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Familial HypercholesterolemiaPhase 3NCT00943306

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

A Phase 3 study testing lomitapide in people with Familial Hypercholesterolemia, run by Aegerion Pharmaceuticals, Inc. at 10 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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 more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Familial Hypercholesterolemia
What is being tested?
lomitapide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
10 sitesin 4 countries
Study dates
29 Oct 2009to 1 Dec 2014
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

10 sites in 4 countries.

10 sites in 4 countries

Italy4 sites

  • Medicina Interna Universitaria

    Ferrara, Sicily

  • Centro Universitario Dislipidemie

    Milan

  • Dipartimento di Medicina Clinica e Delle Patologie Emergenti

    Palermo, Sicily

  • DAI Ematologia, Oncologia, Anatomia Patologica e Medicina

    Roma

Other trials in Italy

United States2 sites

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

Other trials in United States

Canada2 sites

  • Lipid Clinic and University of Montreal Community Genomic Medicine Center

    Chicoutimi, Quebec, G7H 5H6

  • Robarts Research Institute

    London, Ontario, N6A 5K8

Other trials in Canada

South Africa2 sites

  • Cardiology Research

    Bloemfontein, 9300

  • University of Capetown

    Cape Town, 7925

Other trials in South Africa

About this study

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

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).

Being tested
lomitapide
Keywords
Homozygous, Familial, Hypercholesterolemia, MTP, Apheresis, lomitapide, Homozygous Familial Hypercholesterolemia (HoFH)
Medical subject headings
Hyperlipoproteinemia Type II, Hypercholesterolemia, Homozygous Familial Hypercholesterolemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • lomitapideDrug

    Also called AEGR-733, BMS-201038

    5-60 mg po every day

Study groups in NCT00943306
Study groupRoleWhat they receive
lomitapideExperimentalMaximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Receives: Drug: lomitapide

What is being measured

The primary outcome is the main question the study is designed to answer.

  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).

    Measured: Baseline and Week 126

Secondary outcomes (53)

  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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: 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).

    Measured: Baseline and Week 294

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Jul 2009 (estimated)
  2. Study start29 Oct 2009
  3. Primary completion17 Sep 2012
  4. Study completion1 Dec 2014

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00943306
Sponsor's protocol ID
AEGR-733-012
Status last verified
May 2018
First submitted
21 Jul 2009
Last updated
13 Jun 2018
Data monitoring committee
No

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

  • Larrey D, D'Erasmo L, O'Brien S, Arca M; Italian Working Group on Lomitapide. Long-term hepatic safety of lomitapide in homozygous familial hypercholesterolaemia. Liver Int. 2023 Feb;43(2):413-423. doi: 10.1111/liv.15497. Epub 2022 Dec 30.

    PubMed 36520008

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing lomitapide in people with Familial Hypercholesterolemia, run by Aegerion Pharmaceuticals, Inc. at 10 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in Italy, United States, Canada, South Africa. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 May 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) (time frame: Baseline and Week 126). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00943306, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jun 2018.

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