Familial hyperlipidemia

This study tested the safety and effectiveness of inclisiran sodium, a cholesterol-lowering injection, in 65 Indian adults with high cholesterol or mixed dyslipidemia. Participants received three doses over 270 days. The goal was to see how well it lowers LDL cholesterol and if a…

This study tested an experimental drug called MGL-3196 (resmetirom) in 116 adults with heterozygous familial hypercholesterolemia, a genetic condition causing very high cholesterol. Participants took either the drug or a placebo daily for 12 weeks. The main goal was to see if the…

This Phase 3 study tested a drug called plozasiran (ARO-APOC3) in 75 adults with familial chylomicronemia syndrome, a rare genetic disorder causing extremely high blood fat levels and risk of pancreatitis. Participants received four injections of the drug or a placebo over severa…

This observational registry followed 260 patients with homozygous familial hypercholesterolemia (a severe genetic condition causing very high cholesterol) who were taking lomitapide. The goal was to monitor long-term safety, especially liver problems and gastrointestinal issues, …

This study looked at the long-term safety of a drug called ARO-APOC3 in adults with dyslipidemia (abnormal fat levels in the blood). Participants who completed a previous 12-month study could continue receiving the drug every 3 or 6 months. The goal was to see if the drug remains…

This study tested whether a special extract from plants (phytosterols and phytostanols) can help lower cholesterol levels in adults with high cholesterol. 42 people took either the extract or a placebo for a period of time. The main goal was to see if the extract could reduce tot…

This study tested a low dose (10 mg) of the statin drug rosuvastatin in 242 Korean adults with high cholesterol or heart disease risk. The goal was to see how much it lowered 'bad' LDL cholesterol after 8 weeks. Results help guide cholesterol treatment in this population.

This phase 2b trial tested plozasiran, an experimental drug, in 353 adults with mixed dyslipidemia (high triglycerides and cholesterol). Participants received either plozasiran or a placebo, and researchers measured changes in blood fat levels over 48 weeks. The goal is to see if…

This study tested whether a probiotic called Lactobacillus mucosae DPC6426 can lower cholesterol in healthy adults with mildly high levels. Over 12 weeks, 94 participants took either the probiotic or a placebo. The goal was to see if this natural approach could offer a heart-heal…

This completed Phase 3 trial tested whether taking two cholesterol-lowering drugs together works better than one alone. 184 adults with high cholesterol or mixed dyslipidemia took either the combo or a single drug daily for 8 weeks. The main goal was to see how much LDL ('bad') c…

This Phase 3 trial tested an oral drug called enlicitide decanoate (MK-0616) in 303 adults with heterozygous familial hypercholesterolemia, an inherited condition causing very high cholesterol. Participants took either the drug or a placebo daily for up to 52 weeks. The main goal…

This study tested a daily pill called ETC-1002 in 130 adults with very high LDL ("bad") cholesterol who either didn't respond well to statins or had side effects from them. Over 52 weeks, researchers checked how safe the drug was and how much it lowered cholesterol. The goal was …

This study tested a drug called inclisiran in teenagers aged 12 to 18 who have a rare inherited condition that causes extremely high cholesterol. The goal was to see if the drug safely lowers their LDL (bad) cholesterol. Participants received either inclisiran or a placebo for on…

This study tested a drug called inclisiran in teenagers aged 12 to 18 who have a genetic condition that causes very high cholesterol. The goal was to see if the drug is safe and can lower bad cholesterol. Participants received either inclisiran or a placebo for one year, then eve…

This study tested whether a drug called alirocumab, which lowers cholesterol, can reduce heart disease risk in people with HIV. The trial involved 118 adults aged 40 and older with well-controlled HIV and high heart disease risk. Participants received either alirocumab or a place…

This pilot study tested whether adding plant sterols (phytosterols) and/or krill oil to a heart-healthy Brazilian diet can lower cholesterol in people with familial hypercholesterolemia (FH), an inherited condition causing very high cholesterol. Fifty-eight adults with FH were ra…

This early-stage study tested a new medicine called ARO-ANG3 in 93 people, including healthy volunteers and patients with high cholesterol or high triglycerides. The main goal was to check if the drug is safe and how the body processes it. This research helps determine if the dru…

This early-stage study tested a new medicine called ARO-APOC3 in healthy volunteers and people with severely high triglycerides or a rare genetic condition called familial chylomicronemia syndrome. The main goal was to check safety and how the drug moves through the body. The stu…

This Phase 3 trial tested the drug SHR-1918 in 55 people with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol. Participants received either the drug or a placebo, and researchers measured how much their LDL (bad) choles…

This study tested two different chemotherapy drugs (Treosulfan and Busulfan) given before a stem cell transplant in 106 children with serious non-cancer diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug le…

This phase 2b trial tested an experimental drug called ARO-ANG3 in 204 adults with mixed dyslipidemia, a condition with high triglycerides and cholesterol. Participants received injections of the drug or a placebo over several months. The goal was to see if the drug safely lowers…

This Phase 3 trial tested a daily pill combining two drugs, obicetrapib and ezetimibe, in 407 people with inherited high cholesterol or heart disease. Participants took the combination or a placebo on top of their usual cholesterol meds. The main goal was to see if the combo lowe…

This study tested a new drug called obicetrapib in over 2,500 people with high cholesterol that wasn't well controlled by their current treatments. Participants took either a 10 mg obicetrapib pill or a placebo daily for about 6 months. The main goal was to see if obicetrapib cou…

This large Phase 3 trial tested an experimental oral drug called enlicitide decanoate (MK-0616) in nearly 3,000 adults with high cholesterol, including those with a genetic form. The goal was to see if the drug safely lowers 'bad' LDL cholesterol more than a placebo over 24 weeks…

This early-phase study tested a single dose of VSA003 in 36 healthy Chinese adults to see if it is safe and how the body handles it. The drug aims to help control high cholesterol and triglycerides. Participants received either VSA003 or a placebo, and researchers monitored for s…

This phase 2 trial tested an investigational drug called SHR-1918 in 26 people with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol. The study measured how much the drug lowered LDL (bad) cholesterol after 12 weeks. All…

This study tested whether offering genetic screening to younger heart attack patients (under 60) can uncover hidden cases of Familial Hypercholesterolemia (FH), an inherited condition causing very high cholesterol. Researchers recruited 140 patients admitted with a heart attack a…

This study tested a new method called global metabolomic profiling to diagnose inborn errors of metabolism, a group of rare genetic disorders. Researchers compared this approach to traditional testing in 240 participants. The goal was to see if the new method could more accuratel…

This study looks at people with a rare inherited condition that causes extremely high cholesterol and early heart disease. Researchers used special tests to measure artery hardening before it becomes life-threatening. The goal was to gather information to help develop future trea…

This study created a registry of medical records from women with inborn errors of metabolism—rare conditions that affect how the body turns food into energy. Researchers collected data from past or current pregnancies and followed babies for one year after birth. The goal was to …

This study aimed to find new gene changes that cause rare lipid disorders. Researchers used advanced genetic testing to look at many genes at once in 140 people and their relatives. The goal was to discover causes that older methods missed.

This completed study looked at how the social networks of caregivers affect their stress and health when caring for someone with an inherited disease. Researchers surveyed over 680 participants, including family members and formal caregivers, to understand caregiving burden and s…

This study creates a registry for people with familial hypercholesterolemia (FH) in Greece, a genetic condition that causes very high cholesterol and early heart disease. Researchers will screen adults aged 18 to 80 to learn how common FH is and describe the health of those affec…

This study looked at how fat-carrying particles called lipoproteins work in healthy people and those with high cholesterol or other lipid disorders. Researchers injected tiny, safe amounts of radioactive lipoproteins to track them in the body. The goal was to better understand ho…

This study looked at 99 people with high heart risk to understand how a protein called CETP affects plaque buildup in arteries. Researchers measured CETP activity, artery thickness, and calcium scores. The goal was to learn more about heart disease, not to test a new treatment.

This study explored how teenagers and their parents react emotionally and mentally when they learn about genetic risks for conditions like hereditary breast cancer, Lynch syndrome, or high cholesterol. Researchers surveyed 162 participants using questionnaires and interviews to m…

This study looked at medical records from over 11,900 adults in China who took either inclisiran or PCSK9 mAbs to lower their high cholesterol. Researchers wanted to see how well these drugs worked in everyday medical practice, not just in controlled trials. The study found that …

This study looked at how an experimental drug called AZD0780 affects the way the body processes metformin, a common diabetes medicine. Fourteen healthy volunteers took both drugs, and researchers measured drug levels in the blood. The goal was purely to gather information on drug…

This early-stage study tested a new drug called DW5124 in 55 healthy adults to see how safe it is and how the body processes it. Researchers also checked whether taking the drug with food changes its effects. The goal was to gather basic information before testing the drug in peo…

This study offered free genetic testing to immediate family members of children found to have a genetic marker for familial hyperlipidemia (FH), a condition that causes very high cholesterol and early heart disease. The goal was to see if large-scale testing can identify at-risk …

This study looked at 2000 adults with early heart disease to understand how inherited high cholesterol (familial dyslipidemia) contributes to heart attacks. Researchers reviewed medical records to find patterns in cholesterol levels. The goal was to highlight the need for better …

This study looked at how many girls and young women aged 11 to 20 with chronic diseases (like diabetes or immune conditions) got the HPV vaccine, compared to those without chronic illness. Researchers reviewed records of 223 participants from a hospital in France. The goal was to…

This study tested whether a whole-food plant-based diet, compared to a standard North American diet, can lower LDL cholesterol in 50 adults with heterozygous familial hypercholesterolemia (HeFH). Participants followed each diet for 28 days in a crossover design. Researchers also …

This completed study tested whether a generic bempedoic acid tablet works the same as the brand-name drug Nilemdo in 32 healthy adults. Participants took a single dose of each under fasting conditions, and researchers measured drug levels in the blood. The goal was to see if the …