Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
- Registration Number
- NCT00943306
- Lead Sponsor
- Aegerion Pharmaceuticals, Inc.
- 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).
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 19
- Completed UP1002 or 733-005.
- Willing and able to provide consent and comply with the requirements of the study protocol.
- Met any of the stopping rules for study discontinuation at the final visit of study UP1002 or 733-005.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description lomitapide lomitapide Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method Percent Change in Apolipoprotein B (Apo B) Baseline and Week 294 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).
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) Baseline and Week 294 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).
Percent Change in Apolipoprotein AI (Apo AI) Baseline and Week 294 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).
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) Baseline and Week 294 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).
Percent Change in Lp(a) Baseline and Week 294 Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Percent Change in Triglycerides Baseline and Week 294 Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) Baseline and Week 294 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).
Percent Change in Total Cholesterol Baseline and Week 294 Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
Percent Change in High Density Lipoprotein Cholesterol (HDL-C) Baseline and Week 294 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).
Trial Locations
- Locations (10)
Robarts Research Institute
🇨🇦London, Ontario, Canada
Lipid Clinic and University of Montreal Community Genomic Medicine Center
🇨🇦Chicoutimi, Quebec, Canada
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
Dipartimento di Medicina Clinica e Delle Patologie Emergenti
🇮🇹Palermo, Sicily, Italy
University of Capetown
🇿🇦Cape town, South Africa
Medicina Interna Universitaria
🇮🇹Ferrara, Sicily, Italy
Centro Universitario Dislipidemie
🇮🇹Milano, Italy
DAI Ematologia, Oncologia, Anatomia Patologica e Medicina
🇮🇹Roma, Italy
Cardiology Research
🇿🇦Bloemfontein, South Africa