Clinical Trials for Cholesterol

High cholesterol, also known as hypercholesterolemia, is an extremely common condition affecting roughly 86 million American adults.1 Most cases of high cholesterol can be treated with changes in diet and exercise habits.2 However, in many cases, medicine is needed to treat high cholesterol, including for individuals at high risk of developing a heart attack or stroke. In addition, some people have a genetic form of high cholesterol known as familial hypercholesterolemia (FH), which can’t be controlled with lifestyle changes alone.3 The U.S. Food and Drug Administration (FDA) has approved several cholesterol-lowering medications, including statins and bile acid sequestrants.4

Doctors and researchers continue to look for new ways to treat high cholesterol and FH . Many of these are experimental therapies that are newly developed, while others are previously approved cholesterol medications that are seeking approval for use in children, including teenagers.

New treatments must first go through clinical trials to be approved by the FDA. These studies recruit people with hyperlipidemia or FH to see if the treatment helps to lower cholesterol. The clinical trials discussed below are just a fraction of current studies.

New Medications for High Cholesterol

Currently, there are several new therapies in clinical trials that are targeting proteins involved in high cholesterol in new ways. Hyperlipidemia is diagnosed when your low-density lipoprotein (LDL) or “bad cholesterol” levels are too high. You have LDL receptors on the outside of your liver cells, which attach to LDL cholesterol to help destroy it.5

In some individuals, PCSK9 proteins break down your LDL receptors, preventing cholesterol from being broken down. As a result, your LDL levels increase. Researchers have developed PCSK9 inhibitors that block this protein, helping to restore your body’s ability to clear LDL cholesterol.

There are currently several PCSK9 inhibitors available, but more have been developed to target the protein in new ways. Many of these medications need to be injected to be effective — however, researchers have developed new inhibitors that can be taken as pills, which are typically preferred over needles. Examples of experimental PCSK9 inhibitors include lerodalcibep and AZD0708.6,7

Researchers have also found another way to treat hyperlipidemia by blocking the transfer of cholesterol from high-density lipoproteins (HDL), or “good cholesterol,” to LDL proteins. One example is obicetrapib.8,9

Previously Approved Treatments

Several FDA-approved treatments for high cholesterol are back in clinical trials to test that they’re safe and effective for children and teenagers to use. According to the Centers for Disease Control and Prevention (CDC), almost 7 percent of children and teenagers aged 6 to 19 have elevated cholesterol levels.1

In some cases, hyperlipidemia can be treated with a healthy diet and exercise. However, these lifestyle changes may not always help. Currently, only a handful of treatments are approved for treating children and teenagers with FH.10,11

Examples of medications being studied for treating children and teenagers with high cholesterol include:

  • Bempedoic acid (Nexletol®): Previously approved for treating FH in adults by reducing cholesterol made by your liver; it’s now being studied for treating FH in children and teenagers12,13
  • Inclisiran (Leqvio®): A small piece of RNA that blocks the PCSK9 gene to lower LDL cholesterol; previously approved for treating FH in adults, it’s now being studied for treating teenagers with FH14,15

Doctors and researchers also monitor drugs for several years after their approval to ensure they’re safe in the long term. These are known as observational clinical trials, and they help the FDA look for any unwanted side effects and adverse events (cancer, heart problems, etc.). If you have been prescribed long-term cholesterol medication, you may be able to participate.16

Joining a Cholesterol Clinical Trial

Find Clinical Trials Near Me

Interested in clinical trials? Simply search by postal code and type of condition to see what’s going on in your area.


If you or your child are living with hyperlipidemia or FH, you may be interested in learning more about clinical trials. Start by looking for studies at hospitals or trial centers nearby that you or your child may be eligible for. In every research study, there are certain requirements set by investigators that must be met to join the clinical trial. These inclusion and exclusion criteria are set to help ensure accurate data collection and safety during the study. To join a cholesterol trial, you or your child may have to:

  • Meet certain age and health requirements
  • Have cholesterol levels within certain thresholds
  • Have previously taken other cholesterol-lowering medications

Choosing to join a clinical trial is a major decision — some people join to experience potential benefits from experimental therapies, while others are eager to help advance science. No matter your decision to join, it should be made with your best interests in mind.