RecruitingPhase 3NCT06597019

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol


Sponsor

Novartis Pharmaceuticals

Enrollment

51 participants

Start Date

Dec 9, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).


Eligibility

Min Age: 6 YearsMax Age: 11 Years

Plain Language Summary

Simplified for easier understanding

This study is testing inclisiran, an injectable cholesterol-lowering medicine, in children aged 6 to under 12 who have heterozygous familial hypercholesterolemia (HeFH) — an inherited condition causing high LDL cholesterol. **You may be eligible if...** - Your child is between 6 and under 12 years old - Your child has HeFH confirmed by genetic testing or clinical criteria - Your child's LDL cholesterol is above 130 mg/dL - If your child is 8 or older, they are on the best tolerated statin dose **You may NOT be eligible if...** - Your child has used PCSK9 antibody medications in the past 90 days - Your child has high cholesterol from another cause (like thyroid or kidney disease) - Your child has the more severe form (homozygous FH, HoFH) - Your child weighs less than 16 kg - Your child has active liver disease - Your child is pregnant or nursing Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGInclisiran

Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg and inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.

DRUGPlacebo

Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)


Locations(66)

UC San Francisco Medical Center

San Francisco, California, United States

UC San Francisco Medical Center

San Francisco, California, United States

Children's National Hospital

Washington D.C., District of Columbia, United States

Childrens National Hospital

Washington D.C., District of Columbia, United States

Excel Medical Clinical Trials LLC

Boca Raton, Florida, United States

Icahn School of Med at Mt Sinai

New York, New York, United States

Primary Childrens Medical Center

Salt Lake City, Utah, United States

Primary Childrens Medical Center

Salt Lake City, Utah, United States

Virginia Commonwealth University

Richmond, Virginia, United States

West Virginia Childrens Hospital

Morgantown, West Virginia, United States

West Virginia Childrens Hospital

Morgantown, West Virginia, United States

Novartis Investigative Site

Buenos Aires, Argentina

Novartis Investigative Site

CABA, Argentina

Novartis Investigative Site

Salzburg, Austria

Novartis Investigative Site

Vienna, Austria

Novartis Investigative Site

Brussels, Belgium

Novartis Investigative Site

Leuven, Belgium

Novartis Investigative Site

Fortaleza, Ceará, Brazil

Novartis Investigative Site

Rio de Janeiro, Rio de Janeiro, Brazil

Novartis Investigative Site

Porto Alegre, Rio Grande do Sul, Brazil

Novartis Investigative Site

São Paulo, São Paulo, Brazil

Novartis Investigative Site

Beijing, Beijing Municipality, China

Novartis Investigative Site

Shanghai, China

Novartis Investigative Site

Prague, Czechia

Novartis Investigative Site

Prague, Czechia

Novartis Investigative Site

Marseille, France

Novartis Investigative Site

Nantes, France

Novartis Investigative Site

Paris, France

Novartis Investigative Site

Freiburg im Breisgau, Baden-Wurttemberg, Germany

Novartis Investigative Site

Frankfurt am Main, Hesse, Germany

Novartis Investigative Site

Hanover, Germany

Novartis Investigative Site

Athens, Greece

Novartis Investigative Site

Ioannina, Greece

Novartis Investigative Site

Hong Kong, Hong Kong

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Budapest, Hungary

Novartis Investigative Site

Jerusalem, Israel

Novartis Investigative Site

Ramat Gan, Israel

Novartis Investigative Site

Milan, MI, Italy

Novartis Investigative Site

Roma, RM, Italy

Novartis Investigative Site

Torino, TO, Italy

Novartis Investigative Site

Verona, VR, Italy

Novartis Investigative Site

Kuala Lumpur, Kuala Lumpur, Malaysia

Novartis Investigative Site

Amsterdam, North Holland, Netherlands

Novartis Investigative Site

Bialystok, Poland

Novartis Investigative Site

Gdansk, Poland

Novartis Investigative Site

Lodz, Łódź Voivodeship, Poland

Novartis Investigative Site

Coimbra, Portugal

Novartis Investigative Site

Lisbon, Portugal

Novartis Investigative Site

Porto, Portugal

Novartis Investigative Site

Porto, Portugal

Novartis Investigative Site

Bloemfontein, Free State, South Africa

Novartis Investigative Site

Elche, Alicante, Spain

Novartis Investigative Site

Cadiz, Andalusia, Spain

Novartis Investigative Site

Esplugues, Barcelona, Spain

Novartis Investigative Site

Badalona, Catalonia, Spain

Novartis Investigative Site

Pamplona, Navarre, Spain

Novartis Investigative Site

Barcelona, Spain

Novartis Investigative Site

Málaga, Spain

Novartis Investigative Site

Seville, Spain

Novartis Investigative Site

Taipei, Taiwan

Novartis Investigative Site

Taipei, Taiwan

Novartis Investigative Site

Adana, Saricam, Turkey (Türkiye)

Novartis Investigative Site

Ankara, Yenimahalle, Turkey (Türkiye)

Novartis Investigative Site

Izmir, Turkey (Türkiye)

Novartis Investigative Site

West Midlands, Birmingham, United Kingdom

Novartis Investigative Site

London, United Kingdom

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NCT06597019


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