Condition

Familial Hypercholesterolemia clinical trials and research

• 13Clinical trials
• 15NIH research projects
• 9Countries

Newest Familial Hypercholesterolemia clinical trials

All recruiting trials (13)

• ORIGIN-FH: Newborn screening to detect familial high cholesterol

  This project will test a genetic cheek swab of parents plus blood screening of newborns to find familial high cholesterol in babies born to parents with familial hypercholesterolemia.

  Recruiting University of Wisconsin, Madison · Madison, Wisconsin · NCT07470723

• Registry for hereditary cardiovascular diseases in Brazil

  This study is trying to create a registry of Brazilian patients with hereditary heart diseases to see how common certain genetic changes are in people with these conditions.

  Recruiting at 27 sites Hospital do Coracao · Rio Branco, Acre and 26 other locations · NCT06546137

• Exploring YOLT-101 for treating Familial Hypercholesterolemia

  This study is testing a new treatment called YOLT-101 to see if it can safely lower cholesterol levels in people with familial hypercholesterolemia.

  Recruiting Early Phase 1 RenJi Hospital · Shanghai, Shanghai Municipality · NCT06458010

• Atlantic program to optimize cholesterol-lowering care

  This program will try coordinated outreach with primary care providers to lower LDL-C in people with familial hypercholesterolemia or LDL-C ≥160 mg/dL.

  Recruiting Atlantic Health System · Clark, New Jersey and 1 other locations · NCT06439654

• Observational study on genetic lipid disorders in Italy

  This study looks at families with high cholesterol to see how genetics affect their condition and treatment, using information gathered during regular doctor visits.

  Recruiting Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) · Sesto San Giovanni, Milano · NCT06362473

• Improving detection of familial hypercholesterolemia through lab comments on cholesterol levels

  This study is testing if adding helpful comments about high cholesterol levels in lab reports can lead to more people being referred for familial hypercholesterolemia diagnosis and treatment in Southern Denmark.

  Recruiting at 2 sites Odense University Hospital · Odense, Funen and 1 other locations · NCT05614219

• Comparing direct and indirect methods for screening family members at risk for genetic conditions

  This study is testing two ways to reach out to family members at risk for Long QT Syndrome and Familial Hypercholesterolemia to see which method helps them get screened better, while also offering free genetic testing and counseling.

  Recruiting University of Maryland, Baltimore · Lancaster, Pennsylvania · NCT05348564

• Using 2-HOBA to improve HDL function in patients with Familial Hypercholesterolemia

  This study is testing if a new treatment called 2-HOBA can help improve cholesterol function in people with Familial Hypercholesterolemia.

  Recruiting Phase 2 Vanderbilt University Medical Center · Nashville, Tennessee · NCT04941599

• Screening children and parents for familial hypercholesterolemia

  This study is testing a screening program for families to find out if children and their parents have a genetic condition that causes high cholesterol and could lead to heart problems.

  Recruiting at 6 sites Children's Hospital of Fudan University · Hefei, Anhui and 5 other locations · NCT04529967

• Detecting Familial Hypercholesterolemia in Children

  This study is trying to find out if we can identify children with a genetic condition that causes high cholesterol so they can get early treatment and avoid heart problems later in life.

  Recruiting at 2 sites Institut Investigacio Sanitaria Pere Virgili · Reus, Tarragona and 1 other locations · NCT04370899

• Global collaboration to improve diagnosis and treatment of familial hypercholesterolemia

  This study is trying to find better ways to diagnose and treat familial hypercholesterolemia, a genetic condition that causes high cholesterol and increases heart disease risk, by working with experts from around the world.

  Recruiting Imperial College London · London · NCT04272697

• Registry for Familial Hypercholesterolemia in Russia

  This study is testing how common Familial Hypercholesterolemia is in Russia by screening 18,000 people for high cholesterol levels to help improve diagnosis and treatment.

  Recruiting Russian Cardiology Research and Production Center · Chelyabinsk and 4 other locations · NCT02208869

• Registry for Familial Hypercholesterolemia in Canada

  This study is setting up a registry for people with familial hypercholesterolemia in Canada to help doctors manage their care and support research on this genetic condition.

  Recruiting McGill University Health Centre/Research Institute of the McGill University Health Centre · Montreal, Quebec · NCT02009345

NIH-funded research (15)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Using behavioral nudges to improve family screening for inherited high cholesterol

  This project uses two patient-facing approaches based on behavioral economics to help people with familial hypercholesterolemia get their relatives screened for the condition.

  NIH research Northwestern University · Chicago, United States · NIH-11392680

• Improving Risk Stratification for Familial Hypercholesterolemia (FH)

  A patient-facing look at a study that aims to better predict who with familial hypercholesterolemia is at highest risk for early heart disease.

  NIH research Geisinger Clinic · Danville, United States · NIH-11379914

• Nonviral CRISPR-Cas9 delivery to liver cells to treat familial hypercholesterolemia

  A new approach uses nonviral methods to deliver CRISPR-Cas9 to liver cells and select edited cells with acetaminophen, aiming to permanently lower bad cholesterol in people with familial hypercholesterolemia.

  NIH research Clemson University · Clemson, United States · NIH-11370567

• Implementing multi-component approaches to help blood donors with familial hypercholesterolemia(

  A program to help people who donate blood and have familial hypercholesterolemia get the cholesterol-lowering treatment they need through a blood-donation center.

  NIH research Ut Southwestern Medical Center · Dallas, United States · NIH-11324217

• Helping families find and treat inherited high cholesterol

  This project tests a clinic-based program to help primary care doctors find and treat children and adults with inherited high cholesterol (familial hypercholesterolemia).

  NIH research Geisinger Clinic · Danville, United States · NIH-11304545

• Familial hypercholesterolemia: closing gaps in prevention of heart disease

  A new NIH grant is exploring why people with familial hypercholesterolemia often go undiagnosed or receive uneven care, with the goal of improving early detection and prevention of heart disease.

  NIH research University of California-Irvine · Irvine, United States · NIH-11293026

• Better heart-risk prediction for inherited high cholesterol

  This project aims to give people with familial high cholesterol clearer, more personalized estimates of their future heart disease risk using genetic information, cholesterol measures, and other health factors.

  NIH research Geisinger Clinic · Danville, United States · NIH-11158873

• Finding family members at risk for inherited high cholesterol and Long QT heart rhythm problems

  This project compares contacting relatives directly versus asking patients to tell family members to find people at risk for familial hypercholesterolemia (FH) and Long QT syndrome (LQTS), focusing on Amish communities where two actionable genetic variants are common.

  NIH research University of Maryland Baltimore · Baltimore, United States · NIH-11097301

• Improving treatment for blood donors with high cholesterol

  This study is looking to make it easier for blood donors with Familial Hypercholesterolemia (FH) to get the cholesterol-lowering medications they need, by finding out what challenges they face and testing new ways to help them access treatment better.

  NIH research Ut Southwestern Medical Center · Dallas, United States · NIH-11085084

• Fixing Genes for Familial High Cholesterol

  This project is developing new ways to fix the genes that cause a severe form of very high cholesterol called Familial Hypercholesterolemia.

  NIH research Baylor College of Medicine · Houston, United States · NIH-11059209

• Gene Editing for Familial High Cholesterol

  This project explores a new way to use gene editing to help people with familial hypercholesterolemia, a condition causing very high cholesterol.

  NIH research Clemson University · Clemson, United States · NIH-11047691

• Improving diagnosis and care for familial hypercholesterolemia

  This study is working to make it easier for doctors to spot and treat familial hypercholesterolemia (FH), a genetic condition that causes high cholesterol and can lead to heart problems, so that both kids and adults can get the help they need earlier and stay healthier.

  NIH research Geisinger Clinic · Danville, United States · NIH-11043251

• Using nonviral methods to deliver CRISPR technology for treating high cholesterol

  This study is testing a new way to help people with familial hypercholesterolemia, a genetic condition that causes high cholesterol, by using a safe method to change liver cells and lower cholesterol levels, which could lead to better heart health.

  NIH research Clemson University · Clemson, United States · NIH-10882890

• Understanding and improving risk assessment in familial hypercholesterolemia

  This study is looking at familial hypercholesterolemia (FH), a genetic condition that causes high cholesterol and heart disease risk, to better understand its different types and how they affect heart health, so doctors can give clearer advice to patients from all backgrounds.

  NIH research Geisinger Clinic · Danville, United States · NIH-10872313

• Using mobile health technology to help teens with familial hypercholesterolemia take their statin medication

  This study is looking at how using mobile health apps can help teenagers with high cholesterol from their families remember to take their medication regularly, by finding fun and motivating ways to encourage them.

  NIH research Boston Children's Hospital · Boston, United States · NIH-10685374

Related topics

Quick answers

How many Familial Hypercholesterolemia clinical trials are recruiting right now?

13, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 9 countries. The most sites are in United States (4 trials), China (2 trials), United Kingdom (1 trial), Spain (1 trial), Brazil (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 1, Early Phase 1: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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