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Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

RecruitingPhase 3Ages 2 Years - 11 YearsAllNovartis Pharmaceuticals
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In plain words

What the trial is, and whether it could suit you

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * Male or female participants, 2 to \<12 years of age at screening * HoFH diagnosed by genetic confirmation \- Note: Participants with known null (negative) mutations in both LDLR alleles are not eligible (see also exclusion criteria) * Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening * On an optimal dose of statin (investigator's discretion), unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe) * Participants on lipid-lowering therapies (such as e.g. statins, ezetimibe) must be on a stable dose for โ‰ฅ30 days before screening with no planned medication or dose changes during study participation * Participants on a documented regimen of LDL-apheresis for โ‰ฅ 3 months before screening will be allowed to continue the apheresis during the study, if needed. The apheresis schedule/settings/duration must be stable prior to screening, are not allowed to change during the double-blind period of the trial and must permit that an apheresis coincides with each study visit. Exclusion Criteria: * Documented evidence of a null (negative) mutation in both LDLR alleles * Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9 * History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. \<15% reduction in LDL-C) * Treatment with mipomersen or lomitapide (within 5 months of screening) * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome * Heterozygous familial hypercholesterolemia (HeFH) * Body weight (at the screening and/or randomization (Day 1) visit) \<16 kg for participants 6 to \<12 years (at screening) or \<11 kg for participants 2 to \<6 years (at screening) * 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
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What it tests

  • Inclisiran
  • Placebo
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Where

19 sites
UC San Francisco Medical Center
San Francisco, United States
Childrens National Hospital
Washington D.C., United States
Washington Univ School Of Medicine
St Louis, United States
Primary Childrens Medical Center
Salt Lake City, United States
Novartis Investigative Site
Vienna, Austria
Novartis Investigative Site
Beijing, China
Novartis Investigative Site
Frankfurt am Main, Germany
Novartis Investigative Site
Ioannina, Greece

โ€ฆand 11 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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