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Type IIa hyperlipidaemia – Trials in Disease

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Ongoing Clinical Trials for Type IIa Hyperlipidaemia

There are currently 10 clinical trials investigating new treatments for Type IIa Hyperlipidaemia, also known as heterozygous familial hypercholesterolemia. These studies are being conducted across multiple European countries and are testing medications designed to lower LDL cholesterol levels in both adults and children with inherited forms of high cholesterol. The trials include various approaches, from oral tablets to injectable medications, and range from short-term studies to long-term safety assessments.

Clinical trial locations

Study of laroprovstat (AZD0780) tablets to lower cholesterol levels in adults with inherited high cholesterol condition (heterozygous familial hypercholesterolemia)

This trial is evaluating a new medication called laroprovstat (AZD0780), which comes as a tablet taken by mouth. The study is designed for adults who have heterozygous familial hypercholesterolemia, an inherited condition that causes high levels of LDL cholesterol in the blood. The main purpose is to see how well AZD0780 works compared to placebo in lowering LDL cholesterol levels over 52 weeks.

Inclusion criteria: Participants must be at least 18 years old with confirmed heterozygous familial hypercholesterolemia, either through genetic testing or clinical diagnosis. They need to have specific cholesterol levels: at least 55 mg/dL for those with heart disease or at least 70 mg/dL for those without. Participants should already be taking the highest tolerated dose of statin medication for at least 28 days, preferably also with ezetimibe.

Exclusion criteria: The trial excludes people below 18 or above 65 years old, pregnant or breastfeeding women, those with severe allergic reactions to medications, current participants in other trials, uncontrolled high blood pressure, recent heart attack or stroke within 6 months, severe liver or kidney disease, active cancer, inability to provide consent, substance abuse in the past year, medications that may interact with the study drug, mental health conditions affecting participation, and history of non-compliance with treatments.

Study focus: The trial will measure changes in LDL cholesterol and related substances at 12, 28, and 52 weeks. Participants will continue their usual cholesterol-lowering medications throughout the study and will receive either AZD0780 or placebo without knowing which they are taking. The medication aims to target specific pathways in cholesterol metabolism to reduce levels of bad cholesterol and other related lipids.

Investigational drug: AZD0780 is being tested to see if it can help lower LDL cholesterol in patients with the genetic condition that makes it difficult to manage cholesterol with standard medications alone.

Study on Inclisiran, Alirocumab, and Rosuvastatin for Children with Familial Hypercholesterolemia

This trial is testing different treatment combinations for children aged 10 to 15.5 years with heterozygous familial hypercholesterolemia. The study compares rosuvastatin (an oral medication) combined with either alirocumab or inclisiran (both given as injections under the skin) against rosuvastatin alone. The goal is to determine which treatment combination is most effective in helping children reach healthier cholesterol levels after 104 weeks.

Inclusion criteria: Children must obtain informed written consent, be between 10 and 15.5 years old, and have specific LDL cholesterol levels. Those with LDL at least 190 mg/dL can participate regardless of family history. With a positive family history, LDL must be at least 160 mg/dL. With confirmed genetic mutation in at least one parent, LDL must be at least 130 mg/dL. Menstruating girls need a negative pregnancy test and must agree to use contraception if applicable.

Exclusion criteria: Children without heterozygous familial hypercholesterolemia, those outside the age range, unwilling or unable to follow study procedures, those with interfering medical conditions or medications, pregnant or breastfeeding participants, recent participants in other trials, and those with allergic reactions to study medications cannot participate.

Study focus: The trial lasts about two years with regular check-ups to monitor cholesterol levels and overall health. It will also assess the impact on artery thickness using carotid intima-media thickness measurements and evaluate quality of life through questionnaires. The study aims to find better ways to manage high cholesterol in children with this genetic condition.

Investigational drugs: Alirocumab is an injection that blocks a protein in the liver to help remove LDL cholesterol. Rosuvastatin is an oral statin that reduces cholesterol production by the liver. Inclisiran is an injection that targets a genetic process to help the liver remove more LDL cholesterol from the blood.

Study on the Effectiveness of Enlicitide Chloride in Reducing Cardiovascular Events in Patients with Atherosclerotic Cardiovascular Disease

This trial evaluates MK-0616 (enlicitide chloride) in people at high risk for serious heart problems due to atherosclerotic cardiovascular disease. The study examines whether this oral medication can reduce the risk of major heart events such as heart attacks or strokes compared to placebo. Participants will be randomly assigned to receive either MK-0616 or placebo and will be monitored throughout the study.

Inclusion criteria: Participants must be at least 18 years old with a history of major ASCVD events or at high risk for a first major event. They need specific cholesterol levels measured at screening and must be taking moderate or high-intensity statin medication. For those with a history of major events, LDL-C should be at least 70 mg/dL or non-HDL-C at least 100 mg/dL. For high-risk individuals, LDL-C should be at least 90 mg/dL or non-HDL-C at least 120 mg/dL. All participants must be on stable doses of cholesterol-lowering medications for at least 30 days.

Exclusion criteria: Those with severe allergic reactions to medications, uncontrolled high blood pressure, severe liver or kidney disease, recent heart attack or stroke, active cancer, pregnancy or breastfeeding, current participation in other trials, and certain medical conditions that could interfere with the study cannot participate.

Study focus: The trial will monitor participants to see if MK-0616 can delay or prevent serious cardiovascular events. Regular assessments will include cholesterol level monitoring and evaluation of heart health markers throughout the study period, which is estimated to conclude in November 2029.

Investigational drug: MK-0616 targets specific pathways involved in cholesterol metabolism to reduce the risk of coronary heart disease-related events and works to lower cholesterol levels in the blood.

Study on the Safety and Effectiveness of Inclisiran for Children Aged 6 to Under 12 with Familial Hypercholesterolemia

This study focuses on children aged 6 to under 12 years with heterozygous familial hypercholesterolemia. It evaluates the safety and effectiveness of inclisiran, an injectable medication designed to lower LDL cholesterol levels. The study is divided into two years: in the first year, participants receive either inclisiran or placebo; in the second year, all participants receive inclisiran.

Inclusion criteria: Participants must be male or female between 6 and under 12 years old at screening with a diagnosis of heterozygous familial hypercholesterolemia confirmed by genetic testing or clinical characteristics. They must have fasting LDL-C levels greater than 130 mg/dL. Those aged 8 to under 12 should be on optimal statin doses unless intolerant, possibly with other treatments like ezetimibe. For those under 8, treatment use is determined by the study doctor. Any existing cholesterol medications must be at stable doses for at least 30 days.

Exclusion criteria: Children not between 6 and 12 years old, those without heterozygous familial hypercholesterolemia, or without elevated LDL-C levels cannot participate.

Study focus: The primary goal is to demonstrate that inclisiran is superior to placebo in reducing LDL cholesterol by Day 330. The study continues monitoring through Day 720, tracking changes in cholesterol levels, growth, vital signs, and development to ensure safety and effectiveness.

Investigational drug: Inclisiran works by helping the liver remove more LDL cholesterol from the bloodstream. It is a small interfering RNA therapy that targets and reduces production of a protein involved in cholesterol regulation.

Study on the Safety and Effectiveness of Inclisiran in Children Aged 2 to Under 12 with Homozygous Familial Hypercholesterolemia

This trial tests inclisiran in children aged 2 to under 12 years with homozygous familial hypercholesterolemia, a rare genetic disorder causing very high cholesterol levels from birth. The study is structured in two parts: a one-year comparison between inclisiran and placebo, followed by a second year where all participants receive inclisiran.

Inclusion criteria: Participants must be male or female aged 2 to under 12 years with genetically confirmed homozygous familial hypercholesterolemia and fasting LDL-C greater than 130 mg/dL. They should be on optimal statin doses unless intolerant, possibly with other treatments like ezetimibe. Any cholesterol-lowering medications must be stable for at least 30 days. Those undergoing LDL-apheresis must have been on a stable regimen for at least 3 months with schedules remaining stable throughout the study.

Exclusion criteria: Children without homozygous familial hypercholesterolemia, those not between 2 and under 12 years old, or without elevated LDL-C levels cannot participate.

Study focus: The trial evaluates how much inclisiran can lower LDL cholesterol by Day 330 and continues monitoring through Day 720. Regular assessments check for side effects, monitor growth, and evaluate development. The study aims to ensure the medication is both safe and effective for this young age group.

Investigational drug: Inclisiran is given as an injection under the skin and works by inhibiting a protein involved in cholesterol production, helping the liver remove more LDL cholesterol from the blood.

Study on MK-0616 for Reducing Heart Disease Events in Patients with Atherosclerotic Cardiovascular Disease

This trial evaluates MK-0616, an oral film-coated tablet, in people at high risk for cardiovascular issues due to atherosclerotic cardiovascular disease. The study examines whether the medication can help delay or prevent serious heart-related events such as heart attacks, strokes, or other complications.

Inclusion criteria: Participants must be at least 18 years old with a history of major ASCVD events or at high risk for a first major event. Specific fasting cholesterol levels are required at screening: for those with major event history, LDL-C at least 70 mg/dL or non-HDL-C at least 100 mg/dL; for high-risk individuals, LDL-C at least 90 mg/dL or non-HDL-C at least 120 mg/dL. All must be taking moderate or high-intensity statins on stable doses for at least 30 days.

Exclusion criteria: Those with different heart conditions unrelated to ASCVD, individuals outside the specified age range, those not part of specified trial groups, or those in vulnerable populations cannot participate.

Study focus: The trial monitors participants to evaluate if MK-0616 can increase the time before experiencing major cardiovascular events. Regular follow-up visits include monitoring for events like heart attacks or strokes and blood tests to measure cholesterol changes. The study is estimated to conclude by November 2029.

Investigational drug: MK-0616 targets specific pathways involved in cholesterol metabolism to reduce plaque buildup in arteries and improve heart health.

Study of Bempedoic Acid for Children Aged 6-17 with Familial Hypercholesterolemia

This study focuses on bempedoic acid, available as both film-coated tablets and oral suspension, for children aged 6 to 17 years with heterozygous familial hypercholesterolemia. The trial examines how the body processes the medication and how it affects cholesterol levels over an 8-week treatment period.

Inclusion criteria: Participants must have parental consent and child agreement, be between 6 and 17 years old, weigh at least 16 kilograms, and have heterozygous familial hypercholesterolemia confirmed by genetic test or cholesterol levels with family history. They must be taking approved cholesterol medications at stable doses for at least 4 weeks (6 weeks for fibrates, but gemfibrozil is not allowed with statins) and have fasting LDL-C at least 130 mg/dL. Females must not be pregnant, planning pregnancy, or breastfeeding, and must agree to birth control if sexually active.

Exclusion criteria: Those not between 6 and 17 years old, without heterozygous familial hypercholesterolemia, or in vulnerable populations cannot participate.

Study focus: The trial monitors medication levels in the blood and observes cholesterol changes during the 8-week period. It also assesses how well children tolerate the medication, including taste and ease of swallowing. The study aims to provide information about safety and effectiveness in young patients.

Investigational drug: Bempedoic acid works by inhibiting an enzyme called ATP citrate lyase, which plays a role in cholesterol synthesis in the liver, thereby helping to lower cholesterol levels.

Study of MK-0616 for Adults with Familial Hypercholesterolemia

This trial tests MK-0616 (enlicitide chloride), an oral film-coated tablet, for adults with heterozygous familial hypercholesterolemia. The study evaluates how effective and safe the medication is in reducing LDL cholesterol levels over 52 weeks, with main assessments at 24 and 52 weeks.

Inclusion criteria: Participants must have a diagnosis of heterozygous familial hypercholesterolemia confirmed by locally accepted methods. They need LDL-C levels of at least 55 mg/dL or 70 mg/dL depending on medical history, must be treated with moderate or high-intensity statins, and be on stable doses of all cholesterol-lowering therapies with no planned changes.

Exclusion criteria: Details not fully specified in the source data, but standard exclusions likely apply.

Study focus: The double-blind study randomly assigns participants to receive either MK-0616 or placebo while continuing existing cholesterol-lowering therapies. The 24-week treatment period focuses on evaluating LDL-C level changes, with extended follow-up potentially continuing to 52 weeks for long-term assessment.

Investigational drug: MK-0616 inhibits a specific protein involved in cholesterol regulation, reducing LDL cholesterol levels in the bloodstream and potentially providing a new treatment option for managing this condition.

Study on Long-Term Effects of Lerodalcibep for Lowering Cholesterol in Patients with Familial Hypercholesterolemia or Cardiovascular Disease Risks

This trial evaluates the long-term safety and effectiveness of lerodalcibep (LIB003), an injectable solution given under the skin, for patients with homozygous or heterozygous familial hypercholesterolemia or those at high cardiovascular risk. The medication is administered once monthly for up to 72 weeks.

Inclusion criteria: Participants must have completed a previous Phase 3 study with LIB003 without serious adverse events, provide written consent, and be considered healthy based on medical assessments. Female participants of childbearing potential must use highly effective birth control and have negative pregnancy tests. Males must use contraception if their partners can become pregnant and must not donate sperm for 90 days after the last dose. Participants must maintain proper diet and continue current cholesterol-lowering therapies, with some possibly needing apheresis after 12 weeks.

Exclusion criteria: Those who haven’t completed previous LIB003 studies, not on stable cholesterol-lowering therapy, not at high cardiovascular risk, or without homozygous or heterozygous familial hypercholesterolemia cannot participate.

Study focus: The study monitors the efficacy and safety of the treatment through regular assessments measuring cholesterol changes at Weeks 48 and 72, checking for adverse effects, and monitoring for anti-LIB003 antibodies. The trial provides additional information on long-term cholesterol management.

Investigational drug: LIB003 inhibits a specific protein involved in cholesterol regulation to further reduce LDL-C levels in patients already on stable cholesterol-lowering therapy.

Study on Long-Term Safety of Inclisiran for Patients with Familial Hypercholesterolemia Who Completed Previous Adolescent Studies

This study focuses on the long-term safety and tolerability of inclisiran in individuals with familial hypercholesterolemia who have already completed previous studies named ORION-16 or ORION-13. The injectable medication is given under the skin while participants continue their current cholesterol-lowering treatments.

Inclusion criteria: Participants must be male or female with heterozygous or homozygous familial hypercholesterolemia who completed ORION-16 or ORION-13 studies. The study doctor must believe they benefited from inclisiran treatment in previous studies. Participants must continue current cholesterol-lowering treatments like statins or ezetimibe without planned changes in medication or dosage.

Exclusion criteria: Those without familial hypercholesterolemia, outside the specified age range, not part of specified trial groups, or in vulnerable populations cannot participate.

Study focus: The trial monitors any side effects or adverse events, changes in vital signs, growth, and laboratory results throughout the study period, which extends until February 2028. Regular assessments measure changes in LDL cholesterol levels from the beginning to the end of the study.

Investigational drug: Inclisiran helps the liver remove more LDL cholesterol from the blood by targeting and reducing production of a protein involved in cholesterol regulation. It is classified as a small interfering RNA therapy.

Summary

The ten clinical trials for Type IIa Hyperlipidaemia reveal a strong focus on developing new treatment options for both adults and children with familial hypercholesterolemia. Several patterns emerge from the available studies. Germany, the Netherlands, and Spain appear most frequently as trial locations, suggesting these countries have well-established research infrastructure for cholesterol-related conditions.

The trials test diverse approaches, including oral medications like laroprovstat (AZD0780), bempedoic acid, rosuvastatin, and MK-0616, as well as injectable treatments such as inclisiran, alirocumab, and lerodalcibep. Inclisiran appears in multiple studies across different age groups, indicating significant research interest in this medication’s potential. Several trials specifically address pediatric populations, with age ranges from 2 to 17 years, demonstrating increasing attention to early intervention for inherited high cholesterol.

Most studies require participants to continue their existing statin therapy, suggesting the new medications are intended to work alongside current treatments rather than replace them. The trial durations vary considerably, from short 8-week studies to long-term assessments extending up to 72 weeks or more, reflecting different research objectives from initial safety evaluation to long-term effectiveness monitoring. Some trials also include cardiovascular disease risk assessment beyond cholesterol management, particularly those studying MK-0616 and lerodalcibep.

Ongoing Clinical Trials on Type IIa hyperlipidaemia

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