Familial Hypercholesterolemia
Conditions
Keywords
Homozygous, Familial, Hypercholesterolemia, MTP, Apheresis, lomitapide, Homozygous Familial Hypercholesterolemia (HoFH)
Brief summary
This was a long term follow on study to assess the continued long term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia.
Detailed description
This was a phase III open label clinical trial to evaluate the long-term efficacy and safety of lomitapide at the maximum tolerated dose (for each patient) established during the clinical trial 733-005/UP1002. Subjects completing the 78-week treatment period in study 733-005/UP1002 who have not met any of the stopping criteria will be eligible to participate in 733-012. The treatment period continued until a decision was made by the local competent authority regarding marketing authorization. Lomitapide will be given orally once daily. Patient specific doses will be carried forward from 733-005/UP1002, but will not exceed the maximum tolerated dose the patient received during 733-005 /UP1002. The maximum dose for any patient was 80 mg/day. There was no reference therapy in this trial. The effects of the study drug were compared to baseline data (from 733-005/UP1002). Concomitant lipid-lowering therapy including plasmapheresis or LDL apheresis is permitted. Note that hereafter, Week 0 in Study 733-005/UP1002 will be referred to as Baseline and Week 48 in Study AEGR-733-012 will be referred to as Week 126 (of overall treatment).
Interventions
DRUGlomitapide
5-60 mg po every day
Sponsors
Aegerion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Completed UP1002 or 733-005. 2. Willing and able to provide consent and comply with the requirements of the study protocol.
Exclusion criteria
1. Met any of the stopping rules for study discontinuation at the final visit of study UP1002 or 733-005.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 126 | 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). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in Total Cholesterol | Baseline and Week 126 | Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012). |
| Percent Change in Apolipoprotein B (Apo B) | Baseline and Week 126 | 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). |
| Percent Change in Triglycerides | Baseline and Week 126 | Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012). |
| Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) | Baseline and Week 126 | 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). |
| Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | Baseline and Week 174 | 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). |
| Percent Change in Lp(a) | Baseline and Week 126 | Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012). |
| Percent Change in High Density Lipoprotein Cholesterol (HDL-C) | Baseline and Week 126 | 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). |
| Percent Change in Apolipoprotein AI (Apo AI) | Baseline and Week 126 | 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). |
| Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) | Baseline and Week 126 | 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). |
Countries
Canada, Italy, South Africa, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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 | 19 |
| Total | 19 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 1 |
| Overall Study | Physician Decision | 1 |
| Overall Study | Sponsor decision due to non-compliance | 1 |
Baseline characteristics
| Characteristic | Lomitapide |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 19 Participants |
| Age, Continuous | 30.4 years STANDARD_DEVIATION 11.74 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) White | 17 Participants |
| Region of Enrollment Canada | 2 participants |
| Region of Enrollment Italy | 5 participants |
| Region of Enrollment South Africa | 9 participants |
| Region of Enrollment United States | 3 participants |
| Sex: Female, Male Female | 9 Participants |
| Sex: Female, Male Male | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 19 |
| other Total, other adverse events | 17 / 19 |
| serious Total, serious adverse events | 7 / 19 |
Outcome results
Primary
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
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
Population: Week 126 Completers Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | -45.5 Percent Change | Standard Deviation 31.35 |
Secondary
Percent Change in Apolipoprotein AI (Apo AI)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein AI (Apo AI) | -30.5 Percent Change | Standard Deviation 22.2 |
Secondary
Percent Change in Apolipoprotein AI (Apo AI)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein AI (Apo AI) | -17.8 Percent Change | Standard Deviation 20.12 |
Secondary
Percent Change in Apolipoprotein AI (Apo AI)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein AI (Apo AI) | -16.8 Percent Change | Standard Deviation 26.62 |
Secondary
Percent Change in Apolipoprotein AI (Apo AI)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein AI (Apo AI) | -2.7 Percent Change | Standard Deviation 33.3 |
Secondary
Percent Change in Apolipoprotein AI (Apo AI)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein AI (Apo AI) | -8.2 Percent Change | Standard Deviation 20.12 |
Secondary
Percent Change in Apolipoprotein AI (Apo AI)
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
Population: Week 126 Completers Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein AI (Apo AI) | -14.0 Percent Change | Standard Deviation 17.71 |
Secondary
Percent Change in Apolipoprotein B (Apo B)
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
Population: Week 126 Completers Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein B (Apo B) | -53.6 Percent Change | Standard Deviation 23.74 |
Secondary
Percent Change in Apolipoprotein B (Apo B)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein B (Apo B) | -60.9 Percent Change | Standard Deviation 12.17 |
Secondary
Percent Change in Apolipoprotein B (Apo B)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein B (Apo B) | -76.7 Percent Change | Standard Deviation 16.09 |
Secondary
Percent Change in Apolipoprotein B (Apo B)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein B (Apo B) | -65.9 Percent Change | Standard Deviation 15.76 |
Secondary
Percent Change in Apolipoprotein B (Apo B)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein B (Apo B) | -65.1 Percent Change | Standard Deviation 20.72 |
Secondary
Percent Change in Apolipoprotein B (Apo B)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Apolipoprotein B (Apo B) | -59.4 Percent Change | Standard Deviation 12.6 |
Secondary
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in High Density Lipoprotein Cholesterol (HDL-C) | -23.5 Percent Change | Standard Deviation 2.27 |
Secondary
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
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
Population: Week 126 Completers Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in High Density Lipoprotein Cholesterol (HDL-C) | -8.3 Percent Change | Standard Deviation 19.28 |
Secondary
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in High Density Lipoprotein Cholesterol (HDL-C) | 3.8 Percent Change | Standard Deviation 26.51 |
Secondary
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in High Density Lipoprotein Cholesterol (HDL-C) | -2.7 Percent Change | Standard Deviation 21.22 |
Secondary
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in High Density Lipoprotein Cholesterol (HDL-C) | -12.5 Percent Change | Standard Deviation 19.17 |
Secondary
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in High Density Lipoprotein Cholesterol (HDL-C) | -10.3 Percent Change | Standard Deviation 27.68 |
Secondary
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | -51.1 Percent Change | Standard Deviation 10.11 |
Secondary
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | -60.1 Percent Change | Standard Deviation 18.51 |
Secondary
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | -58.5 Percent Change | Standard Deviation 24.25 |
Secondary
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | -74.0 Percent Change | Standard Deviation 19.1 |
Secondary
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) | -51.0 Percent Change | Standard Deviation 16.03 |
Secondary
Percent Change in Lp(a)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Lp(a) | -10.4 Percent Change | Standard Deviation 35.64 |
Secondary
Percent Change in Lp(a)
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
Population: Week 126 Completers Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Lp(a) | 5.5 Percent Change | Standard Deviation 43.62 |
Secondary
Percent Change in Lp(a)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Lp(a) | 10.2 Percent Change | Standard Deviation 60.64 |
Secondary
Percent Change in Lp(a)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Lp(a) | -12.8 Percent Change | Standard Deviation 49 |
Secondary
Percent Change in Lp(a)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Lp(a) | 3.4 Percent Change | Standard Deviation 54.22 |
Secondary
Percent Change in Lp(a)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Lp(a) | -6.6 Percent Change | Standard Deviation 48.99 |
Secondary
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
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
Population: Week 126 Completers Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) | -47.1 Percent Change | Standard Deviation 27.83 |
Secondary
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) | -53.5 Percent Change | Standard Deviation 16.42 |
Secondary
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) | -57.0 Percent Change | Standard Deviation 24.07 |
Secondary
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) | -58.8 Percent Change | Standard Deviation 17.73 |
Secondary
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) | -71.5 Percent Change | Standard Deviation 17.65 |
Secondary
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) | -46.6 Percent Change | Standard Deviation 5.19 |
Secondary
Percent Change in Total Cholesterol
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
Population: Week 126 Completers Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Total Cholesterol | -43.2 Percent Change | Standard Deviation 25.35 |
Secondary
Percent Change in Total Cholesterol
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Total Cholesterol | -54.1 Percent Change | Standard Deviation 16.88 |
Secondary
Percent Change in Total Cholesterol
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Total Cholesterol | -65.2 Percent Change | Standard Deviation 15.97 |
Secondary
Percent Change in Total Cholesterol
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Total Cholesterol | -43.9 Percent Change | Standard Deviation 5.01 |
Secondary
Percent Change in Total Cholesterol
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Total Cholesterol | -46.9 Percent Change | Standard Deviation 15.21 |
Secondary
Percent Change in Total Cholesterol
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Total Cholesterol | -51.0 Percent Change | Standard Deviation 21.34 |
Secondary
Percent Change in Triglycerides
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
Population: Week 126 Completers Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Triglycerides | -37.5 Percent Change | Standard Deviation 42.52 |
Secondary
Percent Change in Triglycerides
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Triglycerides | -27.6 Percent Change | Standard Deviation 48.88 |
Secondary
Percent Change in Triglycerides
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Triglycerides | -41.9 Percent Change | Standard Deviation 33.16 |
Secondary
Percent Change in Triglycerides
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Triglycerides | -48.5 Percent Change | Standard Deviation 34.24 |
Secondary
Percent Change in Triglycerides
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Triglycerides | 29.2 Percent Change | Standard Deviation 58.74 |
Secondary
Percent Change in Triglycerides
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Triglycerides | -31.7 Percent Change | Standard Deviation 37.09 |
Secondary
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) | -48.7 Percent Change | Standard Deviation 33.41 |
Secondary
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) | -41.4 Percent Change | Standard Deviation 34.2 |
Secondary
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) | -26.3 Percent Change | Standard Deviation 49.94 |
Secondary
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) | -31.5 Percent Change | Standard Deviation 36.32 |
Secondary
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
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
Population: Week 126 Completers Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) | -36.8 Percent Change | Standard Deviation 43.9 |
Secondary
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
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
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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Lomitapide | Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) | 30.6 Percent Change | Standard Deviation 59.94 |