ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00943306
Recruitment Status : Completed
First Posted : July 22, 2009
Results First Posted : May 10, 2018
Last Update Posted : May 10, 2018
Sponsor:
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.

July 21, 2009
July 22, 2009
February 29, 2016
May 10, 2018
May 10, 2018
October 29, 2009
September 17, 2012   (Final data collection date for primary outcome measure)
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: Baseline and Week 126 ]
Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
Percent change in LDL-C [ Time Frame: 48 weeks ]
Complete list of historical versions of study NCT00943306 on ClinicalTrials.gov Archive Site
  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: Baseline and Week 174 ]
    Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: Baseline and Week 222 ]
    Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: Baseline and Week 246 ]
    Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: Baseline and Week 270 ]
    Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
  • Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) [ Time Frame: Baseline and Week 294 ]
    Percent change in Low Density Lipoprotein Cholesterol (LDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
  • Percent Change in Total Cholesterol [ Time Frame: Baseline and Week 126 ]
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
  • Percent Change in Total Cholesterol [ Time Frame: Baseline and Week 174 ]
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
  • Percent Change in Total Cholesterol [ Time Frame: Baseline and Week 222 ]
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
  • Percent Change in Total Cholesterol [ Time Frame: Baseline and Week 246 ]
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
  • Percent Change in Total Cholesterol [ Time Frame: Baseline and Week 270 ]
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
  • Percent Change in Total Cholesterol [ Time Frame: Baseline and Week 294 ]
    Percent change in Total Cholesterol from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein B (Apo B) [ Time Frame: Baseline and Week 126 ]
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein B (Apo B) [ Time Frame: Baseline and Week 174 ]
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein B (Apo B) [ Time Frame: Baseline and Week 222 ]
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein B (Apo B) [ Time Frame: Baseline and Week 246 ]
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein B (Apo B) [ Time Frame: Baseline and Week 270 ]
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein B (Apo B) [ Time Frame: Baseline and Week 294 ]
    Percent change in Apolipoprotein B (Apo B) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
  • Percent Change in Triglycerides [ Time Frame: Baseline and Week 126 ]
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
  • Percent Change in Triglycerides [ Time Frame: Baseline and Week 174 ]
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
  • Percent Change in Triglycerides [ Time Frame: Baseline and Week 222 ]
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
  • Percent Change in Triglycerides [ Time Frame: Baseline and Week 246 ]
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
  • Percent Change in Triglycerides [ Time Frame: Baseline and Week 270 ]
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
  • Percent Change in Triglycerides [ Time Frame: Baseline and Week 294 ]
    Percent change in Triglycerides from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
  • Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: Baseline and Week 126 ]
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
  • Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: Baseline and Week 174 ]
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
  • Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: Baseline and Week 222 ]
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
  • Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: Baseline and Week 246 ]
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
  • Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: Baseline and Week 270 ]
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
  • Percent Change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: Baseline and Week 294 ]
    Percent change in Non High Density Lipoprotein Cholesterol (Non-HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
  • Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: Baseline and Week 126 ]
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
  • Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: Baseline and Week 174 ]
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
  • Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: Baseline and Week 222 ]
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
  • Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: Baseline and Week 246 ]
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
  • Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: Baseline and Week 270 ]
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
  • Percent Change in Very Low Density Lipoprotein Cholesterol (VLDL-C) [ Time Frame: Baseline and Week 294 ]
    Percent change in Very Low Density Lipoprotein Cholesterol (VLDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
  • Percent Change in Lp(a) [ Time Frame: Baseline and Week 126 ]
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
  • Percent Change in Lp(a) [ Time Frame: Baseline and Week 174 ]
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
  • Percent Change in Lp(a) [ Time Frame: Baseline and Week 222 ]
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
  • Percent Change in Lp(a) [ Time Frame: Baseline and Week 246 ]
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
  • Percent Change in Lp(a) [ Time Frame: Baseline and Week 270 ]
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
  • Percent Change in Lp(a) [ Time Frame: Baseline and Week 294 ]
    Percent change in Lp(a) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
  • Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: Baseline and Week 126 ]
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
  • Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: Baseline and Week 174 ]
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
  • Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: Baseline and Week 222 ]
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
  • Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: Baseline and Week 246 ]
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
  • Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: Baseline and Week 270 ]
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
  • Percent Change in High Density Lipoprotein Cholesterol (HDL-C) [ Time Frame: Baseline and Week 294 ]
    Percent change in High Density Lipoprotein Cholesterol (HDL-C) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: Baseline and Week 126 ]
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 126 (Week 48 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: Baseline and Week 174 ]
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 174 (Week 96 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: Baseline and Week 222 ]
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 222 (Week 144 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: Baseline and Week 246 ]
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 246 (Week 168 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: Baseline and Week 270 ]
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 270 (Week 192 in Study AEGR-733-012).
  • Percent Change in Apolipoprotein AI (Apo AI) [ Time Frame: Baseline and Week 294 ]
    Percent change in Apolipoprotein AI (Apo AI) from Baseline (Week 0 of Study 733-005/UP1002) to Week 294 (Week 216 in Study AEGR-733-012).
  • Percent change in lipid parameters [ Time Frame: 48 weeks ]
  • Long term safety [ Time Frame: 48 Weeks ]
  • Percent hepatic fat by MRI [ Time Frame: 48 Weeks ]
Not Provided
Not Provided
 
Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
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.

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).

Interventional
Phase 3
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Familial Hypercholesterolemia
Drug: lomitapide
5-60 mg po every day
Other Names:
  • AEGR-733
  • BMS-201038
Experimental: lomitapide
Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Intervention: Drug: lomitapide

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
19
25
December 1, 2014
September 17, 2012   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
Canada,   Italy,   South Africa,   United States
 
 
NCT00943306
AEGR-733-012
No
Not Provided
Not Provided
Aegerion Pharmaceuticals, Inc.
Aegerion Pharmaceuticals, Inc.
Not Provided
Principal Investigator: Marina Cuchel, MD, PhD University of Pennsylvania
Study Chair: Mark Sumeray, MD Aegerion Pharmaceuticals, Inc.
Aegerion Pharmaceuticals, Inc.
April 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP