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RECRUITINGPhase 3INTERVENTIONAL

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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).

Who May Be Eligible (Plain English)

Who May Qualify: - 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. Who Should NOT Join This Trial: - 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 Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

DRUG

Inclisiran

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.

DRUG

Placebo

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

Locations (20)

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