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NCT06597019PHASE3Recruiting

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

Novartis Pharmaceuticals

Start Date

12/9/2024

Completion Date

4/15/2029

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).

Detailed Description

This is a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter 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 (LDL-C) on stable standard of care background lipid-lowering therapy.

Eligibility Criteria

Age Range: 6 years to 11 years

Inclusion Criteria: * Male or female participants, 6 to \<12 years of age at screening * HeFH diagnosed either by genetic testing or on phenotypic criteria * Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening * For participants 8 to \<12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants \<8 years, the use of background lipid-lowering treatment is based on investigator's discretion. * Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation. Exclusion Criteria: * Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9 * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome * Homozygous familial hypercholesterolemia (HoFH) * Body weight \<16 kg at the screening and/or randomization (Day 1) visit * Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation \>3x ULN, or total bilirubin elevation \>2x ULN (except patients with Gilbert's syndrome) * Pregnant or nursing females * Recent and/or planned use of other investigational medicinal products or devices

Interventions

DRUG

Inclisiran

DRUG

Placebo

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Conditions

Familial Hypercholesterolemia - Heterozygous

Locations

UC San Francisco Medical Center

San Francisco, California 94143-0348

United States

UC San Francisco Medical Center

San Francisco, California 94143

United States

Children's National Hospital

Washington D.C., District of Columbia 20010

United States

Childrens National Hospital

Washington D.C., District of Columbia 20010

United States

Excel Medical Clinical Trials LLC

Boca Raton, Florida 33434

United States

Icahn School of Med at Mt Sinai

New York, New York 10029

United States

Primary Childrens Medical Center

Salt Lake City, Utah 84113

United States

Primary Childrens Medical Center

Salt Lake City, Utah 84113

United States

Virginia Commonwealth University

Richmond, Virginia 23298

United States

West Virginia Childrens Hospital

Morgantown, West Virginia 26506

United States

West Virginia Childrens Hospital

Morgantown, West Virginia 26506

United States

Novartis Investigative Site

Buenos Aires, C1245AAM

Argentina

Novartis Investigative Site

CABA, C1181ACH

Argentina

Novartis Investigative Site

Salzburg, 5020

Austria

Novartis Investigative Site

Vienna, 1090

Austria

Novartis Investigative Site

Brussels, 1200

Belgium

Novartis Investigative Site

Leuven, 3000

Belgium

Novartis Investigative Site

Fortaleza, Ceará 60430-275

Brazil

Novartis Investigative Site

Rio de Janeiro, Rio de Janeiro 20211-340

Brazil

Novartis Investigative Site

Porto Alegre, Rio Grande do Sul 90020-020

Brazil

Novartis Investigative Site

São Paulo, São Paulo 05403 000

Brazil

Novartis Investigative Site

Beijing, Beijing Municipality 100013

China

Novartis Investigative Site

Shanghai, 200127

China

Novartis Investigative Site

Prague, 128 08

Czechia

Novartis Investigative Site

Prague, 150 06

Czechia

Novartis Investigative Site

Marseille, 13885

France

Novartis Investigative Site

Nantes, 44093

France

Novartis Investigative Site

Paris, 75012

France

Novartis Investigative Site

Freiburg im Breisgau, Baden-Wurttemberg 79106

Germany

Novartis Investigative Site

Frankfurt am Main, Hesse 60590

Germany

Novartis Investigative Site

Hanover, 30173

Germany

Novartis Investigative Site

Athens, 115 27

Greece

Novartis Investigative Site

Ioannina, 455 00

Greece

Novartis Investigative Site

Hong Kong, 999077

Hong Kong

Novartis Investigative Site

Budapest, 1026

Hungary

Novartis Investigative Site

Jerusalem, 9112001

Israel

Novartis Investigative Site

Ramat Gan, 5265601

Israel

Novartis Investigative Site

Milan, MI 20162

Italy

Novartis Investigative Site

Roma, RM 00165

Italy

Novartis Investigative Site

Torino, TO 10126

Italy

Novartis Investigative Site

Verona, VR 37126

Italy

Novartis Investigative Site

Kuala Lumpur, Kuala Lumpur 50586

Malaysia

Novartis Investigative Site

Amsterdam, North Holland 1105 AZ

Netherlands

Novartis Investigative Site

Bialystok, 15-274

Poland

Novartis Investigative Site

Gdansk, 80 952

Poland

Novartis Investigative Site

Lodz, Łódź Voivodeship 93-338

Poland

Novartis Investigative Site

Coimbra, 3000-602

Portugal

Novartis Investigative Site

Lisbon, 1649-035

Portugal

Novartis Investigative Site

Porto, 4050-651

Portugal

Novartis Investigative Site

Porto, 4200 319

Portugal

Novartis Investigative Site

Bloemfontein, Free State 9301

South Africa

Novartis Investigative Site

Elche, Alicante 03203

Spain

Novartis Investigative Site

Cadiz, Andalusia 11009

Spain

Novartis Investigative Site

Badalona, Barcelona 08916

Spain

Novartis Investigative Site

Esplugues, Barcelona 08950

Spain

Novartis Investigative Site

Pamplona, Navarre 31008

Spain

Novartis Investigative Site

Barcelona, 08041

Spain

Novartis Investigative Site

Málaga, 29011

Spain

Novartis Investigative Site

Seville, 41013

Spain

Novartis Investigative Site

Taipei, 111045

Taiwan

Novartis Investigative Site

Taipei, 11217

Taiwan

Novartis Investigative Site

Adana, Saricam 01330

Turkey (Türkiye)

Novartis Investigative Site

Ankara, Yenimahalle 06500

Turkey (Türkiye)

Novartis Investigative Site

Izmir, 35100

Turkey (Türkiye)

Novartis Investigative Site

West Midlands, Birmingham B4 6NH

United Kingdom

Novartis Investigative Site

London, NW3 2QG

United Kingdom