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

Aegerion Pharmaceuticals, Inc.19 enrolled

Overview

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.

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).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Familial Hypercholesterolemia

Interventions

lomitapideDRUG

5-60 mg po every day

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (10)

Cedars-Sinai Medical Center

Los Angeles, California, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Robarts Research Institute

London, Ontario, Canada

Outcomes

Primary Outcomes

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

Secondary Outcomes

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

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

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

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

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

Data from ClinicalTrials.gov

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Lipid Clinic and University of Montreal Community Genomic Medicine Center

Chicoutimi, Quebec, Canada

Medicina Interna Universitaria

Ferrara, Sicily, Italy

Dipartimento di Medicina Clinica e Delle Patologie Emergenti

Palermo, Sicily, Italy

Centro Universitario Dislipidemie

Milan, Italy

DAI Ematologia, Oncologia, Anatomia Patologica e Medicina

Roma, Italy

Cardiology Research

Bloemfontein, South Africa

University of Capetown

Cape Town, South Africa

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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