Study on the Safety and Effectiveness of Inclisiran for Children Aged 6 to Under 12 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, which is a genetic disorder that causes high levels of low-density lipoprotein cholesterol (LDL-C), often referred to as “bad cholesterol.” The study is specifically for children aged 6 to under 12 years who have this condition. The treatment being tested is a medication called Inclisiran, which is also known by its code name KJX839. Inclisiran is given as an injection and is designed to help lower LDL-C levels in the blood.

The purpose of the study is to evaluate the safety and effectiveness of Inclisiran in reducing LDL-C levels in children with this condition. The study is divided into two parts. In the first year, participants will receive either Inclisiran or a placebo, which is a substance with no active medication. In the second year, all participants will receive Inclisiran. The study will monitor changes in LDL-C levels and other health markers over time to assess the impact of the treatment.

Throughout the study, participants will have regular check-ups to track their health and any changes in their cholesterol levels. The study aims to provide valuable information on how well Inclisiran works in children with Heterozygous Familial Hypercholesterolemia and to ensure that it is safe for use in this age group. This research could lead to better treatment options for managing high cholesterol in children with this genetic condition.

1 initial assessment

The study begins with an initial assessment to confirm eligibility. Participants must be between 6 and less than 12 years old, diagnosed with heterozygous familial hypercholesterolemia, and have a fasting LDL cholesterol level greater than 130 mg/dL.

Participants aged 8 to less than 12 years should be on a stable dose of statin unless intolerant, with or without other lipid-lowering therapy. Those under 8 years may have different treatment based on the investigator’s discretion.

2 year 1: double-blind phase

Participants receive either inclisiran or a placebo through subcutaneous injection. The goal is to evaluate the reduction in LDL cholesterol by Day 330.

The primary objective is to demonstrate the superiority of inclisiran compared to placebo in reducing LDL cholesterol levels.

3 year 2: open-label phase

All participants receive inclisiran during this phase. The focus is on long-term safety, tolerability, and efficacy.

The study continues to monitor changes in LDL cholesterol and other lipid parameters up to Day 720.

4 monitoring and assessments

Throughout the study, regular assessments are conducted to monitor LDL cholesterol levels, other lipid parameters, and any adverse events.

Participants’ growth, vital signs, and development are also tracked to ensure safety and well-being.

Who Can Join the Study?

  • Participants must be male or female and between 6 to less than 12 years old at the time of screening.
  • Participants must have a condition called Heterozygous Familial Hypercholesterolemia (HeFH), which can be diagnosed through genetic testing or based on certain physical characteristics.
  • Participants must have a fasting LDL-C level greater than 130 mg/dL (3.4 mmol/L) at screening. LDL-C stands for low-density lipoprotein cholesterol, often referred to as “bad” cholesterol.
  • For participants aged 8 to less than 12 years, they should be on an optimal dose of statin medication, unless they cannot tolerate statins. They may also be on other cholesterol-lowering treatments like ezetimibe. For participants younger than 8 years, the use of cholesterol-lowering treatments is decided by the study doctor.
  • Participants who are already taking cholesterol-lowering medications, such as statins or ezetimibe, must have been on a stable dose for at least 30 days before screening and should not have any planned changes to their medication or dosage during the study.

Who Cannot Join the Study?

  • Children who are not between the ages of 6 and 12 years old.
  • Children who do not have a condition called Heterozygous Familial Hypercholesterolemia, which is a genetic disorder that causes high levels of cholesterol in the blood.
  • Children who do not have elevated levels of LDL-C, which stands for low-density lipoprotein cholesterol, often referred to as “bad” cholesterol.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Hospital Universitario De Navarra Pamplona Spain
Medical University Of Vienna Vienna Austria
Centro Hospitalar Universitario Sao Joao E.P.E. Porto Portugal
Katholieke Universiteit te Leuven Leuven Belgium
Unidade Local De Saúde De Santa Maria, E.P.E. Lisbon Portugal

Other Sites

Site Name City Country Status
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Hospital Universitario Puerta Del Mar Cadiz Spain
Hannoversche Kinderheilanstalt Hanover Germany
Instytut Centrum Zdrowia Matki Polki Lodz Poland
Nosokomeio Paidon I Agia Sofia Athens Greece
University General Hospital Of Ioannina Ioannina Greece
ASST Grande Ospedale Metropolitano Niguarda Milan Italy
Universita’ Degli Studi Di Verona Verona Italy
Medical Center – University Of Freiburg Freiburg Im Breisgau Germany
Centre Hospitalier Universitaire De Nantes Nantes France
Uniwersytecki Dzieciecy Szpital Kliniczny Im. L. Zamenhofa W Bialymstoku samodzielny publiczny zakład opieki zdrowotnej Bialystok Poland
Cortex Study Center Budapest Hungary
Virgen del Rocío University Hospital Sevilla Spain
Assistance Publique Hopitaux De Paris Paris France
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
Centro Hospitalar Universitario De Santo Antonio E.P.E. Porto Portugal
University Of Pecs Pecs Hungary
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Uniklinikum Salzburg Salzburg Austria
Culpgqgot Uxyafzqvzhtmgo Sbsphiitc Woluwe-Saint-Lambert Belgium
Abjknaneo Uiy Amsterdam The Netherlands
Akitnwvxdg Pyzawpvv Hhajzqtx Dx Mcqwmmusy Marseille France
Achtewy Olkndauvekq Ukwokkmpnyiif Clqlrzcjreem Djtmu Slaqyq E Dytek Szhrgpy Dg Tnffik Turin Italy
Gnykdg Uobluytoyn Frbxxhmdn Frankfurt Germany
Uerqzwxzzwsnhw Cfthezv Keukntgov Gdansk Poland
Hupnotzi Dg Lj Soecp Cemq I Sesx Pwv Barcelona Spain
Fudgvgbf npqzkghvt Mlafi a Hqadtdq Prague Czechia
Hamwoehr Ukdgpzoubfcll Heiburaz Tvrqy y Pfrgqj Ixefdukx Cpfsms djixqozcuxnepflry (ftzn Badalona Spain
Cvredp Hlzigdosvd E Uglbrkzluazxu Dq Ccolkwk Evzskn Coimbra Portugal

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

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
26.02.2025
Belgium Belgium
Not recruiting
26.02.2025
Czechia Czechia
Not recruiting
26.02.2025
France France
Not recruiting
26.02.2025
Germany Germany
Not recruiting
26.02.2025
Greece Greece
Not recruiting
26.02.2025
Hungary Hungary
Not recruiting
26.02.2025
Italy Italy
Not recruiting
26.02.2025
Poland Poland
Not recruiting
26.02.2025
Portugal Portugal
Not recruiting
26.02.2025
Spain Spain
Not recruiting
26.02.2025
The Netherlands The Netherlands
Not recruiting
26.02.2025

Trial locations

Investigated drugs:

Inclisiran: Inclisiran is a medication used to lower levels of LDL cholesterol, often referred to as “bad” cholesterol, in the blood. It works by helping the liver remove more LDL cholesterol from the bloodstream. This medication is being studied for its effectiveness and safety in children with a condition called heterozygous familial hypercholesterolemia, which causes high levels of LDL cholesterol. The trial aims to see if inclisiran can significantly reduce LDL cholesterol levels in these children.

Heterozygous Familial Hypercholesterolemia – This is a genetic disorder characterized by high levels of low-density lipoprotein cholesterol (LDL-C) in the blood. It is caused by a mutation in one of the genes responsible for removing LDL-C from the bloodstream. Individuals with this condition inherit one copy of the mutated gene from one parent. As a result, they have elevated cholesterol levels from a young age, which can lead to cholesterol deposits in various parts of the body. Over time, these deposits can cause narrowing of the arteries. This condition is often identified in childhood or early adulthood due to its genetic nature.

Trial ID:
2024-514594-21-00
Protocol code:
CKJX839C12303
Trial Phase:
Therapeutic confirmatory (Phase III)

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