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Sitosterolemia Research Studies

Sitosterolemia Research Study - Treatment with Ezetimibe (SCH-58235)

If you have homozygous sitosterolemia, you are invited to participate in a study that will evaluate a new investigation drug that inhibits cholesterol absorption. Ezetimibe (SCH-58235) reduces plant sterol concentrations in patients with sitosterolemia with short term treatment. Ezetimibe was well tolerated in these patients. The long term safety and benefit is being evaluated in this one year study.

Sitosterolemia should be suspected in children with hypercholesterolemia, tuberous and tendinous xanthomas, recurrent arthritis and an unusually good improvement in cholesterol levels due to diet and a bile acid resin binder.

Eligible persons will undergo a comprehensive evaluation. All patients will remain under the care of their own physician who will receive results of the laboratory and diagnostic tests. In most cases, you will be invited back for yearly evaluations related to other studies. There is no cost to you for travel, medical testing or the ezetimibe. 

For further information please contact our research coordinator (tel. 301-496-1500, e-mail: 

Last Updated: August 3, 2015