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Completed

NCT Number: NCT00943306

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

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.

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Key information

Conditions

Familial Hypercholesterolemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Homozygous Familial Hypercholesterolemia Hypercholesterolemia Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Robarts Research Institute, London, Ontario, Canada

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About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Completed UP1002 or 733-005.
  • Willing and able to provide consent and comply with the requirements of the study protocol.

Exclusion criteria

  • Met any of the stopping rules for study discontinuation at the final visit of study UP1002 or 733-005.

Treatment and study plan

lomitapide

Drug

5-60 mg po every day

Other names: AEGR-733, BMS-201038

Primary outcomes

  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 outcomes

  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).

Sponsors and collaborators

Lead sponsor

Aegerion Pharmaceuticals, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2009
Primary completion
2012
Study completion
2014
First posted
Jul 22, 2009
Registry last updated
Jun 13, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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