A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy - ND

• Conditions: Homozygous Familial Hypercholesterolemia; MedDRA version: 9.1Level: LLTClassification code 10048373Term: Hypercholesterolaemia aggravated

• Registration Number: EUCTR2008-007058-36-IT
• Lead Sponsor: Aegerion Pharmaceuticals

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-06-03
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

1. Males and females &#8805; 18 years of age 2. Diagnosis of functional homozygous FH by at least one of the following clinical criteria: a. documented functional mutation(s) in both LDL receptor alleles or alleles known to affect LDL receptor functionality OR b. skin fibroblast LDL receptor activity < 20% normal OR c. untreated TC > 500 mg/dL and TG < 300 mg/dL AND both parents with documented untreated TC > 250 mg/dL 3. Subject understands and agrees that concurrent lipid lowering medication must be stable for &#8805; 6 weeks before the baseline visit and must remain stable through week 26. These include but are not limited to: statins, ezetimibe, nicotinic acid, bile acid sequestrants, fibrates and LDL apheresis. 4. Body weight &#8805; 40 kg and < 136 kg (due to MRI weight limit) 5. Negative screening pregnancy test if female of child-bearing potential (females of childbearing potential and all males must be following a medically accepted form of contraception) 6. Subjects must be willing to comply with all study-related procedures.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Exclusion Criteria: 1. Uncontrolled hypertension defined as: systolic blood pressure >180 mmHg, diastolic blood pressure > 95 mmHg on medication 2. History of chronic renal insufficiency (serum creatinine >2.5 mg/dL) 3. History of biopsy proven cirrhosis or abnormal LFTs at screening (AST or ALT >2x upper limit of normal and/or Total Bilirubin of > 1.5 mg/dl unless patient has unconjugated hyperbilirubinemia due to Gilbert?s syndrome) 4. Chronic hepatitis B as defined by positive for HBs Ag 5. Chronic hepatitis C as defined by positive for HepC Ab 6. Any major surgical procedure occurring less than 3 months prior to the screening visit 7. Cardiac insufficiency defined by the NYHA classification as functional Class III or Class IV 8. Previous organ transplantation 9. History of a non-skin malignancy within the previous 3 years 10. 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). 11. Participation in an investigational drug study within 6 weeks prior to the screening visit 12. Known significant gastrointestinal bowel disease or malabsorption such as inflammatory bowel disease or chronic pancreatitis requiring use of daily pancreatic enzymes. 13. 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. 14. Certain prohibited medications known to be potentially hepatotoxic, especially those that can induce microvesicular or macrovesicular steatosis. These include but are not limited to: acutane, amiodarone, heavy acetaminophen use (4g/day > 3 x q week), methotrexate, tetracyclines, and tamoxiphen 15. Documented diagnosis of any of the following pulmonary conditions: a. Asthma b. Chronic Obstructive Pulmonary Disease (COPD) c. Idiopathic pulmonary fibrosis 16. Documented diagnosis of any of the following liver diseases: a. Nonalcoholic Steatohepatitis b. Alcoholic liver disease c. Autoimmune hepatitis d. primary biliary cirrhosis e. primary sclerosing cholangitis f. Wilson?s disease g. hemochromatosis h. &#945;1 anti-trypsin deficiency 17. Current use of corticosteroids or betaine

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified