Ignite Creation Date:
2024-05-05 @ 11:38 AM
Last Modification Date:
2024-10-26 @ 9:11 AM
Study NCT ID:
NCT00099996
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2004-12-21
Brief Title:
Higher-Dose Ezetimibe to Treat Homozygous Sitosterolemia
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Study to Assess Adding Ezetimibe 30 mg to Ongoing Treatment With Ezetimibe 10 mg in Patients With Homozygous Sitosterolemia
Status:
COMPLETED
Status Verified Date:
2005-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will test the safety and effectiveness of 40 mg of ezetimibe Zetia daily in lowering blood levels of cholesterol and of the plant sterols sitosterol and campesterol in patients with homozygous sitosterolemia an inherited disorder of sterol metabolism Sterols are alcohol substances found in animal and plant fats In this disorder an excess of many plant sterols is absorbed and not enough excreted Patients can develop atherosclerosis and coronary heart disease as early as childhood as well as other problems including arthritis arthralgia and tendon xanthomas lipid deposits Current treatment consists of ezetimibe 10 mg dietary restriction of plant and shellfish sterols and bile salt binding resins Ezetimibe is a cholesterol-lowering drug that inhibits intestinal absorption of cholesterol and structurally related plant sterols across the intestinal wall
Patients with homozygous sitosterolemia who are between 18 and 85 years of age have completed NHLBIs 1-year study of ezetimibe at 10 mg a day may be eligible for this study
All participants maintain their current stable diet and take a 10-mg pill of ezetimibe daily for 26 weeks They are also randomly selected to take either an additional 30-mg pill of ezetimibe or a placebo look-alike pill with no active ingredients Patients fast for at least 12 hours before each of 6 visits scheduled during the course of the study At these visits patients undergo some or all of the following procedures for monitoring their health and evaluating their response to treatment
Medical history and review of medications
Physical examination
Measurement of vital signs pulse rate blood pressure breathing rate and temperature
Review of dietary maintenance
Measurements of height weight and waist circumference
Measurement with ruler and photographs of non-Achilles xanthoma
X-ray of Achilles tendon
Blood draw and urine collection
Pregnancy test for women of childbearing potential
Patients with homozygous sitosterolemia who are between 18 and 85 years of age have completed NHLBIs 1-year study of ezetimibe at 10 mg a day may be eligible for this study
All participants maintain their current stable diet and take a 10-mg pill of ezetimibe daily for 26 weeks They are also randomly selected to take either an additional 30-mg pill of ezetimibe or a placebo look-alike pill with no active ingredients Patients fast for at least 12 hours before each of 6 visits scheduled during the course of the study At these visits patients undergo some or all of the following procedures for monitoring their health and evaluating their response to treatment
Medical history and review of medications
Physical examination
Measurement of vital signs pulse rate blood pressure breathing rate and temperature
Review of dietary maintenance
Measurements of height weight and waist circumference
Measurement with ruler and photographs of non-Achilles xanthoma
X-ray of Achilles tendon
Blood draw and urine collection
Pregnancy test for women of childbearing potential
Detailed Description:
Homozygous Sitosterolemia is an inherited autosomal recessive disorder of sterol metabolism Patients with homozygous sitosterolemia experience accelerated atherosclerosis with initial coronary heart disease CHD events occurring in childhood Plasma concentrations of sitosterol and other dietary plant sterols are markedly elevated in homozygous sitosterolemic patients and are characteristic of this disorder Sitosterolemic individuals demonstrate a range of abnormalities in sterol absorption metabolism and excretion Recent reports have shown that sitosterolemia can result from mutations in 1 of 2 ATP-binding cassette half-transporters ABCG5 or ABCG8 which are responsible for regulation of non-cholesterol sterols in the body
Current treatment of homozygous sitosterolemia consists of ezetimibe 10 mg dietary restriction of plant and shellfish sterols as well as the use of bile salt binding resins Ezetimibe is the first member of a new class of cholesterol-lowering agents that inhibits the intestinal absorption of cholesterol and structurally-related noncholesterol sterols plant sterols across the intestinal wall Importantly ezetimibe is not an inhibitor or inducer of CYP450 reducing the potential for drug-drug interactions which renders ezetimibe a particularly appealing candidate with other drugs Ezetimibe has proved to be generally safe and well-tolerated as monotherapy or when coadministered with statins with an overall clinical adverse experience profile similar to placebo In clinical studies with hypercholesterolemic patients ezetemibe doses ranging from 025 to 40 mg daily for periods of 8 to 12 weeks were more effective than placebo in lowering plasma TC and LDL-C concentrations There were no dose-related increase in adverse experiences or laboratory abnormalities in these studies We will investigate whether a higher dose of ezetimibe is safe and efficacious in lowering plant sterols in patients with sitosterolemia
Current treatment of homozygous sitosterolemia consists of ezetimibe 10 mg dietary restriction of plant and shellfish sterols as well as the use of bile salt binding resins Ezetimibe is the first member of a new class of cholesterol-lowering agents that inhibits the intestinal absorption of cholesterol and structurally-related noncholesterol sterols plant sterols across the intestinal wall Importantly ezetimibe is not an inhibitor or inducer of CYP450 reducing the potential for drug-drug interactions which renders ezetimibe a particularly appealing candidate with other drugs Ezetimibe has proved to be generally safe and well-tolerated as monotherapy or when coadministered with statins with an overall clinical adverse experience profile similar to placebo In clinical studies with hypercholesterolemic patients ezetemibe doses ranging from 025 to 40 mg daily for periods of 8 to 12 weeks were more effective than placebo in lowering plasma TC and LDL-C concentrations There were no dose-related increase in adverse experiences or laboratory abnormalities in these studies We will investigate whether a higher dose of ezetimibe is safe and efficacious in lowering plant sterols in patients with sitosterolemia
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 05-H-0060 | None | None | None |