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Sponsors and Collaborators: |
Aegerion Pharmaceuticals, Inc. FDA Office of Orphan Products Development |
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Information provided by: | Aegerion Pharmaceuticals, Inc. |
ClinicalTrials.gov Identifier: | NCT00603161 |
The purpose of this study is to determine the safety and effectiveness of long term use of AEGR-733 on cholesterol in patients with homozygous familial hypercholesterolemia
Condition | Intervention | Phase |
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Homozygous Familial Hypercholesterolemia |
Drug: AEGR-733 |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Dose Comparison, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients With Homozygous Familial Hypercholesterolemia on Current Lipid-Lowering Therapy |
Estimated Enrollment: | 25 |
Study Start Date: | December 2007 |
Estimated Study Completion Date: | January 2011 |
Estimated Primary Completion Date: | November 2009 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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AEGR-733: Experimental
Microsomal Triglyceride Transfer Protein Inhibitor
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Drug: AEGR-733
Microsomal triglyceride transfer protein inhibitor
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Homozygous familial hypercholesterolemia (FH) is a serious life-threatening genetic disease. Total plasma cholesterol levels are generally over 500 mg/dl and markedly premature cardiovascular disease is the major consequence. Untreated, most patients develop atherosclerosis before age 20 and generally do not survive past age 30. The primary goal of therapy involves reducing cholesterol (specifically, LDL cholesterol) and preventing coronary artery disease. Unfortunately, patients with homozygous FH are minimally responsive or unresponsive to available drug therapy and thus there are limited treatment options. The current standard of care is LDL apheresis, a physical method of removing the plasma of LDL cholesterol which can transiently reduce cholesterol by more than 50%. However, there is rapid re-accumulation of LDL cholesterol in plasma, and therefore apheresis has to be repeated frequently (every 1-2 weeks) and requires 2 separate sites for IV access. Although anecdotally this procedure may delay the onset of atherosclerosis, it is laborious, expensive, and not readily available. Furthermore, although it is a procedure that is generally well tolerated, the fact that it needs frequent repetition and IV access can be challenging for many of these young patients. Therefore, there is a tremendous unmet medical need for new medical therapies for this orphan disease.
AEGR-733 is a novel oral therapeutic agent for hypercholesterolemia. Its mechanism involves inhibition of microsomal triglyceride transfer protein, resulting in a reduction of LDL cholesterol. Earlier studies in patients with homozygous FH reveal AEGR-733 is highly effective in lowering LDL cholesterol, yet long term safety and efficacy need to be established.
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Diagnosis of functional homozygous FH by at least one of the following clinical criteria:
Exclusion Criteria:
Documented diagnosis of any of the following pulmonary conditions:
Documented diagnosis of any of the following liver diseases:
Contact: Matthew Parris, BSc | 908 707 2142 | mparris@aegerion.com |
Contact: Marina Cuchel, MD, PhD | 215-746-2834 | mcuchel@mail.med.upenn.edu |
United States, California | |
Cedars-Sinai Medical Center | Recruiting |
Los Angeles, California, United States, 90048 | |
Contact: Linn Defensor, RN 310-423-6034 Defensor@cshs.org | |
Contact: David Gallegos, RN 310-423-4993 gallegosd@cshs.org | |
Principal Investigator: Prediman K Shah, MD | |
United States, Pennsylvania | |
University of Pennsylvania | Recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
Contact: Amanda Baer 215-746-3423 baer2@mail.med.upenn.edu | |
Contact: Marina Cuchel, MD, PhD 215 746 2834 mcuchel@mail.med.upenn.edu | |
Principal Investigator: Marina Cuchel, MD, PhD | |
Sub-Investigator: Emma Meagher, MD |
Principal Investigator: | Marina Cuchel, MD, PhD | University of Pennsylvania |
Principal Investigator: | Emma Meagher, MD | University of Pennsylvania |
Responsible Party: | Aegerion Pharmaceuticals ( William J Sasiela, PhD Chief Medical Officer ) |
Study ID Numbers: | UP1002 |
Study First Received: | January 15, 2008 |
Last Updated: | January 9, 2009 |
ClinicalTrials.gov Identifier: | NCT00603161 |
Health Authority: | United States: Food and Drug Administration |
MTP inhibition Homozygous familial hypercholesterolemia LDL cholesterol |
Lipid Metabolism, Inborn Errors Hypercholesterolemia, autosomal dominant Metabolism, Inborn Errors Metabolic Diseases Hyperlipidemias Genetic Diseases, Inborn |
Hyperlipoproteinemia Type II Metabolic disorder Hypercholesterolemia Dyslipidemias Hyperlipoproteinemias Lipid Metabolism Disorders |