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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT00943306
First received: July 21, 2009
Last updated: January 15, 2014
Last verified: January 2014

July 21, 2009
January 15, 2014
September 2009
December 2013   (final data collection date for primary outcome measure)
Percent change in LDL-C [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT00943306 on ClinicalTrials.gov Archive Site
  • Percent change in lipid parameters [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
  • Long term safety [ Time Frame: Every 12 weeks until the study is completed ] [ Designated as safety issue: Yes ]
    This is an open ended study that will continue until a local market authorization decision is made in each region
  • Percent hepatic fat by NMRS [ Time Frame: Every 24 weeks until the study is completed ] [ Designated as safety issue: Yes ]
    This is an open ended study that will remain open until a local decision regarding market authorization is made
  • Percent change in lipid parameters [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]
  • Long term safety [ Time Frame: 48 Weeks ] [ Designated as safety issue: Yes ]
  • Percent hepatic fat by MRI [ Time Frame: 48 Weeks ] [ Designated as safety issue: Yes ]
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 is a long term follow on study to assess the continued long term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia.

This is 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 study 733-005/UP1002 who have not met any of the stopping criteria will be eligible to participate in 733-012. The treatment period will continue until a decision has been 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 is 80 mg/day. There is no reference therapy in this trial. The effects of the study drug will be compared to baseline data (from 733-005/UP1002). Concomitant lipid-lowering therapy including plasmapheresis or LDL apheresis is permitted.

Interventional
Phase 3
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 (up to 80mg/day) 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.
 
Active, not recruiting
25
December 2014
December 2013   (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.
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
South Africa,   United States,   Italy,   Canada
 
NCT00943306
733-012
No
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.
January 2014

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