Clinical trials
Familial Hypercholesterolemia clinical trials
Below are recruiting familial hypercholesterolemia clinical trials, each written for real people, not researchers. We’re tracking 43 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07470723Recruiting
Early cholesterol check for newborns of parents with lipid disorder
This study tests whether checking a newborn's blood spot for a genetic condition that causes very high cholesterol can help detect it early. It may help families understand their baby's risk and start care sooner if needed.
Madison, WisconsinAges 18 years+ - NCT04370899Recruiting
Early checkup for inherited high cholesterol in children
This study looks at children who may have inherited high cholesterol, especially when one parent is affected. It may help doctors detect the condition earlier and understand how it runs in families.
Reus, TarragonaAges 2–18 - NCT06597019RecruitingPhase 3
Study of inclisiran for children with high cholesterol
This trial tests a medicine called inclisiran to lower high cholesterol in children ages 6 to 12 with a genetic condition called heterozygous familial hypercholesterolemia (HeFH). It may help children who still have high cholesterol despite taking other treatments.
San Francisco, CaliforniaAges 6–11 - NCT06597006RecruitingPhase 3
Study of inclisiran for children with homozygous familial high cholesterol
This trial tests a new medicine called inclisiran to lower very high cholesterol in children aged 2 to 12 who have a specific genetic condition (homozygous familial hypercholesterolemia, or HoFH). You may not need to change your current cholesterol treatments, and the study will check if inclisiran can help reduce your LDL cholesterol further.
San Francisco, CaliforniaAges 2–11 - NCT05750667Enrolling by invitation
Family screening for familial high cholesterol
This trial is about helping families with familial hypercholesterolemia (a genetic condition that causes very high cholesterol) get screened. It tests a way to reach close relatives so they can learn their risk and consider next steps.
Philadelphia, PennsylvaniaAges 18 years+ - NCT04929457Enrolling by invitation
Screening to detect inherited high cholesterol
This study tests a digital screening method to help identify and diagnose familial hypercholesterolemia, an inherited condition that can cause very high cholesterol. If you participate, you may be invited to complete screening steps to see whether they can find the condition early.
StockholmAges Any age - NCT02208869Recruiting
Registry for inherited high cholesterol (familial hypercholesterolemia)
This registry collects information from people with inherited high cholesterol to better understand the condition and care needs. You may be invited to share blood and medical test results, especially your cholesterol and related health history.
ChelyabinskAges 7–80 - NCT07473843RecruitingPhase 3
Study of zodasiran for teens with homozygous familial hypercholesterolemia
This study tests an experimental medicine called zodasiran in teenagers who have a rare inherited condition that causes very high cholesterol. The goal is to see if it can lower cholesterol levels and reduce the risk of heart problems.
St Louis, MissouriAges 12–17 - NCT06634160Recruiting
Fatty liver disease in people with high cholesterol
This study checks how common fatty liver disease is in people with a genetic form of high cholesterol. It may help researchers understand the link between these two conditions.
AngersAges 35 years+ - NCT06458010RecruitingEarly Phase 1
Gene therapy trial for inherited high cholesterol
This trial tests a new gene therapy called YOLT-101 for people with familial hypercholesterolemia, a genetic condition that causes very high cholesterol. The treatment aims to lower cholesterol levels and reduce heart risks.
Shanghai, Shanghai MunicipalityAges 18–75 - NCT06125847RecruitingEarly Phase 1
Gene therapy for inherited high cholesterol
This trial tests a one-time gene therapy to lower extremely high cholesterol in people with a rare inherited condition. It may help reduce heart attack risk in those who haven't responded to standard treatments.
Xi'an, ShaanxiAges 12–55 - NCT04941599RecruitingPhase 2
2-HOBA to improve “good cholesterol” function in FH
This Phase 2 trial tests whether 2-HOBA can improve how “good cholesterol” (HDL) works in people with familial hypercholesterolemia (FH). If HDL works better, it may help lower heart risk markers. You would need to meet several heart, health, and medication-related safety checks.
Nashville, TennesseeAges 18–69 - NCT04529967Recruiting
Screening kids for inherited high cholesterol
This study checks children ages 1 to 3 during routine care to see who may have familial hypercholesterolemia, a genetic condition that causes high cholesterol. Early identification can help families learn about next steps and prevention.
Hefei, AnhuiAges 1–3 - NCT07491172RecruitingPhase 1
Trial of CTX310 for stubborn high cholesterol and fats
This trial tests a new medicine, CTX310, for people with persistently high levels of fats (triglycerides) and cholesterol that have not improved with standard treatments. It aims to see if the drug is safe and tolerable.
Hialeah, FloridaAges 18–75 - NCT07465263RecruitingPhase 3
Study of SYH2053 Injection for High Cholesterol in HeFH
This study tests a new injection called SYH2053 for people with HeFH, a genetic condition that causes very high cholesterol. It looks at whether the injection can safely lower LDL cholesterol when added to standard statin therapy.
Beijing, Beijing MunicipalityAges 18 years+ - NCT07447648Recruiting
Evinacumab for plaque buildup in HoFH
This observational study looks at how adding evinacumab to standard cholesterol-lowering therapy affects plaque in the heart arteries of people with homozygous familial hypercholesterolemia (HoFH). It compares those who started evinacumab within the last 2 years to those on standard therapy alone, using CT scans taken before and after treatment.
ParisAges 12 years+ - NCT06555120Recruiting
Screening kids for inherited high cholesterol
This study checks children for a genetic condition that causes very high cholesterol from a young age. If found early, steps can be taken to lower heart disease risk later in life.
AmiensAges 2–18 - NCT01109368Recruiting
Registry for people with inherited very high cholesterol
This trial is a registry that collects information from people with homozygous familial hypercholesterolemia (an inherited condition causing extremely high “bad” cholesterol). It may help researchers better understand the condition and support future studies.
New York, New YorkAges Any age - NCT05284513Enrolling by invitation
Care team study for families with high cholesterol
This study tests a “reach everyone” approach in primary care clinics to improve care for people with familial hypercholesterolemia (families with very high cholesterol). It may help families get recognized sooner and get the right follow-up.
Multiple Locations, PennsylvaniaAges 26 years+ - NCT02009345Recruiting
Study for inherited high cholesterol in families
This trial focuses on people with familial hypercholesterolemia (FH), an inherited condition that causes very high cholesterol. It aims to better understand and manage severe cholesterol problems that raise heart risk, especially in families.
Montreal, QuebecAges Any age - NCT07058077RecruitingPhase 2/Phase 3
Study of oral cholesterol-lowering pill in children with HeFH
This study tests an experimental daily pill (enlicitide decanoate) that lowers LDL ('bad') cholesterol in children and teens with a genetic condition called heterozygous familial hypercholesterolemia (HeFH). It aims to see if this medicine can safely help reduce their high cholesterol levels.
Wilmington, DelawareAges 6–17 - NCT06960902Recruiting
Study of Heart Artery Plaque in Familial Hypercholesterolemia
This study uses a heart CT scan to look for plaque buildup in the arteries of people with familial hypercholesterolemia (a genetic high-cholesterol condition). It aims to understand why some people with FH develop heart disease while others don't.
DijonAges 40 years+ - NCT06568471RecruitingPhase 3
Study of HST101 for high cholesterol in Chinese patients
This study tests a new injectable medication called HST101 for lowering cholesterol in people who are already taking cholesterol pills but still have high levels. It may help if you have heart disease or are at high risk.
Beijing, Beijing MunicipalityAges 18 years+ - NCT07421297RecruitingPhase 3
Study of SYH2053 for high cholesterol in Chinese adults
This trial tests a new medication called SYH2053 for people with high cholesterol or mixed high cholesterol and triglycerides. If you have high LDL cholesterol and are not taking strong cholesterol medicines, you may be able to join. The study will see if this drug is safe and works well.
Beijing, Beijing MunicipalityAges 18 years+
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Familial Hypercholesterolemia trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for familial hypercholesterolemia?
- Yes. Clin2 currently lists 43 recruiting familial hypercholesterolemia studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a familial hypercholesterolemia trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a familial hypercholesterolemia trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
- Heterozygous Familial Hypercholesterolemia
- Heterozygous Familial Hypercholesterolemia (HeFH)
- Homozygous Familial Hypercholesterolemia
- Homozygous Familial Hypercholesterolemia (HoFH)
- Non-familial Hypercholesterolemia and Mixed Hyperlipidemia
- Hypercholesterolemia
- Familial Mediterranean Fever
- Familial Adenomatous Polyposis
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.