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

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ClinicalTrials.gov Identifier: NCT00943306
Recruitment Status : Completed
First Posted : July 22, 2009
Results First Posted : May 10, 2018
Last Update Posted : June 13, 2018
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Familial Hypercholesterolemia Drug: lomitapide Phase 3

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).


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor 'Lomitapide' (LOMITAPIDE) in Patients With Homozygous Familial Hypercholesterolemia
Actual Study Start Date : October 29, 2009
Actual Primary Completion Date : September 17, 2012
Actual Study Completion Date : December 1, 2014

Resource links provided by the National Library of Medicine

Drug Information available for: Lomitapide

Arm Intervention/treatment
Experimental: lomitapide
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Drug: lomitapide
5-60 mg po every day
Other Names:
  • AEGR-733
  • BMS-201038




Primary Outcome Measures :
  1. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: 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 :
  1. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: 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).

  2. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: Baseline and Week 222 ]
    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).

  3. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: Baseline and Week 246 ]
    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).

  4. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: Baseline and Week 270 ]
    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).

  5. Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: 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).

  6. Percent Change in Total Cholesterol [ Time Frame: 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).

  7. Percent Change in Total Cholesterol [ Time Frame: Baseline and Week 174 ]
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

  8. Percent Change in Total Cholesterol [ Time Frame: Baseline and Week 222 ]
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

  9. Percent Change in Total Cholesterol [ Time Frame: Baseline and Week 246 ]
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

  10. Percent Change in Total Cholesterol [ Time Frame: Baseline and Week 270 ]
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

  11. Percent Change in Total Cholesterol [ Time Frame: 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).

  12. Percent Change in Apolipoprotein B (Apo B) [ Time Frame: 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).

  13. Percent Change in Apolipoprotein B (Apo B) [ Time Frame: Baseline and Week 174 ]
    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).

  14. Percent Change in Apolipoprotein B (Apo B) [ Time Frame: Baseline and Week 222 ]
    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).

  15. Percent Change in Apolipoprotein B (Apo B) [ Time Frame: Baseline and Week 246 ]
    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).

  16. Percent Change in Apolipoprotein B (Apo B) [ Time Frame: Baseline and Week 270 ]
    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).

  17. Percent Change in Apolipoprotein B (Apo B) [ Time Frame: 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).

  18. Percent Change in Triglycerides [ Time Frame: 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).

  19. Percent Change in Triglycerides [ Time Frame: Baseline and Week 174 ]
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

  20. Percent Change in Triglycerides [ Time Frame: Baseline and Week 222 ]
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

  21. Percent Change in Triglycerides [ Time Frame: Baseline and Week 246 ]
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

  22. Percent Change in Triglycerides [ Time Frame: Baseline and Week 270 ]
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

  23. Percent Change in Triglycerides [ Time Frame: 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).

  24. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: 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).

  25. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: Baseline and Week 174 ]
    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).

  26. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: Baseline and Week 222 ]
    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).

  27. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: Baseline and Week 246 ]
    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).

  28. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: Baseline and Week 270 ]
    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).

  29. Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: 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).

  30. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: 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).

  31. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: Baseline and Week 174 ]
    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).

  32. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: Baseline and Week 222 ]
    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).

  33. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: Baseline and Week 246 ]
    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).

  34. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: Baseline and Week 270 ]
    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).

  35. Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: 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).

  36. Percent Change in Lp(a) [ Time Frame: 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).

  37. Percent Change in Lp(a) [ Time Frame: Baseline and Week 174 ]
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).

  38. Percent Change in Lp(a) [ Time Frame: Baseline and Week 222 ]
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).

  39. Percent Change in Lp(a) [ Time Frame: Baseline and Week 246 ]
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).

  40. Percent Change in Lp(a) [ Time Frame: Baseline and Week 270 ]
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).

  41. Percent Change in Lp(a) [ Time Frame: 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).

  42. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: 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).

  43. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: Baseline and Week 174 ]
    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).

  44. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: Baseline and Week 222 ]
    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).

  45. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: Baseline and Week 246 ]
    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).

  46. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: Baseline and Week 270 ]
    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).

  47. Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: 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).

  48. Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: 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).

  49. Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: Baseline and Week 174 ]
    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).

  50. Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: Baseline and Week 222 ]
    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).

  51. Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: Baseline and Week 246 ]
    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).

  52. Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: Baseline and Week 270 ]
    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).

  53. Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: 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).



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00943306


Locations
United States, California
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
United States, Pennsylvania
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Canada, Ontario
Robarts Research Institute
London, Ontario, Canada, N6A 5K8
Canada, Quebec
Lipid Clinic and University of Montreal Community Genomic Medicine Center
Chicoutimi, Quebec, Canada, G7H 5H6
Italy
Medicina Interna Universitaria
Ferrara, Sicily, Italy
Dipartimento di Medicina Clinica e Delle Patologie Emergenti
Palermo, Sicily, Italy
Centro Universitario Dislipidemie
Milano, Italy
DAI Ematologia, Oncologia, Anatomia Patologica e Medicina
Roma, Italy
South Africa
Cardiology Research
Bloemfontein, South Africa, 9300
University of Capetown
Cape town, South Africa, 7925
Sponsors and Collaborators
Aegerion Pharmaceuticals, Inc.
Investigators
Principal Investigator: Marina Cuchel, MD, PhD University of Pennsylvania
Study Chair: Mark Sumeray, MD Aegerion Pharmaceuticals, Inc.

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

Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT00943306     History of Changes
Other Study ID Numbers: AEGR-733-012
First Posted: July 22, 2009    Key Record Dates
Results First Posted: May 10, 2018
Last Update Posted: June 13, 2018
Last Verified: May 2018

Keywords provided by Aegerion Pharmaceuticals, Inc.:
Homozygous
Familial
Hypercholesterolemia
MTP
Apheresis
lomitapide
Homozygous Familial Hypercholesterolemia (HoFH)

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias