Found 9 translational-medicine-and-human-genetics trials
A listing of translational-medicine-and-human-genetics medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
LCAT Deficiency Disorders: natural history and identification of biomarkers
Familial lecithin:cholesterol acyl transferase (LCAT) deficiency (FLD) is a rare, hereditary disease with no treatment. It is caused by mutations in the gene for LCAT, an enzyme that is made in the liver and plays a role in the body’s ability to process (“metabolize”) cholesterol. People with FLD have very …
Assessing the Impact of Lipoprotein (a) Lowering With TQJ230 on Major Cardiovascular Events in Patients With CVD (Lp(a)HORIZON)
Phase 3
This is a pivotal phase 3 study designed to support an indication for the reduction of cardiovascular risk in patients with established CVD and elevated Lp(a)
Study to determine safety and tolerability of oral lucerastat in adult subjects with Fabry disease
Phase 3
The purpose of this study is to study how a new investigational drug called lucerastat works in research subjects. Taking part in a research study is different from getting regular medical care. The aim of regular care is to improve your health. The aim of a research study is to …
An Open-Label Extension Study of AKCEA-APOCIII-LRX Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
Phase 3
This study seeks to roll over subjects from ISIS 678354-CS3 (the index study; Penn IRB#850921). Patients will receive drug at the dose of 80 mg every 4 weeks (or 50 mg every-4-weeks if their dose was permanently reduced from 80 mg to 50 mg (ISIS 678354 or placebo) in the …
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Patients with Severe Hypertriglyceridemia
This study seeks enroll 390 adult males and females with Severe Hypertriglyceridemia and with fasting TG 500 mg/dL (5.65 mmol/L) that are over the age of 18. The primary objective is to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting TG from Baseline.
Obicetrapib on Lipoprotein Metabolism
Please see section 2 of the protocol, "Study Objectives and Hypotheses" Please see section 2 of the protocol, "Study Objectives and Hypotheses" Please see section 2 of the protocol, "Study Objectives and Hypotheses"
Amryt METRE
This is a 12-month, multicenter, randomized, double-blind, placebo-controlled Phase 3 study to evaluate the safety and efficacy of daily subcutaneous metreleptin treatment in subjects with Familial Partial Lipodystrophy (FPLD). Subjects will be assigned to 1 of 3 groups depending on HbA1c and fasting TGs levels. Participants will complete 10 visits …
Impact of returning PRS on CAD outcomes
This study aims to identify how useful polygenic and integrated risk is in both helping doctors to treat and manage their patients, and identifying people at risk of developing coronary artery disease. Targeted population is previously genotyped individuals in the PMBB. We will calculate an integrated risk score for coronary …
Lp(a) FRONTIERS CAVS
Requesting a reliance agreement with an external IRB. The purpose of this study is to assess if the study treatment, pelacarsen (TQJ230), taken by participants with mild or moderate calcific aortic valve stenosis (CAVS) and elevated lipoprotein(a) [Lp(a)] can safely help slow the progression of CAVS. Patients will be between …
