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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00943306
Recruitment Status : Completed
First Posted : July 22, 2009
Results First Posted : May 11, 2018
Last Update Posted : June 13, 2018
Sponsor:
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Familial Hypercholesterolemia
Intervention Drug: lomitapide
Enrollment 19
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Lomitapide
Hide Arm/Group Description

Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.

lomitapide: 5-60 mg po every day

Period Title: Overall Study
Started 19
Completed 16
Not Completed 3
Reason Not Completed
Physician Decision             1
Death             1
Sponsor decision due to non-compliance             1
Arm/Group Title Lomitapide
Hide Arm/Group Description

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 Number of Baseline Participants 19
Hide Baseline Analysis Population Description
Baseline from initiation of treatment in Study 733-005/UP1002 (Safety Population)
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants
<=18 years
0
   0.0%
Between 18 and 65 years
19
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 19 participants
30.4  (11.74)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants
Female
9
  47.4%
Male
10
  52.6%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
   5.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
17
  89.5%
More than one race
0
   0.0%
Unknown or Not Reported
1
   5.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 19 participants
Canada 2
United States 3
South Africa 9
Italy 5
1.Primary Outcome
Title Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Hide Description 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).
Time Frame Baseline and Week 126
Hide Outcome Measure Data
Hide Analysis Population Description
Week 126 Completers Population
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: Percent Change
-45.5  (31.35)
2.Secondary Outcome
Title Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Hide Description 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).
Time Frame Baseline and Week 174
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Percent Change
-51.0  (16.03)
3.Secondary Outcome
Title Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Hide Description 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).
Time Frame Baseline and Week 222
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
-58.5  (24.25)
4.Secondary Outcome
Title Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Hide Description 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).
Time Frame Baseline and Week 246
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: Percent Change
-60.1  (18.51)
5.Secondary Outcome
Title Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Hide Description 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).
Time Frame Baseline and Week 270
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: Percent Change
-74.0  (19.10)
6.Secondary Outcome
Title Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Hide Description 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).
Time Frame Baseline and Week 294
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: Percent Change
-51.1  (10.11)
7.Secondary Outcome
Title Percent Change in Total Cholesterol
Hide Description Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame Baseline and Week 126
Hide Outcome Measure Data
Hide Analysis Population Description
Week 126 Completers Population
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: Percent Change
-43.2  (25.35)
8.Secondary Outcome
Title Percent Change in Total Cholesterol
Hide Description Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame Baseline and Week 174
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Percent Change
-46.9  (15.21)
9.Secondary Outcome
Title Percent Change in Total Cholesterol
Hide Description Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame Baseline and Week 222
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
-51.0  (21.34)
10.Secondary Outcome
Title Percent Change in Total Cholesterol
Hide Description Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame Baseline and Week 246
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: Percent Change
-54.1  (16.88)
11.Secondary Outcome
Title Percent Change in Total Cholesterol
Hide Description Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame Baseline and Week 270
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: Percent Change
-65.2  (15.97)
12.Secondary Outcome
Title Percent Change in Total Cholesterol
Hide Description Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame Baseline and Week 294
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: Percent Change
-43.9  (5.01)
13.Secondary Outcome
Title Percent Change in Apolipoprotein B (Apo B)
Hide Description 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).
Time Frame Baseline and Week 126
Hide Outcome Measure Data
Hide Analysis Population Description
Week 126 Completers Population
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: Percent Change
-53.6  (23.74)
14.Secondary Outcome
Title Percent Change in Apolipoprotein B (Apo B)
Hide Description 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).
Time Frame Baseline and Week 174
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Percent Change
-59.4  (12.60)
15.Secondary Outcome
Title Percent Change in Apolipoprotein B (Apo B)
Hide Description 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).
Time Frame Baseline and Week 222
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
-65.1  (20.72)
16.Secondary Outcome
Title Percent Change in Apolipoprotein B (Apo B)
Hide Description 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).
Time Frame Baseline and Week 246
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: Percent Change
-65.9  (15.76)
17.Secondary Outcome
Title Percent Change in Apolipoprotein B (Apo B)
Hide Description 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).
Time Frame Baseline and Week 270
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: Percent Change
-76.7  (16.09)
18.Secondary Outcome
Title Percent Change in Apolipoprotein B (Apo B)
Hide Description 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).
Time Frame Baseline and Week 294
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: Percent Change
-60.9  (12.17)
19.Secondary Outcome
Title Percent Change in Triglycerides
Hide Description Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame Baseline and Week 126
Hide Outcome Measure Data
Hide Analysis Population Description
Week 126 Completers Population
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: Percent Change
-37.5  (42.52)
20.Secondary Outcome
Title Percent Change in Triglycerides
Hide Description Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame Baseline and Week 174
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Percent Change
-31.7  (37.09)
21.Secondary Outcome
Title Percent Change in Triglycerides
Hide Description Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame Baseline and Week 222
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
-27.6  (48.88)
22.Secondary Outcome
Title Percent Change in Triglycerides
Hide Description Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame Baseline and Week 246
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: Percent Change
-41.9  (33.16)
23.Secondary Outcome
Title Percent Change in Triglycerides
Hide Description Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame Baseline and Week 270
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: Percent Change
-48.5  (34.24)
24.Secondary Outcome
Title Percent Change in Triglycerides
Hide Description Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame Baseline and Week 294
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: Percent Change
29.2  (58.74)
25.Secondary Outcome
Title Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Hide Description 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).
Time Frame Baseline and Week 126
Hide Outcome Measure Data
Hide Analysis Population Description
Week 126 Completers Population
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: Percent Change
-47.1  (27.83)
26.Secondary Outcome
Title Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Hide Description 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).
Time Frame Baseline and Week 174
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Percent Change
-53.5  (16.42)
27.Secondary Outcome
Title Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Hide Description 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).
Time Frame Baseline and Week 222
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
-57.0  (24.07)
28.Secondary Outcome
Title Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Hide Description 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).
Time Frame Baseline and Week 246
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: Percent Change
-58.8  (17.73)
29.Secondary Outcome
Title Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Hide Description 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).
Time Frame Baseline and Week 270
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: Percent Change
-71.5  (17.65)
30.Secondary Outcome
Title Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Hide Description 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).
Time Frame Baseline and Week 294
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: Percent Change
-46.6  (5.19)
31.Secondary Outcome
Title Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Hide Description 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).
Time Frame Baseline and Week 126
Hide Outcome Measure Data
Hide Analysis Population Description
Week 126 Completers Population
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: Percent Change
-36.8  (43.90)
32.Secondary Outcome
Title Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Hide Description 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).
Time Frame Baseline and Week 174
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Percent Change
-31.5  (36.32)
33.Secondary Outcome
Title Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Hide Description 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).
Time Frame Baseline and Week 222
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
-26.3  (49.94)
34.Secondary Outcome
Title Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Hide Description 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).
Time Frame Baseline and Week 246
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: Percent Change
-41.4  (34.20)
35.Secondary Outcome
Title Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Hide Description 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).
Time Frame Baseline and Week 270
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: Percent Change
-48.7  (33.41)
36.Secondary Outcome
Title Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Hide Description 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).
Time Frame Baseline and Week 294
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: Percent Change
30.6  (59.94)
37.Secondary Outcome
Title Percent Change in Lp(a)
Hide Description Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Time Frame Baseline and Week 126
Hide Outcome Measure Data
Hide Analysis Population Description
Week 126 Completers Population
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Percent Change
5.5  (43.62)
38.Secondary Outcome
Title Percent Change in Lp(a)
Hide Description Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
Time Frame Baseline and Week 174
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
10.2  (60.64)
39.Secondary Outcome
Title Percent Change in Lp(a)
Hide Description Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
Time Frame Baseline and Week 222
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: Percent Change
-12.8  (49.00)
40.Secondary Outcome
Title Percent Change in Lp(a)
Hide Description Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
Time Frame Baseline and Week 246
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: Percent Change
3.4  (54.22)
41.Secondary Outcome
Title Percent Change in Lp(a)
Hide Description Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
Time Frame Baseline and Week 270
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: Percent Change
-6.6  (48.99)
42.Secondary Outcome
Title Percent Change in Lp(a)
Hide Description Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Time Frame Baseline and Week 294
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: Percent Change
-10.4  (35.64)
43.Secondary Outcome
Title Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Hide Description 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).
Time Frame Baseline and Week 126
Hide Outcome Measure Data
Hide Analysis Population Description
Week 126 Completers Population
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: Percent Change
-8.3  (19.28)
44.Secondary Outcome
Title Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Hide Description 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).
Time Frame Baseline and Week 174
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Percent Change
3.8  (26.51)
45.Secondary Outcome
Title Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Hide Description 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).
Time Frame Baseline and Week 222
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
-2.7  (21.22)
46.Secondary Outcome
Title Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Hide Description 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).
Time Frame Baseline and Week 246
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: Percent Change
-12.5  (19.17)
47.Secondary Outcome
Title Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Hide Description 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).
Time Frame Baseline and Week 270
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: Percent Change
-10.3  (27.68)
48.Secondary Outcome
Title Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Hide Description 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).
Time Frame Baseline and Week 294
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: Percent Change
-23.5  (2.27)
49.Secondary Outcome
Title Percent Change in Apolipoprotein AI (Apo AI)
Hide Description 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).
Time Frame Baseline and Week 126
Hide Outcome Measure Data
Hide Analysis Population Description
Week 126 Completers Population
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 17
Mean (Standard Deviation)
Unit of Measure: Percent Change
-14.0  (17.71)
50.Secondary Outcome
Title Percent Change in Apolipoprotein AI (Apo AI)
Hide Description 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).
Time Frame Baseline and Week 174
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: Percent Change
-8.2  (20.12)
51.Secondary Outcome
Title Percent Change in Apolipoprotein AI (Apo AI)
Hide Description 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).
Time Frame Baseline and Week 222
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 15
Mean (Standard Deviation)
Unit of Measure: Percent Change
-2.7  (33.30)
52.Secondary Outcome
Title Percent Change in Apolipoprotein AI (Apo AI)
Hide Description 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).
Time Frame Baseline and Week 246
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: Percent Change
-16.8  (26.62)
53.Secondary Outcome
Title Percent Change in Apolipoprotein AI (Apo AI)
Hide Description 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).
Time Frame Baseline and Week 270
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: Percent Change
-17.8  (20.12)
54.Secondary Outcome
Title Percent Change in Apolipoprotein AI (Apo AI)
Hide Description 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).
Time Frame Baseline and Week 294
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Lomitapide
Hide Arm/Group Description:

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 Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: Percent Change
-30.5  (22.2)
Time Frame Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Lomitapide
Hide Arm/Group Description

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

All-Cause Mortality
Lomitapide
Affected / at Risk (%)
Total   1/19 (5.26%) 
Hide Serious Adverse Events
Lomitapide
Affected / at Risk (%)
Total   7/19 (36.84%) 
Cardiac disorders   
Angina Pectoris  1  1/19 (5.26%) 
Aortic Value Incompetence  1  1/19 (5.26%) 
Coronary Artery Disease  1  1/19 (5.26%) 
Gastrointestinal disorders   
Diarrhoea  1  1/19 (5.26%) 
Reflux Oesophagitis  1  1/19 (5.26%) 
General disorders   
Sudden Cardiac Death  1  1/19 (5.26%) 
Hepatobiliary disorders   
Hepatotoxicity  1  1/19 (5.26%) 
Infections and infestations   
Lower Respiratory Tract Infection  1  1/19 (5.26%) 
Injury, poisoning and procedural complications   
Subdural Haematoma  1  1/19 (5.26%) 
Investigations   
International Normalized Ratio Increased  1  1/19 (5.26%) 
Metabolism and nutrition disorders   
Dehydration  1  1/19 (5.26%) 
Musculoskeletal and connective tissue disorders   
Rhabdomyolsis  1  1/19 (5.26%) 
Nervous system disorders   
Facial Palsy  1  1/19 (5.26%) 
Surgical and medical procedures   
Anticoagulant Therapy  1  1/19 (5.26%) 
Transfusion  1  1/19 (5.26%) 
Vascular disorders   
Arteriovenous Fistula  1  1/19 (5.26%) 
Hypovolaemic Shock  1  1/19 (5.26%) 
1
Term from vocabulary, MedDRA 11.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Lomitapide
Affected / at Risk (%)
Total   17/19 (89.47%) 
Blood and lymphatic system disorders   
Anaemia  1  2/19 (10.53%) 
Iron Deficiency Anaemia  1  1/19 (5.26%) 
Cardiac disorders   
Angina Pectoris  1  4/19 (21.05%) 
Aortic Valve Incompetence  1  1/19 (5.26%) 
Chest Pain  1  1/19 (5.26%) 
Coronary Artery Disease  1  1/19 (5.26%) 
Sinus Bradycardia  1  1/19 (5.26%) 
Ear and labyrinth disorders   
Tinnitus  1  1/19 (5.26%) 
Vertigo  1  1/19 (5.26%) 
Endocrine disorders   
Hypothyroidism  1  1/19 (5.26%) 
Eye disorders   
Blepharitis  1  1/19 (5.26%) 
Gastrointestinal disorders   
Abdominal Distension  1  3/19 (15.79%) 
Chapped Lips  1  1/19 (5.26%) 
Dental Caries  1  1/19 (5.26%) 
Diarrhoea  1  8/19 (42.11%) 
Dyspepsia  1  2/19 (10.53%) 
Epigastric Discomfort  1  2/19 (10.53%) 
Flatulence  1  1/19 (5.26%) 
Gastrooesophageal Reflux Disease  1  1/19 (5.26%) 
Gingival Bleeding  1  1/19 (5.26%) 
Haemorrhoidal Haemorrhage  1  1/19 (5.26%) 
Hiatus Hernia  1  1/19 (5.26%) 
Intestinal Mass  1  1/19 (5.26%) 
Nausea  1  6/19 (31.58%) 
Reflux Oesophagitis  1  1/19 (5.26%) 
Stomach Discomfort  1  1/19 (5.26%) 
Toothache  1  1/19 (5.26%) 
Vomiting  1  5/19 (26.32%) 
General disorders   
Asthenia  1  1/19 (5.26%) 
Chest Pain  1  1/19 (5.26%) 
Fatigue  1  1/19 (5.26%) 
Influenza  1  1/19 (5.26%) 
Oedema Peripheral  1  2/19 (10.53%) 
Pain  1  1/19 (5.26%) 
Pyrexia  1  1/19 (5.26%) 
Vestibulitis  1  1/19 (5.26%) 
Sudden Cardiac Death  1  1/19 (5.26%) 
Hepatobiliary disorders   
Hepatotoxicity  1  2/19 (10.53%) 
Immune system disorders   
Drug Hypersensitivity  1  1/19 (5.26%) 
Infections and infestations   
Bronchitis  1  3/19 (15.79%) 
Gastroenteritis  1  3/19 (15.79%) 
Gastrointestinal Infection  1  1/19 (5.26%) 
Influenza  1  5/19 (26.32%) 
Lower Respiratory Tract Infection  1  1/19 (5.26%) 
Nasopharyngitis  1  4/19 (21.05%) 
Sinusitus  1  3/19 (15.79%) 
Tooth Abscess  1  2/19 (10.53%) 
Upper Respiratory Tract Infection  1  1/19 (5.26%) 
Urinary Tract Infection  1  2/19 (10.53%) 
Viral Infection  1  1/19 (5.26%) 
Injury, poisoning and procedural complications   
Fall  1  1/19 (5.26%) 
Joint Injury  1  1/19 (5.26%) 
Post Procedural Diarrhoea  1  1/19 (5.26%) 
Procedural Headache  1  1/19 (5.26%) 
Skeletal Injury  1  1/19 (5.26%) 
Subdural Haematoma  1  1/19 (5.26%) 
Investigations   
Alanine Aminotransferase Increased  1  3/19 (15.79%) 
Asparate Aminotransferase Increased  1  2/19 (10.53%) 
Blood creatine phosphokinase Increased  1  1/19 (5.26%) 
Blood Potassium Increased  1  1/19 (5.26%) 
Blood Pressure Increased  1  1/19 (5.26%) 
Carotene Decreased  1  1/19 (5.26%) 
Carotid Bruit  1  1/19 (5.26%) 
International Normalized Ratio Decreased  1  1/19 (5.26%) 
International Normalized Ratio Increased  1  1/19 (5.26%) 
Liver Function Test Abnormal  1  1/19 (5.26%) 
Prothrombin Time Prolonged  1  1/19 (5.26%) 
Transaminases Increased  1  1/19 (5.26%) 
Vitamin K Decreased  1  1/19 (5.26%) 
Weight Decreased  1  2/19 (10.53%) 
White Blood Cell Count Decreased  1  1/19 (5.26%) 
Metabolism and nutrition disorders   
Decreased Appetite  1  1/19 (5.26%) 
Dehydration  1  1/19 (5.26%) 
Iron Deficiency  1  1/19 (5.26%) 
Oral Intake Reduced  1  1/19 (5.26%) 
Vitamin E Deficiency  1  1/19 (5.26%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  2/19 (10.53%) 
Back Pain  1  3/19 (15.79%) 
Musculoskeletal Pain  1  1/19 (5.26%) 
Neck Pain  1  1/19 (5.26%) 
Rhabdomyolysis  1  1/19 (5.26%) 
Tendonitis  1  2/19 (10.53%) 
Nervous system disorders   
Dizziness  1  1/19 (5.26%) 
Facial Palsy  1  1/19 (5.26%) 
Headache  1  6/19 (31.58%) 
Hypoaesthesia  1  2/19 (10.53%) 
Migraine  1  1/19 (5.26%) 
Paraesthesia  1  2/19 (10.53%) 
Sensory Disturbance  1  1/19 (5.26%) 
Syncope  1  1/19 (5.26%) 
Psychiatric disorders   
Anxiety  1  2/19 (10.53%) 
Depression  1  2/19 (10.53%) 
Stress  1  1/19 (5.26%) 
Renal and urinary disorders   
Nephrolithiasis  1  1/19 (5.26%) 
Reproductive system and breast disorders   
Dysmenorrhoea  1  1/19 (5.26%) 
Respiratory, thoracic and mediastinal disorders   
Apnoea  1  1/19 (5.26%) 
Cough  1  2/19 (10.53%) 
Dyspnoea  1  1/19 (5.26%) 
Dyspnoea Exertional  1  1/19 (5.26%) 
Epistaxis  1  1/19 (5.26%) 
Painful Respiration  1  1/19 (5.26%) 
Pharyngolaryngeal Pain  1  1/19 (5.26%) 
Sinus Congestion  1  1/19 (5.26%) 
Skin and subcutaneous tissue disorders   
Alopecia  1  1/19 (5.26%) 
Dry Skin  1  1/19 (5.26%) 
Hair growth abnormal  1  1/19 (5.26%) 
Scar  1  1/19 (5.26%) 
Surgical and medical procedures   
Anticoagulant Therapy  1  1/19 (5.26%) 
Transfusion  1  1/19 (5.26%) 
Vascular disorders   
Arterial Stenosis  1  1/19 (5.26%) 
Arteriovenous Fistula  1  1/19 (5.26%) 
Haematoma  1  1/19 (5.26%) 
Hypertension  1  1/19 (5.26%) 
Hypotension  1  1/19 (5.26%) 
Hypovolaemic Shock  1  1/19 (5.26%) 
1
Term from vocabulary, MedDRA 11.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Alison Long, MD - VP Clinical
Organization: Aegerion Pharmaceuticals, Inc.
Phone: 857-242-5142
EMail: alison.long@aegerion.com
Publications:
Cuchel M, Meagher E, Marais AD, et.al. Abstract 1077: A phase III study of microsomal triglyceride transfer protein inhibitor lomitapide (AEGR-733) in patients with homozygous familial hypercholesterolemia: interim results at 6 months. Circulation, Nov 2009; 120: S441
Layout table for additonal information
Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT00943306    
Other Study ID Numbers: AEGR-733-012
First Submitted: July 21, 2009
First Posted: July 22, 2009
Results First Submitted: February 29, 2016
Results First Posted: May 11, 2018
Last Update Posted: June 13, 2018