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

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What is this study about?

This clinical trial is focused on studying a condition called Heterozygous Familial Hypercholesterolemia (HeFH), which is a genetic disorder that causes high levels of cholesterol in the blood. The treatment being tested in this study is a medication called Bempedoic acid, also known by its code name ETC-1002. This medication is being tested in the form of a film-coated tablet and an oral suspension, which means it can be taken by mouth either as a pill or a liquid.

The purpose of the study is to understand how Bempedoic acid works in children aged 6 to 17 who have HeFH. The study will look at how the body processes the medication and how it affects cholesterol levels. Participants will take the medication once daily for a period of 8 weeks. During this time, researchers will monitor the levels of the medication in the blood and observe any changes in cholesterol levels. The study will also assess how well children tolerate the medication, including its taste and ease of swallowing.

This study is important because it aims to provide more information about the safety and effectiveness of Bempedoic acid in young patients with HeFH. By understanding how the medication works in children, researchers hope to find better ways to manage high cholesterol levels in this age group. The study is expected to continue until 2025, allowing enough time to gather comprehensive data on the medication’s effects.

1 joining the study

Upon joining the study, the patient will begin the trial process. This involves understanding the study’s purpose and procedures.

The patient must meet specific criteria, such as being between 6 and 17 years old, having a diagnosis of heterozygous familial hypercholesterolemia, and maintaining stable lipid-modifying therapy.

2 medication administration

The patient will receive bempedoic acid, which is administered orally. The medication is available in the form of a film-coated tablet or oral suspension.

The dosage is once daily, and the treatment duration is 8 weeks.

3 monitoring and assessments

Throughout the 8-week period, the patient’s response to the medication will be monitored. This includes measuring the levels of LDL-C (low-density lipoprotein cholesterol) and other cholesterol markers.

The study will also evaluate the pharmacokinetics, which is how the drug is absorbed, distributed, metabolized, and excreted in the body.

4 end of treatment evaluation

At the end of the 8-week treatment period, the patient will undergo a final assessment to evaluate the effects of the medication.

This includes measuring the trough plasma concentration of the drug and its active metabolite, as well as assessing the overall cholesterol-lowering response.

Who Can Join the Study?

  • The patient’s parent(s) or guardian(s) must agree to sign a form giving permission for the child to join the study, and the child must also agree to participate.
  • The patient must be between 6 and 17 years old.
  • The patient must weigh at least 16 kilograms (about 35 pounds).
  • The patient must have a condition called Heterozygous Familial Hypercholesterolemia (HeFH), which is a genetic disorder that affects cholesterol levels. This can be confirmed by a genetic test or by certain cholesterol levels in the blood, along with family history.
  • The patient must be currently taking approved medications to manage cholesterol levels, such as statins, and these medications must have been at a stable dose for at least 4 weeks before starting the study. Some medications, like fibrates, need to be stable for 6 weeks, but gemfibrozil is not allowed if the patient is taking a statin.
  • The patient must have a fasting LDL-C (a type of cholesterol) level of at least 130 mg/dL.
  • The patient can be male or female. If female, the patient must not be pregnant or planning to become pregnant soon, must not be breastfeeding, and must agree to use birth control if they are sexually active. This requirement starts when the consent form is signed and continues during the study and for at least 30 days after the last dose of the study medication.

Who Cannot Join the Study?

  • Patients who are not between 6 to 17 years old cannot participate.
  • Patients who do not have heterozygous familial hypercholesterolemia cannot participate. This is a condition where there is a high level of cholesterol in the blood due to a genetic issue.
  • Patients who are part of a vulnerable population, such as those who cannot give consent or are in a dependent situation, cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Hospital Jerez de la Frontera Jerez De La Frontera Spain

Other Sites

Site Name City Country Status
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Hospital Universitario 12 De Octubre Madrid Spain
Region Midtjylland Aarhus Denmark
Rigshospitalet Copenhagen Denmark
Agr dsp Bpuh Kubpptz uqr Jjxrzgtvqlwqixeoq Hanover Germany
Pjqj Taxyi Hqeyzgab Uxkulkqtrgrt Sabadell Spain
Awyxbpsbd Ukm Amsterdam The Netherlands
Ejzwcxu Uljlmtoqpeur Mxvjyxp Cyhstdn Rdsjwnivp (vnzpjjv Mil Rotterdam The Netherlands
Gxupab Ujdxolpdak Fvujbqgry Frankfurt Germany
Hkzyznzx Usncgkothowtf dg A Cvbmmg A Coruna Galicia Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Denmark Denmark
Not recruiting
01.08.2023
Germany Germany
Not recruiting
01.08.2023
Spain Spain
Not recruiting
01.08.2023
The Netherlands The Netherlands
Not recruiting
01.08.2023

Trial locations

Investigated drugs:

Bempedoic Acid is a medication being studied for its effects on cholesterol levels in children and teenagers with a condition called heterozygous familial hypercholesterolemia. This condition causes high cholesterol levels, which can lead to heart problems. The study aims to understand how the body processes and responds to this medication, as well as to evaluate its safety in young patients.

Heterozygous Familial Hypercholesterolemia – This is a genetic disorder characterized by high levels of low-density lipoprotein (LDL) cholesterol in the blood. It is caused by a mutation in one of the genes responsible for removing LDL cholesterol from the bloodstream. Individuals with this condition often have cholesterol deposits in the skin or tendons and may develop cardiovascular issues at an earlier age. The condition is inherited in an autosomal dominant pattern, meaning one copy of the altered gene in each cell is sufficient to cause the disorder. Over time, the high cholesterol levels can lead to the buildup of plaques in the arteries, a process known as atherosclerosis. This can result in reduced blood flow and potential complications related to heart health.

Trial ID:
2024-515864-30-00
Protocol code:
1002-041
NCT ID:
NCT05694260
Trial Phase:
Therapeutic exploratory (Phase II)

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