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CompletedNCT00943306Updated Jun 13, 2018Results posted

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

A Phase 3 interventional study of lomitapide in Familial Hypercholesterolemia, sponsored by Aegerion Pharmaceuticals, Inc.. Completed at 10 sites in 4 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-06-13.

Primary conditionHomozygous Familial HypercholesterolemiaRegistry: Hyperlipoproteinemia Type IIReclassifiedWhy we changed it
Registry
Hyperlipoproteinemia Type II — the first condition term the registry derived for this study.
ClinicalTrials.gg
Homozygous Familial Hypercholesterolemia
Why
  1. The study's title name Homozygous Familial Hypercholesterolemia, a narrower term within Hyperlipoproteinemia Type II.
Confidence
High: the new condition is named in the study's own title. Of 46 hand-checked studies reclassified this way, 72% were exactly right and 89% in the right disease family; the registry's first term was right for 4% (measured 2026-10-21). How we classify →
Wrong?
Tell us the study's main condition →

Sponsored by Aegerion Pharmaceuticals, Inc. (part of Chiesi Farmaceutici S.p.A.) · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
19
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study 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.

Read the 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).

02

Conditions studied

  • Familial Hypercholesterolemia

Keywords

  • Homozygous
  • Familial
  • Hypercholesterolemia
  • MTP
  • Apheresis
  • lomitapide
  • Homozygous Familial Hypercholesterolemia (HoFH)
03

In context

Homozygous Familial Hypercholesterolemia Reclassified

68 studies on the registry are indexed under Homozygous Familial Hypercholesterolemia; 13 are open to participants now.

This study's enrollment of 19 is close to the median of 21 across 51 interventional studies indexed under Homozygous Familial Hypercholesterolemia.

Browse Homozygous Familial Hypercholesterolemia studies →

Lead sponsor

Aegerion Pharmaceuticals, Inc. is the lead sponsor of 17 studies on the registry; none are open to participants now.

First submitted before Aegerion Pharmaceuticals, Inc. became part of Chiesi Farmaceutici S.p.A..

Browse Aegerion Pharmaceuticals, Inc. studies →All Chiesi Farmaceutici S.p.A. studies →

Compared within Homozygous Familial Hypercholesterolemia, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Hyperlipoproteinemia Type II. The counts are of registry records indexed under each term, refreshed daily. How we classify →

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

Exclusion Criteria:

  1. Met any of the stopping rules for study discontinuation at the final visit of study UP1002 or 733-005.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
19 participants (actual)

Study arms

  • Experimental
    lomitapide

    Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.

    Drug: lomitapide

Interventions

  • Druglomitapide

    5-60 mg po every day

    Also known as: AEGR-733, BMS-201038

06

What researchers measure

Primary outcomes

  1. 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

  1. 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

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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

  10. 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

  11. 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

  12. 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

  13. 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

  14. 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

  15. 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

  16. 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

  17. 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

  18. 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

  19. 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

  20. 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

  21. 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

  22. 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

  23. 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

  24. 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

  25. 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

  26. 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

  27. 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

  28. 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

  29. 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

  30. 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

  31. 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

  32. 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

  33. 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

  34. 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

  35. 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

  36. 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

  37. 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

  38. 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

  39. 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

  40. 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

  41. 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

  42. 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

  43. 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

  44. 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

  45. 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

  46. 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

  47. 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

  48. 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

  49. 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

  50. 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

  51. 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

  52. 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

  53. 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

07

Results

Posted May 11, 2018

Participant flow

Participant flow — Overall Study
MilestoneLomitapide
Started19
Completed16
Not completed3
Withdrew: Physician decision1
Withdrew: Death1
Withdrew: Sponsor decision due to non-compliance1

Outcome measures

PrimaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Percent ChangeLomitapide
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)-45.5 ± 31.35
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Percent ChangeLomitapide
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)-51.0 ± 16.03
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Percent ChangeLomitapide
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)-58.5 ± 24.25
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Percent ChangeLomitapide
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)-60.1 ± 18.51
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Percent ChangeLomitapide
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)-74.0 ± 19.10
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Percent ChangeLomitapide
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)-51.1 ± 10.11
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Total Cholesterol
Percent ChangeLomitapide
Percent Change in Total Cholesterol-43.2 ± 25.35
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Total Cholesterol
Percent ChangeLomitapide
Percent Change in Total Cholesterol-46.9 ± 15.21
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Total Cholesterol
Percent ChangeLomitapide
Percent Change in Total Cholesterol-51.0 ± 21.34
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Total Cholesterol
Percent ChangeLomitapide
Percent Change in Total Cholesterol-54.1 ± 16.88
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Total Cholesterol
Percent ChangeLomitapide
Percent Change in Total Cholesterol-65.2 ± 15.97
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Total Cholesterol
Percent ChangeLomitapide
Percent Change in Total Cholesterol-43.9 ± 5.01
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein B (Apo B)
Percent ChangeLomitapide
Percent Change in Apolipoprotein B (Apo B)-53.6 ± 23.74
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein B (Apo B)
Percent ChangeLomitapide
Percent Change in Apolipoprotein B (Apo B)-59.4 ± 12.60
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein B (Apo B)
Percent ChangeLomitapide
Percent Change in Apolipoprotein B (Apo B)-65.1 ± 20.72
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein B (Apo B)
Percent ChangeLomitapide
Percent Change in Apolipoprotein B (Apo B)-65.9 ± 15.76
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein B (Apo B)
Percent ChangeLomitapide
Percent Change in Apolipoprotein B (Apo B)-76.7 ± 16.09
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein B (Apo B)
Percent ChangeLomitapide
Percent Change in Apolipoprotein B (Apo B)-60.9 ± 12.17
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Triglycerides
Percent ChangeLomitapide
Percent Change in Triglycerides-37.5 ± 42.52
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Triglycerides
Percent ChangeLomitapide
Percent Change in Triglycerides-31.7 ± 37.09
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Triglycerides
Percent ChangeLomitapide
Percent Change in Triglycerides-27.6 ± 48.88
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Triglycerides
Percent ChangeLomitapide
Percent Change in Triglycerides-41.9 ± 33.16
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Triglycerides
Percent ChangeLomitapide
Percent Change in Triglycerides-48.5 ± 34.24
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Triglycerides
Percent ChangeLomitapide
Percent Change in Triglycerides29.2 ± 58.74
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Percent ChangeLomitapide
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)-47.1 ± 27.83
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Percent ChangeLomitapide
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)-53.5 ± 16.42
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Percent ChangeLomitapide
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)-57.0 ± 24.07
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Percent ChangeLomitapide
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)-58.8 ± 17.73
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Percent ChangeLomitapide
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)-71.5 ± 17.65
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Percent ChangeLomitapide
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)-46.6 ± 5.19
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Percent ChangeLomitapide
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)-36.8 ± 43.90
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Percent ChangeLomitapide
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)-31.5 ± 36.32
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Percent ChangeLomitapide
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)-26.3 ± 49.94
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Percent ChangeLomitapide
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)-41.4 ± 34.20
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Percent ChangeLomitapide
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)-48.7 ± 33.41
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Percent ChangeLomitapide
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)30.6 ± 59.94
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Lp(a)
Percent ChangeLomitapide
Percent Change in Lp(a)5.5 ± 43.62
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Lp(a)
Percent ChangeLomitapide
Percent Change in Lp(a)10.2 ± 60.64
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Lp(a)
Percent ChangeLomitapide
Percent Change in Lp(a)-12.8 ± 49.00
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Lp(a)
Percent ChangeLomitapide
Percent Change in Lp(a)3.4 ± 54.22
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Lp(a)
Percent ChangeLomitapide
Percent Change in Lp(a)-6.6 ± 48.99
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Lp(a)
Percent ChangeLomitapide
Percent Change in Lp(a)-10.4 ± 35.64
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Percent ChangeLomitapide
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)-8.3 ± 19.28
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Percent ChangeLomitapide
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)3.8 ± 26.51
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Percent ChangeLomitapide
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)-2.7 ± 21.22
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Percent ChangeLomitapide
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)-12.5 ± 19.17
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Percent ChangeLomitapide
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)-10.3 ± 27.68
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Percent ChangeLomitapide
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)-23.5 ± 2.27
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein AI (Apo AI)
Percent ChangeLomitapide
Percent Change in Apolipoprotein AI (Apo AI)-14.0 ± 17.71
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein AI (Apo AI)
Percent ChangeLomitapide
Percent Change in Apolipoprotein AI (Apo AI)-8.2 ± 20.12
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein AI (Apo AI)
Percent ChangeLomitapide
Percent Change in Apolipoprotein AI (Apo AI)-2.7 ± 33.30
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein AI (Apo AI)
Percent ChangeLomitapide
Percent Change in Apolipoprotein AI (Apo AI)-16.8 ± 26.62
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein AI (Apo AI)
Percent ChangeLomitapide
Percent Change in Apolipoprotein AI (Apo AI)-17.8 ± 20.12
SecondaryPercent 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
Reported as:
Mean · Percent Change
Percent Change in Apolipoprotein AI (Apo AI)
Percent ChangeLomitapide
Percent Change in Apolipoprotein AI (Apo AI)-30.5 ± 22.2

Adverse events

Collected over Week 78 of Study 733-005/UP1002 to Week 294 of Study 733-005/UP1002 (Week 216 of Study AEGR-733-012). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Lomitapide1/19 (5.3%)7/19 (36.8%)17/19 (89.5%)
Most frequent serious events
Showing 10 of 17
Most frequent serious events
EventLomitapide
Angina PectorisCardiac disorders1/19
Aortic Value IncompetenceCardiac disorders1/19
Coronary Artery DiseaseCardiac disorders1/19
DiarrhoeaGastrointestinal disorders1/19
Reflux OesophagitisGastrointestinal disorders1/19
Sudden Cardiac DeathGeneral disorders1/19
HepatotoxicityHepatobiliary disorders1/19
Lower Respiratory Tract InfectionInfections and infestations1/19
Subdural HaematomaInjury, poisoning and procedural complications1/19
International Normalized Ratio IncreasedInvestigations1/19
Most frequent other events
Showing 10 of 115
Most frequent other events
EventLomitapide
DiarrhoeaGastrointestinal disorders8/19
NauseaGastrointestinal disorders6/19
HeadacheNervous system disorders6/19
VomitingGastrointestinal disorders5/19
InfluenzaInfections and infestations5/19
Angina PectorisCardiac disorders4/19
NasopharyngitisInfections and infestations4/19
Abdominal DistensionGastrointestinal disorders3/19
BronchitisInfections and infestations3/19
GastroenteritisInfections and infestations3/19

Baseline characteristics

Baseline from initiation of treatment in Study 733-005/UP1002 (Safety Population)

Age, Categorical
Age, Categorical(Participants)Lomitapide
<=18 years0
Between 18 and 65 years19
>=65 years0
Age, Continuous
Age, Continuous(years)Lomitapide
Mean30.4 ± 11.74
Sex: Female, Male
Sex: Female, Male(Participants)Lomitapide
Female9
Male10
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Lomitapide
American Indian or Alaska Native0
Asian1
Native Hawaiian or Other Pacific Islander0
Black or African American0
White17
More than one race0
Unknown or Not Reported1
Region of Enrollment
Region of Enrollment(participants)Lomitapide
Canada2
United States3
South Africa9
Italy5
08

Study locations

10 sites
  • Cedars-Sinai Medical Center
    Los Angeles, California 90048, United States
  • University of Pennsylvania
    Philadelphia, Pennsylvania 19104, United States
  • Robarts Research Institute
    London, Ontario N6A 5K8, Canada
  • Lipid Clinic and University of Montreal Community Genomic Medicine Center
    Chicoutimi, Quebec G7H 5H6, Canada
  • 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
  • Cardiology Research
    Bloemfontein, 9300, South Africa
  • University of Capetown
    Cape town, 7925, South Africa
09

References and documents

Publications

  • Cuchel M, Bloedon LT, Szapary PO, Kolansky DM, Wolfe ML, Sarkis A, Millar JS, Ikewaki K, Siegelman ES, Gregg RE, Rader DJ. Inhibition of microsomal triglyceride transfer protein in familial hypercholesterolemia. N Engl J Med. 2007 Jan 11;356(2):148-56. doi: 10.1056/NEJMoa061189. PubMed 17215532 ↗
  • 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
10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 13, 2018, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT00943306
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Jul 22, 2009
Start date
Oct 29, 2009
Primary completion
Sep 17, 2012
Completion
Dec 1, 2014
Results posted
May 11, 2018
Last update
Jun 13, 2018

Study contacts

Marina Cuchel, MD, PhD
principal investigator · University of Pennsylvania
Mark Sumeray, MD
study chair · Aegerion Pharmaceuticals, Inc.

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: BMS201038

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