Status:

TERMINATED

Efficacy of Lapaquistat Acetate Co-Administered With Current Lipid-Lowering Treatment on Blood Cholesterol Levels in Subjects With Homozygous Familial Hypercholesterolemia

Lead Sponsor:

Takeda

Conditions:

Hypercholesterolemia

Eligibility:

All Genders

8+ years

Phase:

PHASE3

Brief Summary

The purpose of the study is to determine the efficacy of lapaquistat acetate, once daily (QD), to lower cholesterol in subjects with homozygous familial hypercholesterolemia undergoing lipid-lowering ...

Detailed Description

According to the World Health Organization, CHD is now the leading cause of death worldwide. In 2001, CHD caused 7.2 million deaths and estimates for 2020 indicate that annual CHD deaths will increase...

Eligibility Criteria

Inclusion

  • 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

  • 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.

Key Trial Info

Start Date :

November 1 2005

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

March 1 2007

Estimated Enrollment :

44 Patients enrolled

Trial Details

Trial ID

NCT00263081

Start Date

November 1 2005

End Date

March 1 2007

Last Update

May 24 2012

Active Locations (10)

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Page 1 of 3 (10 locations)

1

Cincinnati, Ohio, United States

2

Columbus, Ohio, United States

3

Québec, Canada

4

Paris, France