A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of Adding Ezetimibe 30 mg to an Ongoing Regimen of Ezetimibe 10 mg in Patients With Homozygous Sitosterolemia - Sitosterolemia High Dose Study

• Register: EUCTR
• Last refreshed on: 10 February 2014
• Main ID: EUCTR2004-001580-22-DE
• Date of registration: 20/10/2004
• Prospective Registration: Yes
• Primary sponsor: MSD SHARP & DOHME GMBH
• Public title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of Adding Ezetimibe 30 mg to an Ongoing Regimen of Ezetimibe 10 mg in Patients With Homozygous Sitosterolemia - Sitosterolemia High Dose Study
• Scientific title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of Adding Ezetimibe 30 mg to an Ongoing Regimen of Ezetimibe 10 mg in Patients With Homozygous Sitosterolemia - Sitosterolemia High Dose Study
• Date of first enrolment: 21/12/2004
• Target sample size: 30
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2004-001580-22
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Germany, United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
Patient is at least 18 years of age.

Patient has a diagnosis of homozygous sitosterolemia, with a history of plasma sitosterol concentration of >5 mg/dL (0.13 mmol/L).

Patient has been on an on-going stable regimen of ezetimibe 10 mg daily for at least 6 months prior to visit 1.

Current treatment regimen for sitosterolemia and diet must be stable for at least 4 weeks prior to visit 1 and for the duration of the study.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Patient has a condition which might pose a risk to the patient, interfere with participation in the study, or does not meet criteria for this protocol.

Patient has clinically significant lab abnormalities prior to visit 2 (ALT, AST, CPK = 3 x ULN, TSH >6 mIU/L).

Patient has the following conditions: uncontrolled cardiac arrhythmias, severe/unstable peripheral vascular disease, uncontrolled hypertension, impaired renal function, very poorly controlled Type 1 or Type 2 DM, active or chronic hepatobiliary or hepatic disease, positive for HIV.

Patient has the following conditions within 1 month of visit 1: unstable angina pectoris, symptomatic carotid disease, myocardial infarction, coronary artery bypass surgery/other invasive coronary.

Patient is on statin AND is treated with, or likely to require treatment with, an agent that has precautions or contraindications to concomitant use with the statin.
Patient is taking oral corticosteroids UNLESS patient uses as stable replacement therapy for pituitary/adrenal disease.

Patient is on a thiazide diuretic UNLESS treated with a stable regimen for at least 6 weeks prior to visit 1.

Patient has ileal bypass surgery within 3 months prior to visit 1.

Patient has a condition which might pose a risk to the patient, interfere with participation in the study, or does not meet criteria for this protocol.

Patient has clinically significant lab abnormalities prior to visit 2 (ALT, AST, CPK = 3 x ULN, TSH >6 mIU/L).

Patient has the following conditions: uncontrolled cardiac arrhythmias, severe/unstable peripheral vascular disease, uncontrolled hypertension, impaired renal function, very poorly controlled Type 1 or Type 2 DM, active or chronic hepatobiliary or hepatic disease, positive for HIV.

Patient has the following conditions within 1 month of visit 1: unstable angina pectoris, symptomatic carotid disease, myocardial infarction, coronary artery bypass surgery/other invasive coronary.

Patient is on statin AND is treated with, or likely to require treatment with, an agent that has precautions or contraindications to concomitant use with the statin.

Patient is taking oral corticosteroids UNLESS patient uses as stable replacement therapy for pituitary/adrenal disease.

Patient is on a thiazide diuretic UNLESS treated with a stable regimen for at least 6 weeks prior to visit 1.

Patient has ileal bypass surgery within 3 months prior to visit 1.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Homozygous Sitosterolemia

Intervention(s)

Trade Name: Ezetrol
Product Name: Ezetrol
Product Code: MK-0653
Pharmaceutical Form: Tablet
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10, 40-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: In patients with sitosterolemia who have been taking ezetimibe 10 mg daily for at least 6 months prior to entry, to compare the effects of adding ezetimibe 30 mg versus placebo to ongoing ezetimibe 10-mg treatment for 26 weeks on plasma sitosterol concentration.

Secondary Objective: In patients with sitosterolemia who have been taking ezetimibe 10 mg daily for at least 6 months prior to entry:

1.To compare the effects of adding ezetimibe 30 mg versus placebo to ongoing ezetimibe 10-mg treatment for 26 weeks on plasma campesterol concentration.

2.To assess change in size of Achilles tendon-thickness and non-Achilles xanthomas relative to baseline after adding ezetimibe 30 mg versus placebo to ongoing treatment with ezetimibe 10 mg for 26 weeks.

3.To evaluate the safety and tolerability of ezetimibe 40 mg in patients with sitosterolemia previously treated with ezetimibe 10 mg.

Primary end point(s): Plasma concentrations of sitosterol, campesterol, lathosterol, desmosterol, total cholesterol, LDL-C, radiographic assessment of Achilles tendon thickness and direct measurements of non-Achilles xanthomas.

Secondary ID(s)

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