A Study of Extended-release (ER) Niacin/Laropiprant in Adolescents With Heterozygous Familial Hypercholesterolemia...
HypercholesterolemiaFamilial1 moreThe purpose of this study is to determine the pharmacokinetics of laropiprant following administration of a single dose of 1 (Panel A) and 2 (Panel B) combination tablets of MK-0524A in adolescents with heterozygous familial hypercholesterolemia.
Carotid IMT (Intima Media Thickening) Study (0524A-041)(TERMINATED)
HypercholesterolemiaFamilialThis is a 105-week clinical study in patients with heterozygous familial hypercholesterolemia on intensive Low Density Lipoprotein-cholesterol (LDL-C) lowering therapy intended to assess the affects of MK0524A on carotid intima media thickening using ultrasound compared to patients taking placebo. There will be 12 scheduled clinic visits involving review of medical history, physical exam, vital signs, laboratory testing, ultrasound imaging, and electrocardiograms.
2-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia...
Familial HypercholesterolemiaThe Investigators will test the hypothesis that 2-HOBA will reduce modification of HDL and LDL and improve HDL function in humans with heterozygous FH. The Investigators plan to first study subjects with Familial Hypercholesterolemia (FH), treating them with 750 mg of 2-HOBA or placebo every 8 hours for 6 weeks.
Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED)...
HypercholesterolemiaFamilial2 moreThe purpose of this study is to provide an investigational drug to patients with a specific type of hypercholesterolemia (high cholesterol) or sitosterolemia (unusually high absorption of non-cholesterol sterols) in a treatment use setting.
Study to Evaluate the Effects of MBX-8025 in Patients With HoFH
Homozygous Familial HypercholesterolemiaA 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH).
Safety and Tolerability Study of Ezetimibe (SCH 058235/MK-0653) Plus Atorvastatin or Simvastatin...
HypercholesterolemiaThe primary purpose of this study is to evaluate the long-term safety and tolerability of ezetimibe (SCH 058235/MK-0653) 10 mg dosed daily and co-administered with either atorvastatin or simvastatin for up to 24 months in participants with homozygous familial hypercholesterolemia (FH). Following completion of the 12-week, double-blind, efficacy and safety parent study (P01030/MK-0653-018; NCT03884452) participants will be offered entry into this open-label, 24-month extension study.
A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
Homozygous Familial HypercholesterolemiaThis study was a Phase III,A two-part (double-blind placebo-controlled/open-label) multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in subjects with homozygous familial hypercholesterolemia (HoFH).
An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial...
HypercholesterolaemiaPrimary Objective: To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W) and every 4 weeks (Q4W) versus placebo after 24 weeks of double-blind (DB) treatment on low-density lipoprotein cholesterol (LDL-C) levels in participants with heterozygous familial hypercholesterolemia (heFH) 8 to 17 years of age on optimal stable daily dose of statin therapy ± other lipid modifying therapies (LMTs) or a stable dose of non-statin LMTs in case of intolerance to statins. Secondary Objectives: To evaluate the efficacy of alirocumab versus placebo on LDL-C levels. To evaluate the effects of alirocumab versus placebo on other lipid parameters. To evaluate the safety and tolerability of alirocumab in comparison with placebo. To evaluate the efficacy, safety, and tolerability of alirocumab after open label treatment. To evaluate the development of anti-alirocumab antibodies.
Efficacy of a New Symbiotic Formulation in Children With Familial Hypercholesterolemia
DyslipidemiaFamilial HypercholesterolemiaProbiotics have been proposed for the treatment of dyslipidemia. the investigators aimed to evaluate efficacy, tolerability and safety of a new symbiotic formulation containing a combination of probiotic and prebiotics and amine in the treatment of children affected by familial hypercholesterolemia (FH).
Bone Marrow Stem Cells as a Source of Allogenic Hepatocyte Transplantation in Homozygous Familial...
HypercholesterolemiaFamilialPatients with homozygous familial hypercholesterolemia has very high serum cholesterol levels despite receiving lipid lowering drugs (e.g. statins, etc). Most of such patients die before the age of 20 due to myocardial infarction, etc. Orthotopic liver transplantation (OLT) is an effective treatment for that. Hepatocyte transplantation is an alternative to OLT that may help to overcome the shortage of donor organs. There have been reports of successful treatment of different kinds of metabolic liver disorders by hepatocyte transplantation. The major problem with hepatocyte transplantation is that the source of hepatocytes is very limited. Bone marrow stem cells are the potential source of hepatocytes. In the in-vitro culture system successful and efficient transdifferentiation of mesenchymal stem cells into hepatocytes has been documented. We have already shown that infusion of mesenchymal stem cells is safe and feasible in cirrhosis (Mohamadnejad M, et al. Arch Iran Med 2007; In Press). In this study, 2 patients with homozygous familial hypercholesterolemia will be included. The bone marrow of healthy volunteers with a normal lipid profile will be taken, then bone marrow mesenchymal stem cells (MSCs) will be cultured, and then MSCs will be trans-differentiate into hepatocytes, and the cells will be infused through the portal vein into the patients. The duration of follow up will be 6 months post-transplantation.