SCH-58235 (Ezetimibe) to Treat Homozygous Sitosterolemia

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00045812
• Date of registration: 10/09/2002
• Prospective Registration: No
• Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Public title: SCH-58235 (Ezetimibe) to Treat Homozygous Sitosterolemia
• Scientific title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate SCH-58235 in Homozygous Sitosterolemia
• Date of first enrolment: March 2001
• Target sample size: 5
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00045812
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

INCLUSION/EXCLUSION CRITERIA

Male or female, age greater than or equal to 10 years are eligible.

Patients must have a diagnosis of homozygous sitosterolemia with an elevated plasma
sitosterol level (greater than 5 mg/dL at screening visit) on their current regimen.

All women of childbearing potential must be practicing an effective method of
contraception.

All women of childbearing potential must have a negative urine pregnancy test within 72
hours prior to the start of the study medication.

Patients must not have any condition which, in the opinion of the investigator, would be
likely to render the patient unable to complete the study or for which study participation
would produce significant risk or not be in the best interests of the patient.

Individual with poor mental function, drug or substance abuse, or individuals with
unstable psychiatric illness, which, in the opinion of the investigator, may interfere
with optimal participation in the study will be excluded.

Women must not be pregnant or lactating.

Patients must not have had treatment with any other investigational drug within 30 days
prior to visit 1.

Patients can not newly diagnosed or untreated person who has not been given an opportunity
to consider treatment with standard of care and an opportunity to decline such treatment.

Patients must not have congestive heart failure NYHA class III or IV.

Patients must not have uncontrolled cardiac arrhythmias.

Patients must not have had a myocardial infarction, coronary bypass surgery or angioplasty
within 6 months of the screening visit (Visit 1).

Patients must not have unstable angina pectoris or unstable or severe peripheral vascular
disease.

Patients must not have uncontrolled diabetes mellitus (Hb(A1c) greater than 10%).
Patients with diabetes mellitus should be on a stable antihyperglycemic regimen for at
least 4 weeks prior to the screening visit (Visit 1).

Patients must not have uncontrolled endocrine or metabolic disease known to influence
serum lipids or lipoproteins. Clinically euthyroid patients on stable replacement doses
of thyroid hormone (on the same dose for at least 4 weeks prior to study entry and with
TSH equal to 10 IU/mL) are eligible for enrollment.

Patients must not have uncontrolled hypertension (systolic BP greater than 200 mm Hg
and/or diastolic BP greater than 110 mm Hg).

Patients must not have creatinine greater than 2.0 mg/dL at the screening visit (Visit 1),
or active renal disease with significant proteinuria (greater than 1 albumin/mg
creatinine).

Patients must not have active acute or chronic hepatobiliary disease; AST or ALT greater
than 5 times the upper limit of normal of the reference laboratory at the screening visit
(Visit 1).

Patients must not have disorders of the hematologic, digestive (including malabsorptive
disorders), or central nervous system including cerebrovascular disease and degenerative
diseases that would limit study evaluation or participation.

Patients must not be HIV positive.



Age minimum: N/A
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Lipoidosis

Intervention(s)

Drug: SCH-58235

Secondary ID(s)

01-H-0115

010115

Source(s) of Monetary Support

Please refer to primary and secondary sponsors