14.0 Percent Change
Standard Deviation 17.71

SECONDARY outcome

Timeframe: Baseline and Week 174

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=16 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein AI (Apo AI)
-8.2 Percent Change
Standard Deviation 20.12

SECONDARY outcome

Timeframe: Baseline and Week 222

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=15 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein AI (Apo AI)
-2.7 Percent Change
Standard Deviation 33.30

SECONDARY outcome

Timeframe: Baseline and Week 246

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=14 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein AI (Apo AI)
-16.8 Percent Change
Standard Deviation 26.62

SECONDARY outcome

Timeframe: Baseline and Week 270

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=9 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein AI (Apo AI)
-17.8 Percent Change
Standard Deviation 20.12

SECONDARY outcome

Timeframe: Baseline and Week 294

Population: Safety Population - The number of patients in the Safety Population at time points after Week 126 decreased because the study remained active within each country until either approval of the marketing application was obtained, or, in countries where marketing authorization was not sought for, patients transitioned into an Expanded Access Program.

Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).

Outcome measures

Outcome measures
Measure
Lomitapide
n=3 Participants
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
Percent Change in Apolipoprotein AI (Apo AI)
-30.5 Percent Change
Standard Deviation 22.2

Adverse Events

Lomitapide

Serious events: 7 serious events
Other events: 17 other events
Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure
Lomitapide
n=19 participants at risk
Maximum tolerated dose of lomitapide (up to 80mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
General disorders
Sudden Cardiac Death
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Hepatobiliary disorders
Hepatotoxicity
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Cardiac disorders
Angina Pectoris
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Cardiac disorders
Aortic Value Incompetence
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Cardiac disorders
Coronary Artery Disease
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Diarrhoea
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Reflux Oesophagitis
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Lower Respiratory Tract Infection
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Injury, poisoning and procedural complications
Subdural Haematoma
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
International Normalized Ratio Increased
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Metabolism and nutrition disorders
Dehydration
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Musculoskeletal and connective tissue disorders
Rhabdomyolsis
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Nervous system disorders
Facial Palsy
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Surgical and medical procedures
Anticoagulant Therapy
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Surgical and medical procedures
Transfusion
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Vascular disorders
Arteriovenous Fistula
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Vascular disorders
Hypovolaemic Shock
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)

Other adverse events

Other adverse events
Measure
Lomitapide
n=19 participants at risk
Maximum tolerated dose of lomitapide (up to 80mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis. lomitapide: 5-60 mg po every day
General disorders
Sudden Cardiac Death
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Blood and lymphatic system disorders
Anaemia
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Blood and lymphatic system disorders
Iron Deficiency Anaemia
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Cardiac disorders
Angina Pectoris
21.1%
4/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Cardiac disorders
Aortic Valve Incompetence
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Cardiac disorders
Chest Pain
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Cardiac disorders
Coronary Artery Disease
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Cardiac disorders
Sinus Bradycardia
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Ear and labyrinth disorders
Tinnitus
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Ear and labyrinth disorders
Vertigo
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Endocrine disorders
Hypothyroidism
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Eye disorders
Blepharitis
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Abdominal Distension
15.8%
3/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Chapped Lips
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Dental Caries
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Diarrhoea
42.1%
8/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Dyspepsia
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Epigastric Discomfort
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Flatulence
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Gastrooesophageal Reflux Disease
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Gingival Bleeding
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Haemorrhoidal Haemorrhage
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Hiatus Hernia
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Intestinal Mass
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Nausea
31.6%
6/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Reflux Oesophagitis
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Stomach Discomfort
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Toothache
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Gastrointestinal disorders
Vomiting
26.3%
5/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
General disorders
Asthenia
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
General disorders
Chest Pain
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
General disorders
Fatigue
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
General disorders
Influenza
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
General disorders
Oedema Peripheral
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
General disorders
Pain
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
General disorders
Pyrexia
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
General disorders
Vestibulitis
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Hepatobiliary disorders
Hepatotoxicity
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Immune system disorders
Drug Hypersensitivity
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Bronchitis
15.8%
3/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Gastroenteritis
15.8%
3/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Gastrointestinal Infection
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Influenza
26.3%
5/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Lower Respiratory Tract Infection
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Nasopharyngitis
21.1%
4/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Sinusitus
15.8%
3/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Tooth Abscess
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Upper Respiratory Tract Infection
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Urinary Tract Infection
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Infections and infestations
Viral Infection
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Injury, poisoning and procedural complications
Fall
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Injury, poisoning and procedural complications
Joint Injury
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Injury, poisoning and procedural complications
Post Procedural Diarrhoea
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Injury, poisoning and procedural complications
Procedural Headache
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Injury, poisoning and procedural complications
Skeletal Injury
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Injury, poisoning and procedural complications
Subdural Haematoma
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Alanine Aminotransferase Increased
15.8%
3/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Asparate Aminotransferase Increased
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Blood creatine phosphokinase Increased
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Blood Potassium Increased
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Blood Pressure Increased
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Carotene Decreased
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Carotid Bruit
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
International Normalized Ratio Decreased
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
International Normalized Ratio Increased
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Liver Function Test Abnormal
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Prothrombin Time Prolonged
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Transaminases Increased
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Vitamin K Decreased
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
Weight Decreased
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Investigations
White Blood Cell Count Decreased
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Metabolism and nutrition disorders
Decreased Appetite
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Metabolism and nutrition disorders
Dehydration
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Metabolism and nutrition disorders
Iron Deficiency
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Metabolism and nutrition disorders
Oral Intake Reduced
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Metabolism and nutrition disorders
Vitamin E Deficiency
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Musculoskeletal and connective tissue disorders
Arthralgia
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Musculoskeletal and connective tissue disorders
Back Pain
15.8%
3/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Musculoskeletal and connective tissue disorders
Musculoskeletal Pain
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Musculoskeletal and connective tissue disorders
Neck Pain
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Musculoskeletal and connective tissue disorders
Tendonitis
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Nervous system disorders
Dizziness
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Nervous system disorders
Facial Palsy
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Nervous system disorders
Headache
31.6%
6/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Nervous system disorders
Hypoaesthesia
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Nervous system disorders
Migraine
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Nervous system disorders
Paraesthesia
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Nervous system disorders
Sensory Disturbance
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Nervous system disorders
Syncope
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Psychiatric disorders
Anxiety
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Psychiatric disorders
Depression
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Psychiatric disorders
Stress
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Renal and urinary disorders
Nephrolithiasis
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Reproductive system and breast disorders
Dysmenorrhoea
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Respiratory, thoracic and mediastinal disorders
Apnoea
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Respiratory, thoracic and mediastinal disorders
Cough
10.5%
2/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Respiratory, thoracic and mediastinal disorders
Dyspnoea
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Respiratory, thoracic and mediastinal disorders
Dyspnoea Exertional
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Respiratory, thoracic and mediastinal disorders
Epistaxis
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Respiratory, thoracic and mediastinal disorders
Painful Respiration
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal Pain
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Respiratory, thoracic and mediastinal disorders
Sinus Congestion
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Skin and subcutaneous tissue disorders
Alopecia
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Skin and subcutaneous tissue disorders
Dry Skin
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Skin and subcutaneous tissue disorders
Hair growth abnormal
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Skin and subcutaneous tissue disorders
Scar
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Surgical and medical procedures
Anticoagulant Therapy
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Surgical and medical procedures
Transfusion
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Vascular disorders
Arterial Stenosis
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Vascular disorders
Arteriovenous Fistula
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Vascular disorders
Haematoma
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Vascular disorders
Hypertension
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Vascular disorders
Hypotension
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Vascular disorders
Hypovolaemic Shock
5.3%
1/19 • Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)

Additional Information

Alison Long, MD - VP Clinical

Aegerion Pharmaceuticals, Inc.

Phone: 857-242-5142

Results disclosure agreements

  • Principal investigator is a sponsor employee The CTAs generally envision a multisite publication, with the PI's right to publish individually if the pooled publication does not occur within 12 months of study completion. Sponsor has a 45 to 60 day review/approval period to request deletion of confidential information or to request limited deferral to protect its proprietary technology. In one case, the publication provision is more general, specifying that the Sponsor and clinical site will agree on the manner/terms of publication.
  • Publication restrictions are in place

Restriction type: OTHER