Clinical trial NCT00263081 (phase 3, status: terminated)
Conditions: hypercholesterolemia
ICD codes: E78.01, E78.00, Z83.42
Drugs: lapaquistat acetate and current lipid-lowering treatment, current lipid-lowering treatment
SMILES: CC(O)=O, COC1=CC=CC=C1OC1=C(NS(=O)(=O)C2=CC=C(C=C2)C(C)(C)C)N=C(N=C1OCCO)C1=NC=CC=N1
Reason stopped: 
    overall profile of the compound does not offer significant clinical advantage to patients over
    currently available lipid lowering agents
  
Eligibility criteria:

        Inclusion Criteria:

          -  Must be at least 8 years of age (12 years of age in Poland) and weigh greater than or
             equal to 30 kg.

          -  Must have homozygous FH, as documented by genetic testing that confirmed 2 mutated
             alleles at the LDL-receptor locus, or by the following clinical criteria:

               -  documented history of untreated fasting serum LDL-C >460 mg/dL (11.91 mmol/L).

               -  tendinous or cutaneous xanthomas and/or corneal arcus before age 10 and/or
                  premature coronary heart disease before age 20.

          -  Must be taking a stable, approved lipid-lowering drug regimen for a minimum of 8 weeks
             prior to Screening Visit 1 and were to continue that treatment for the duration of the
             study.

          -  Female participants can not be pregnant, not lactating, not planning on becoming
             pregnant and agree to use adequate contraception throughout the course of the study.

        Exclusion Criteria:

          -  Has alanine aminotransferase or aspartate aminotransferase levels greater than 2 times
             the upper limit of normal, active liver disease, jaundice, or serum creatinine >135
             μmol/L (1.5 mg/dL) at screening.

          -  Has a creatine kinase greater than 3 times the upper limit of normal

          -  Has a positive hepatitis B surface antigen, or hepatitis C virus antibody, as
             determined by medical history.

          -  Has a positive human immunodeficiency virus status or was taking antiretroviral
             medications, as determined by medical history and/or subject's verbal report.

          -  Is unable or unwilling to discontinue excluded medications or to continue stable doses
             of 'stable dose' medications or would require treatment with any excluded medication
             during the study.

          -  Is currently participating in another investigational study or had participated in an
             investigational study within the past 30 days or, for drugs with a long half-life,
             within a period of less than 5 times the drug's half-life.

          -  Has a previous history of cancer that had been in remission for less than 5 years
             prior to the first dose of study drug. This criterion was not to include those
             subjects with basal cell or Stage 1 squamous cell carcinoma of the skin.

          -  Has known hypersensitivity or history of adverse reaction to lapaquistat acetate.

          -  Has a history of fibromyalgia, myopathy, rhabdomyolysis, or unexplained muscle pain.

          -  Has an endocrine disorder, such as Cushing Syndrome, hyperthyroidism, or
             inappropriately treated hypothyroidism, affecting lipid metabolism. Subjects with
             hypothyroidism on appropriate replacement therapy.

          -  Has uncontrolled hypertension despite medical treatment.

          -  Has inflammatory bowel disease or any other malabsorption syndrome or had gastric
             bypass or any other surgical procedure for weight loss.

          -  Has a history of drug abuse or a history of alcohol abuse within the past 2 years.

          -  Has any other serious disease or condition at screening or at randomization that might
             have reduced life expectancy, impaired successful management according to the
             protocol, or made the subject an unsuitable candidate to receive study drug.