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RecruitingPhase 3

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

Novartis Pharmaceuticals · NCT06597019

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 6–11 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 3
Target enrollment
60
US sites recruiting
9
Study start
December 9, 2024
Record last updated
May 15, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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).

Being studied: Inclisiran; Placebo

Conditions: Familial Hypercholesterolemia - Heterozygous

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

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

Recruiting US locations

  • UC San Francisco Medical Center

    San Francisco, CA 94143-0348

    Site contact: Luis Gay · +1 415 476 8338 · luis.gay@ucsf.edu

  • UC San Francisco Medical Center

    San Francisco, CA 94143

    Site contact: Luis Gay · +1 415 990 7296 · Luis.Gay@ucsf.edu

  • Childrens National Hospital

    Washington D.C., DC 20010

    Site contact: Carlos Jesus Carhuas · +1 202 476 5000 · ccarhuas@childrensnational.org

  • Excel Medical Clinical Trials LLC

    Boca Raton, FL 33434

    Site contact: Mariana Sanchez Villa · +1 561 756 8206 · msanchezvilla@flourishresearch.com

  • Icahn School of Med at Mt Sinai

    New York, NY 10029

    Site contact: Jay Krishna Katragadda · +1 212 659 9174 · jaykrishna.katragadda@mssm.edu

  • Primary Childrens Medical Center

    Salt Lake City, UT 84113

    Site contact: Linda Lambert · +1 801 587 9153 · Linda.lambert@hsc.utah.edu

  • Virginia Commonwealth University

    Richmond, VA 23298

    Site contact: Megan Scott · megan.scott@vcuhealth.org

  • West Virginia Childrens Hospital

    Morgantown, WV 26506

    Site contact: Robin Hoffer · +1 724-972-9170 · Robin.Hoffer1@hsc.wvu.edu

  • West Virginia Childrens Hospital

    Morgantown, WV 26506

    Site contact: Robin Hoffer · +1 878 379 4121 · robin.hoffer1@hsc.wvu.edu

Study contacts (from the official record)

  • Novartis Pharmaceuticals · 1-888-669-6682 · novartis.email@novartis.com
  • Novartis Pharmaceuticals · +41613241111
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Study to Evaluate Efficacy and Safety of Inclisiran in Children… (NCT06597019) | Bored Studies