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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
Last Update Posted : March 27, 2015
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Familial Hypercholesterolemia Drug: lomitapide Phase 3

Detailed Description:
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.

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : September 2009
Primary Completion Date : December 2014
Study Completion Date : December 2014

Resource links provided by the National Library of Medicine

Drug Information available for: Lomitapide
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: lomitapide
Maximum tolerated dose of lomitapide (up to 80mg/day) in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.
Drug: lomitapide
5-60 mg po every day
Other Names:
  • AEGR-733
  • BMS-201038


Outcome Measures

Primary Outcome Measures :
  1. Percent change in LDL-C [ Time Frame: 48 weeks ]

Secondary Outcome Measures :
  1. Percent change in lipid parameters [ Time Frame: 48 weeks ]
  2. Long term safety [ Time Frame: Every 12 weeks until the study is completed ]
    This is an open ended study that will continue until a local market authorization decision is made in each region

  3. Percent hepatic fat by NMRS [ Time Frame: Every 24 weeks until the study is completed ]
    This is an open ended study that will remain open until a local decision regarding market authorization is made


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00943306


Locations
United States, California
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
United States, Pennsylvania
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Canada, Ontario
Robarts Research Institute
London, Ontario, Canada, N6A 5K8
Canada, Quebec
Lipid Clinic and University of Montreal Community Genomic Medicine Center
Chicoutimi, Quebec, Canada, G7H 5H6
Italy
Medicina Interna Universitaria
Ferrara, Sicily, Italy
Dipartimento di Medicina Clinica e Delle Patologie Emergenti
Palermo, Sicily, Italy
Centro Universitario Dislipidemie
Milano, Italy
South Africa
Cardiology Research
Bloemfontein, South Africa, 9300
University of Capetown
Cape town, South Africa, 7925
Sponsors and Collaborators
Aegerion Pharmaceuticals, Inc.
Investigators
Principal Investigator: Marina Cuchel, MD, PhD University of Pennsylvania
Study Chair: Mark Sumeray, MD Aegerion Pharmaceuticals, Inc.
More Information

Publications:
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

Responsible Party: Aegerion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT00943306     History of Changes
Other Study ID Numbers: 733-012
First Posted: July 22, 2009    Key Record Dates
Last Update Posted: March 27, 2015
Last Verified: March 2015

Keywords provided by Aegerion Pharmaceuticals, Inc.:
Homozygous
Familial
Hypercholesterolemia
MTP
Apheresis
lomitapide
Homozygous Familial Hypercholesterolemia (HoFH)

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias