View clinical trials related to Hypercholesterolemia, Familial.
Filter by:Statins have been shown to reduce LDL cholesterol (LCLc) levels, stabilizing atheromatous plaque, reversing endothelial dysfunction and decreasing thrombogenesis. Novel pharmacological approaches, such as PCSK9 inhibitors (PCSK9i), effectively reduce LDL-c. In the clinical setting, there are cases of dyslipidemia showing lack of response to statin, known as statin-resistant familial hypercholesterolemia (SR-FH), where patients maintain a high cardiovascular risk despite statin therapy. Then, therapeutic alternatives are required. PCSK9i has shown to reduce cholesterol levels and risk of cardiovascular disease, particularly in patients with statin-resistant familial hypercholesterolemia; and recently, it has been hypothesized that PCSK9i have an effect on inflammation. Aim. To evaluate the effect of anti-PCSK9 treatment on markers related to the inflammatory response in patients with SR-FH. Methods. Non-randomized, non-controlled, before-after comparison, quasiexperimental, single-center study on patients older than 18 years, with diagnosis statin-resistant FH (SR-FH), who were attended at the Cardiology Department, Centro Médico Nacional "20 de Noviembre ISSSTE", Mexico City. SR-FH was defined as symptomatic cardiovascular disease accompanied by LDL-C concentration higher than 160 mg/dL despite maximally tolerated statin dose. Clinical-demographic and anthropometry data were collected during a direct interview. Blood sample was processed to obtain glycated hemoglobin complete blood count and serum lipids. Likewise, flow cytometry was used to characterize baseline circulating M1-, M2-macrophages and monocytes. Multiplexing of plasma samples was used to compare plasma fraktaline, IL-1, IL-4, IL-6, IL-8, IL-10, MCP-1 and TNF-alpha. Endpoints consisted of: 1) lower serum lipids; 2) modification of pro-inflammatory mediators (neutrophils, lymphocytes, NtLR, soluble pro-inflammatory cytokines). Quatitative data were resumed as mean ± SD; while categorical data as n(%).One-way T-test was applied. Statistical significance was considered if p <0.05.
To evaluate the lipoprotein kinetics of subjects enrolled in the R1500-CL-1331 clinical trial (NCT02265952) to assess the mechanism by which the evinacumab may affect lipid levels in HoFH subjects.
To test the hypothesis that in patients with a clinical diagnosis of familial hypercholesterolemia (FH), genetic testing and identification of a causative mutation might enhance the success of family-based cascade screening.
The purpose of the study is to evaluate prospectively the impact of different system alerts on the prescription of lipid panels to pediatric Geisinger patients (9-11 years old), as per the now-universal guidelines. This will help quantify the relative effectiveness of different alerts and combinations of alerts on provider prescribing behavior and patient uptake of screening.
The clinical Investigation will be performed to compare the safety and effectiveness of the CE certified and established lipoprotein apheresis systems MONET vs. DALI and DIAMED vs. DALI for optimizing the individual therapy of patients with severe dyslipidemia using established and novel efficacy parameters.
The purpose of this protocol is to identify and screen potential candidates for future enrollment in a gene therapy clinical trial for HoFH.
Low Density Lipoprotein (LDL)-apheresis refers to a procedure in which blood taken from a patient's vein is cleaned from pathogenic substances, e.g. cholesterol, outside the body and then given back to the patient. In the DALI (Direct Adsorption of Lipoproteins)-system whole blood is pumped over an adsorber containing beads that selectively bind LDL-cholesterol. The MONET (Membrane filtration Optimized Novel Extracorporeal Treatment)-system works with plasma which is cleaned by filtration. This study comprises the recording of safety and efficacy data from patients treated either with the DALI or MONET-system over a period of 2 years.
The objective of this study is to evaluate the efficacy and tolerability of adding anacetrapib to ongoing statin therapy in participants with heterozygous familial hypercholesterolemia (HeFH).
The primary objective of this study was to evaluate the effect of 12 weeks of subcutaneous evolocumab (AMG 145), compared with placebo, on percent change from baseline in LDL-C in adults with heterozygous familial hypercholesterolemia (HeFH).
Patients 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.