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

Primary Purpose

Familial Hypercholesterolemia

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
lomitapide
Sponsored by
Aegerion Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Familial Hypercholesterolemia focused on measuring Homozygous, Familial, Hypercholesterolemia, MTP, Apheresis, lomitapide, Homozygous Familial Hypercholesterolemia (HoFH)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Cedars-Sinai Medical Center
  • University of Pennsylvania
  • Robarts Research Institute
  • Lipid Clinic and University of Montreal Community Genomic Medicine Center
  • Medicina Interna Universitaria
  • Dipartimento di Medicina Clinica e Delle Patologie Emergenti
  • Centro Universitario Dislipidemie
  • DAI Ematologia, Oncologia, Anatomia Patologica e Medicina
  • Cardiology Research
  • University of Capetown

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

lomitapide

Arm Description

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

Outcomes

Primary Outcome Measures

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).

Secondary Outcome Measures

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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).
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).

Full Information

First Posted
July 21, 2009
Last Updated
May 14, 2018
Sponsor
Aegerion Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT00943306
Brief Title
Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2018
Overall Recruitment Status
Completed
Study Start Date
October 29, 2009 (Actual)
Primary Completion Date
September 17, 2012 (Actual)
Study Completion Date
December 1, 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Aegerion Pharmaceuticals, Inc.

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
This was a long term follow on study to assess the continued long term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia.
Detailed Description
This was a phase III open label clinical trial to evaluate the long-term efficacy and safety of lomitapide at the maximum tolerated dose (for each patient) established during the clinical trial 733-005/UP1002. Subjects completing the 78-week treatment period in study 733-005/UP1002 who have not met any of the stopping criteria will be eligible to participate in 733-012. The treatment period continued until a decision was made by the local competent authority regarding marketing authorization. Lomitapide will be given orally once daily. Patient specific doses will be carried forward from 733-005/UP1002, but will not exceed the maximum tolerated dose the patient received during 733-005 /UP1002. The maximum dose for any patient was 80 mg/day. There was no reference therapy in this trial. The effects of the study drug were compared to baseline data (from 733-005/UP1002). Concomitant lipid-lowering therapy including plasmapheresis or LDL apheresis is permitted. Note that hereafter, Week 0 in Study 733-005/UP1002 will be referred to as Baseline and Week 48 in Study AEGR-733-012 will be referred to as Week 126 (of overall treatment).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Hypercholesterolemia
Keywords
Homozygous, Familial, Hypercholesterolemia, MTP, Apheresis, lomitapide, Homozygous Familial Hypercholesterolemia (HoFH)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
19 (Actual)

8. Arms, Groups, and Interventions

Arm Title
lomitapide
Arm Type
Experimental
Arm Description
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Intervention Type
Drug
Intervention Name(s)
lomitapide
Other Intervention Name(s)
AEGR-733, BMS-201038
Intervention Description
5-60 mg po every day
Primary Outcome Measure Information:
Title
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description
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 Outcome Measure Information:
Title
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description
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
Title
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description
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
Title
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description
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
Title
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description
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
Title
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C)
Description
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
Title
Percent Change in Total Cholesterol
Description
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
Title
Percent Change in Total Cholesterol
Description
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
Title
Percent Change in Total Cholesterol
Description
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
Title
Percent Change in Total Cholesterol
Description
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
Title
Percent Change in Total Cholesterol
Description
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
Title
Percent Change in Total Cholesterol
Description
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
Title
Percent Change in Apolipoprotein B (Apo B)
Description
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
Title
Percent Change in Apolipoprotein B (Apo B)
Description
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
Title
Percent Change in Apolipoprotein B (Apo B)
Description
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
Title
Percent Change in Apolipoprotein B (Apo B)
Description
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
Title
Percent Change in Apolipoprotein B (Apo B)
Description
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
Title
Percent Change in Apolipoprotein B (Apo B)
Description
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
Title
Percent Change in Triglycerides
Description
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
Title
Percent Change in Triglycerides
Description
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
Title
Percent Change in Triglycerides
Description
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
Title
Percent Change in Triglycerides
Description
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
Title
Percent Change in Triglycerides
Description
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
Title
Percent Change in Triglycerides
Description
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
Title
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description
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
Title
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description
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
Title
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description
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
Title
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description
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
Title
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description
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
Title
Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C)
Description
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
Title
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description
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
Title
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description
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
Title
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description
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
Title
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description
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
Title
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description
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
Title
Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C)
Description
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
Title
Percent Change in Lp(a)
Description
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
Title
Percent Change in Lp(a)
Description
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
Title
Percent Change in Lp(a)
Description
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
Title
Percent Change in Lp(a)
Description
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
Title
Percent Change in Lp(a)
Description
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
Title
Percent Change in Lp(a)
Description
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
Title
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description
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
Title
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description
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
Title
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description
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
Title
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description
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
Title
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description
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
Title
Percent Change in High Density Lipoprotein Cholesterol (HDL-C)
Description
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
Title
Percent Change in Apolipoprotein AI (Apo AI)
Description
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
Title
Percent Change in Apolipoprotein AI (Apo AI)
Description
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
Title
Percent Change in Apolipoprotein AI (Apo AI)
Description
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
Title
Percent Change in Apolipoprotein AI (Apo AI)
Description
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
Title
Percent Change in Apolipoprotein AI (Apo AI)
Description
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
Title
Percent Change in Apolipoprotein AI (Apo AI)
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Marina Cuchel, MD, PhD
Organizational Affiliation
University of Pennsylvania
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Mark Sumeray, MD
Organizational Affiliation
Aegerion Pharmaceuticals, Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
Cedars-Sinai Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Robarts Research Institute
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5K8
Country
Canada
Facility Name
Lipid Clinic and University of Montreal Community Genomic Medicine Center
City
Chicoutimi
State/Province
Quebec
ZIP/Postal Code
G7H 5H6
Country
Canada
Facility Name
Medicina Interna Universitaria
City
Ferrara
State/Province
Sicily
Country
Italy
Facility Name
Dipartimento di Medicina Clinica e Delle Patologie Emergenti
City
Palermo
State/Province
Sicily
Country
Italy
Facility Name
Centro Universitario Dislipidemie
City
Milano
Country
Italy
Facility Name
DAI Ematologia, Oncologia, Anatomia Patologica e Medicina
City
Roma
Country
Italy
Facility Name
Cardiology Research
City
Bloemfontein
ZIP/Postal Code
9300
Country
South Africa
Facility Name
University of Capetown
City
Cape town
ZIP/Postal Code
7925
Country
South Africa

12. IPD Sharing Statement

Citations:
PubMed Identifier
17215532
Citation
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.
Results Reference
background
Citation
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
Results Reference
background

Learn more about this trial

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

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