NCT00004809

Who might be able to join this trial:

• You have been diagnosed with homozygous familial hypercholesterolemia (a rare inherited condition causing very high cholesterol)
• Your LDL ("bad") cholesterol level is above 500 mg/dL
• The condition runs in your family in a specific inherited pattern (passed down through dominant genes)
• You developed fatty deposits on tendons or skin (called xanthomas) at an early age
• Testing of your skin cells shows your LDL receptors work at 20% or less of normal, OR you have been found to carry two known disease-causing gene variants
• You have serious heart disease with a relatively poor outlook, such as chest pain (angina), a past heart attack, an abnormal exercise stress test, or signs of artery disease seen on imaging
• You have not taken any medications that affect cholesterol, had plasma exchange, or had LDL apheresis (a blood-filtering treatment) for at least 2 weeks before joining
• People of any age may apply

Who may not be able to join:

• You have unstable (unpredictable or severe) chest pain
• Your heart's pumping function is significantly reduced (below 30%)
• You have severe, uncontrolled heart failure
• You have an untreated dangerous heart rhythm problem (ventricular tachycardia)
• You have moderate to severe narrowing of the aortic heart valve
• You have another type of cholesterol or fat disorder beyond this condition
• You have liver or bile duct disease that blocks normal flow
• You have kidney problems, including reduced kidney function or significant protein in your urine
• You have an underactive thyroid (hypothyroidism)
• You have diabetes

Important: Always verify eligibility with the trial site directly before applying.