Status:
COMPLETED
A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)
Lead Sponsor:
Aegerion Pharmaceuticals, Inc.
Collaborating Sponsors:
FDA Office of Orphan Products Development
Conditions:
Homozygous Familial Hypercholesterolemia
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
The goal of this trial is to study the effects of AEGR-733 on LDL cholesterol, other lipids as well as measures of safety over the long-term.
Detailed Description
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 ...
Eligibility Criteria
Inclusion
- Males and females at least 18 years of age
- Diagnosis of functional homozygous FH by at least one (a-c) of the following clinical criteria:
- documented functional mutation(s) in both LDL receptor alleles or alleles known to affect LDL receptor functionality OR
- skin fibroblast LDL receptor activity less than 20% normal OR
- untreated TC greater than 500 mg/dL AND TG less then 300 mg/dL AND both parents have documented TC greater than 250 mg/dL
- Concurrent lipid lowering medication/apheresis must be stable for at least 6 weeks before the baseline visit and must remain stable for the first 26 weeks.
- Body weight at least 40 kg and less than 136 kg
- Negative screening pregnancy test if female of child-bearing potential (females of child-bearing potential and all males must be following a medically accepted form of contraception)
- Subjects must be willing to comply with all study-related procedures
Exclusion
- Uncontrolled hypertension
- History of chronic renal insufficiency
- History of biopsy proven cirrhosis or abnormal LFTs at screening (AST or ALT greater than 2 x upper limit of normal and/or Total Bilirubin greater than or equal to 1.5 mg/dl unless patient has unconjugated hyperbilirubinemia due to Gilbert's syndrome)
- Chronic hepatitis B or chronic hepatitis C
- Any major surgical procedure occurring less than 3 months prior to the screening visit
- Cardiac insufficiency defined by the NYHA classification as functional Class III or Class IV
- Previous organ transplantation
- History of a non-skin malignancy within the previous 3 years
- Male subjects reporting more than 2 drinks per day or females reporting more than 1 drink per day (1 drink= 12 oz beer, 1 oz hard liquor, 5 oz wine).
- Participation in an investigational drug study within 6 weeks prior to the screening visit
- Known significant gastrointestinal bowel disease or malabsorption such as inflammatory bowel disease or chronic pancreatitis requiring use of daily pancreatic enzymes.
- Serious or unstable medical or psychological conditions that, in the opinion of the investigator, would compromise the subject's safety or successful participation in the study.
- Certain prohibited medications known to be potentially hepatotoxic, especially those that can induce microvesicular or macrovesicular steatosis. These include but are not limited to: accutane, amiodarone, heavy acetaminophen use (4g/day greater than 3 x q week), methotrexate, tetracyclines,and tamoxifen
- Documented diagnosis of any of the following pulmonary conditions: Asthma, Chronic Obstructive Pulmonary Disease (COPD), Idiopathic pulmonary fibrosis
- Documented diagnosis of any of the following liver diseases: Nonalcoholic Steatohepatitis, Alcoholic liver disease, Autoimmune hepatitis, primary biliary cirrhosis, primary sclerosing cholangitis, Wilson's disease, hemochromatosis, alpha 1 anti-trypsin deficiency.
- Current use of corticosteroids or betaine
Key Trial Info
Start Date :
December 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2011
Estimated Enrollment :
29 Patients enrolled
Trial Details
Trial ID
NCT00730236
Start Date
December 1 2007
End Date
October 1 2011
Last Update
March 20 2018
Active Locations (10)
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1
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
2
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
3
Robarts Research Institute
London, Ontario, Canada, N6A 5K8
4
Lipid Clinic and University of Montreal Community Genomic Medicine Center
Chicoutimi, Quebec, Canada, G7H 5H6