The purpose of this study is to determine if implitapide, used in conjunction with other lipid-lowering therapies, is safe and effective when compared to placebo in lowering low-density lipoprotein cholesterol (LDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).

Condition	Treatment or Intervention	Phase
Familial Hypercholesterolemia	Drug: Implitapide	Phase II

Ages Eligible for Study:  8 Years   -   70 Years,  Genders Eligible for Study:  Both

Criteria

Inclusion Criteria:

In order to participate in this study, patients must meet all of the following inclusion criteria:

• be between 8 and 70 years old with a diagnosis of HoFH;
• be stable on and maintain concomitant therapy with hypolipidemic drugs or treatments;
• have an appropriate calculated, fasting LDL-C levels and an appropriate triglyceride (TG) level;
• be male or nonpregnant, nonlactating female;
• give informed consent; and
• meet body weight and height requirements.

Exclusion Criteria:

In order to participate in this study, patients must not meet any of the following exclusion criteria:

• recent myocardial infarction, percutaneous transluminal coronary intervention, coronary artery bypass graft surgery, or cerebrovascular accident;
• uncontrolled hypothyroidism or other uncontrolled endocrine disease;
• known, clinically significant eye abnormalities (e.g., cataracts);
• appropriate serum creatinine phosphokinase levels;
• history of liver disease or liver enzyme levels above appropriate levels;
• alkaline phosphatase above appropriate levels;
• liver cirrhosis and severe liver steatosis;
• clinically significant infection, malignancy, or psychosis;
• use of oral anticoagulants or digoxin, unless the dose has been stable for 4 weeks;
• participation in any other investigational study, including device or observational studies, within 30 days;
• lactating or have a positive serum pregnancy test;
• history of or current drug or alcohol abuse; or
• unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the investigator.

Crystal Ellison      919.462.4479    crystal.ellison@rtp.ppdi.com

Ohio
      Metabolic and Atherosclerosis Research Center, Cincinnati,  Ohio,  45229,  United States; Recruiting
Canada, Quebec
      Lipid Clinic and Community Genomic Center, Complexe Hospitalier de la Sagamie, Chicoutimi,  Quebec,  G7H 5H6,  Canada; Recruiting
      Lipid Research Center, CHUL du CHUQ, Sainte-Foy,  Quebec,  G1V 4G2,  Canada; Recruiting
Israel
      Hadassah University Hospital, Jerusalem,  91120,  Israel; Recruiting
Netherlands
      Academic Medical Center Amsterdam, Amsterdam,  1105 AZ,  Netherlands; Recruiting
Norway
      Lipidklinikken - Rikshospitalet, Oslo,  N-0027,  Norway; Recruiting

Study ID Numbers:  MRL 2002-001
Record last reviewed:  September 2004
Record first received:  March 17, 2004
ClinicalTrials.gov Identifier:  NCT00079846
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2004-11-10