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

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the long-term safety and tolerability of a medication called inclisiran in individuals with a condition known as familial hypercholesterolemia. Familial hypercholesterolemia is a genetic disorder that causes high levels of cholesterol in the blood, which can increase the risk of heart disease. The study involves participants who have already completed previous studies named ORION-16 or ORION-13. Inclisiran, also known by its code name KJX839, is administered as a solution for injection under the skin.

The purpose of this study is to evaluate how well participants tolerate inclisiran over a long period. Participants will continue their current cholesterol-lowering treatments, such as statins or ezetimibe, without any planned changes in medication or dosage during the study. The study will monitor any side effects or adverse events that occur, as well as changes in vital signs, growth, and laboratory test results.

Throughout the study, researchers will also measure changes in LDL cholesterol levels, which is often referred to as "bad" cholesterol, from the beginning of the study to the end. This will help determine the effectiveness of inclisiran in managing cholesterol levels in participants with familial hypercholesterolemia. The study is designed to provide valuable information on the long-term use of inclisiran for individuals with this genetic condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after completing the ORION-16 or ORION-13 studies. Eligibility requires a diagnosis of heterozygous or homozygous familial hypercholesterolemia and previous benefit from inclisiran treatment.

    Current lipid-lowering therapies, such as statins or ezetimibe, should be continued without changes in medication or dosage.

  2. Step 2

    Medication administration

    The medication used in this study is inclisiran, administered through subcutaneous use. This means the medication is injected under the skin.

    The frequency and dosage of inclisiran will be determined by the study protocol and communicated during the study visits.

  3. Step 3

    Monitoring and assessments

    Regular monitoring will occur to evaluate the long-term safety and tolerability of inclisiran. This includes checking for any treatment-emergent adverse events, which are side effects that may occur during the study.

    Assessments will also include vital signs, growth measurements, and laboratory tests to ensure overall health and safety.

  4. Step 4

    Study duration

    The study is expected to continue until February 7, 2028. Participation involves regular visits and assessments throughout this period.

    The study aims to observe changes in LDL-C levels, which is a type of cholesterol, from the beginning of the study to its end.

Who can join the trial?

4 criteria

  • Participants must be male or female with a diagnosis of HeFH or HoFH. These are types of familial hypercholesterolemia, a condition where high cholesterol runs in families.
  • Participants should have completed the ORION-16 or ORION-13 studies. This means they received the last dose of the study drug and attended the final study visit as required.
  • The study doctor must believe that the participant benefited from treatment with inclisiran in the ORION-16 or ORION-13 studies. Inclisiran is a medication used to lower cholesterol levels.
  • Participants must continue their current cholesterol-lowering treatments, like statins or ezetimibe, from the previous study. There should be no plans to change these medications or their doses during the study.

Who cannot join the trial?

4 criteria

  • Patients who do not have **familial hypercholesterolemia** cannot participate. This is a genetic condition that causes high cholesterol levels.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are not part of the specified clinical trial groups cannot participate.
  • Patients who are part of a vulnerable population, which may include groups like children or pregnant women, cannot participate.
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Investigated drugs

Inclisiran is a medication being studied for its long-term safety and tolerability in people with familial hypercholesterolemia, a condition characterized by high cholesterol levels. It works by helping to lower cholesterol levels in the blood, which can reduce the risk of heart disease. This study is focused on understanding how safe and well-tolerated inclisiran is when used over a long period.

What is already known about the treatment

Inclisiran – Inclisiran is administered as an injection under the skin. It is currently being studied in clinical trials to assess its long-term safety and tolerability for treating familial hypercholesterolemia, a genetic condition that causes high cholesterol levels. The medication works by targeting a specific molecule in the liver to reduce the production of LDL cholesterol, often referred to as "bad" cholesterol. Inclisiran is classified as a small interfering RNA (siRNA) therapy, which helps lower cholesterol levels by interfering with the genetic instructions that produce a protein involved in cholesterol regulation.

Investigated diseases

Familial Hypercholesterolemia – This is a genetic disorder characterized by high cholesterol levels, specifically low-density lipoprotein cholesterol (LDL-C), in the blood. It is caused by mutations in genes responsible for removing LDL-C from the bloodstream, leading to its accumulation. Over time, this can result in the buildup of cholesterol in the walls of arteries, known as atherosclerosis. Individuals with this condition may develop cholesterol deposits in the skin or tendons and are at risk for cardiovascular issues. 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. Symptoms can vary, but the condition is often present from birth and progresses with age.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-507278-41-00Protocol codeCKJX839C12001BEstimated enrolment196 patientsSponsorNovartis Pharma AG

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