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SCH-58235 (Ezetimibe) to Treat Homozygous Sitosterolemia

NCT00045812 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2008-03-04

No results posted yet for this study

Summary

This study will test the safety and effectiveness of SCH-58235 (Ezetimibe) in lowering sitosterol, plant sterol and cholesterol blood levels in patients with homozygous sitosterolemia when added to the patients' current treatment regimen. Homozygous sitosterolemia is an inherited disorder of sterol metabolism in which an excess of many plant sterols, including sitosterol, is absorbed and not enough excreted. (Sterols are substances used to form hormones, vitamins and membranes found in animal and plant lipids.). Patients can develop atherosclerosis with coronary heart disease as early as childhood, as well as other problems including arthritis, arthralgia, and tendon xanthomas (lipid deposits). Current sitosterolemia treatments may include a low sterol diet, medications, intestinal surgery, or a combination of these. Ezetimibe is a member of a new class of drugs called "specific cholesterol absorption inhibitors" that may lower cholesterol, sitosterol and other plant sterol blood levels.

Patients with homozygous sitosterolemia 10 years of age and older may be eligible for this study. Participants will have a medical history and physical examination and will be randomly assigned to one of two treatment groups. One group, which will include about 80 percent of all study participants, will take 10 mg of Ezetimibe a day, and the second group (20 percent of participants) will take a placebo (an inactive look-a-like pill).

Patients will have 7 clinic visits during the 12-week study, when some or all of the following procedures and tests will be done:

* Measurement of vital signs (heart rate, blood pressure, breathing rate and temperature)
* Dietary maintenance - interview about how well that patient is adhering to the diet
* Medication review - interview about other medications the patient is taking
* Blood draw for tests
* Urine sample for tests
* Pregnancy test for women of childbearing potential
* Electrocardiogram (ECG) to measure the electrical activity of the heart
* Blood draw to determine sitosterol, other plant sterol levels, and lipid levels (cholesterol and other blood lipid concentrations)
* Xanthoma measurement (with a ruler and X-ray of the foot)

Conditions

  • Lipoidosis

Interventions

DRUG

SCH-58235

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    lead NIH

Study Design

Purpose
TREATMENT

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2001-03-31
Completion
2004-04-30

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00045812 on ClinicalTrials.gov