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

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ClinicalTrials.gov Identifier: NCT00943306
Recruitment Status : Completed
First Posted : July 22, 2009
Results First Posted : May 11, 2018
Last Update Posted : June 13, 2018
Sponsor:
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE July 21, 2009
First Posted Date  ICMJE July 22, 2009
Results First Submitted Date  ICMJE February 29, 2016
Results First Posted Date  ICMJE May 11, 2018
Last Update Posted Date June 13, 2018
Actual Study Start Date  ICMJE October 29, 2009
Actual Primary Completion Date September 17, 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 6, 2018)
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).
Original Primary Outcome Measures  ICMJE
 (submitted: July 21, 2009)
Percent change in LDL-C [ Time Frame: 48 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 6, 2018)
  • 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).
Original Secondary Outcome Measures  ICMJE
 (submitted: July 21, 2009)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
Official Title  ICMJE 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
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).

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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 18, 2015)
19
Original Estimated Enrollment  ICMJE
 (submitted: July 21, 2009)
25
Actual Study Completion Date  ICMJE December 1, 2014
Actual Primary Completion Date September 17, 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Italy,   South Africa,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00943306
Other Study ID Numbers  ICMJE AEGR-733-012
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Aegerion Pharmaceuticals, Inc.
Study Sponsor  ICMJE Aegerion Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Marina Cuchel, MD, PhD University of Pennsylvania
Study Chair: Mark Sumeray, MD Aegerion Pharmaceuticals, Inc.
PRS Account Aegerion Pharmaceuticals, Inc.
Verification Date May 2018

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