Recruiting
Phase 3

Inclisiran

Sponsor:

Novartis Pharmaceuticals

Code:

NCT06597019

Conditions

Familial Hypercholesterolemia - Heterozygous

Eligibility Criteria

Sex: All

Age: 6 - 11

Healthy Volunteers: Not accepted

Interventions

Inclisiran

Placebo

Study Details

Brief 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).

Conditions

Familial Hypercholesterolemia - Heterozygous

Study ID

NCT06597019

Start date

Dec 9, 2024

Status verified date

May, 2026

Completion date

Apr 15, 2029

Anticipated

Primary completion date

Mar 21, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6 - 11

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

60 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Inclisiran

Year 1 - inclisiran sodium subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium subcutaneous injection (given at Days 450 and 630)

placebo comparator: Placebo

Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium subcutaneous injection (given at Days 360, 450, and 630)

Interventions

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.

Placebo

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

Primary outcome measure

  • Percentage change in LDL-C from baseline to Day 330 (Year 1) [ Time Frame: Baseline and Day 330 ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682novartis.email@novartis.com

Novartis Pharmaceuticals

+41613241111

Locations

UC San Francisco Medical Center

Recruiting

San Francisco, California, United States, 94143-0348

Contacts

Luis Gay

+1 415 476 8338luis.gay@ucsf.edu

Principal Investigator:

Martin Thelin

UC San Francisco Medical Center

Recruiting

San Francisco, California, United States, 94143

Contacts

Luis Gay

+1 415 990 7296Luis.Gay@ucsf.edu

Principal Investigator:

Martin Thelin

Childrens National Hospital

Recruiting

Washington D.C., District of Columbia, United States, 20010

Contacts

Carlos Jesus Carhuas

+1 202 476 5000ccarhuas@childrensnational.org

Principal Investigator:

Sarah Clauss

Excel Medical Clinical Trials LLC

Recruiting

Boca Raton, Florida, United States, 33434

Contacts

Mariana Sanchez Villa

+1 561 756 8206msanchezvilla@flourishresearch.com

Principal Investigator:

Rasha Youssef

Icahn School of Med at Mt Sinai

Recruiting

New York, New York, United States, 10029

Contacts

Jay Krishna Katragadda

+1 212 659 9174jaykrishna.katragadda@mssm.edu

Principal Investigator:

Joseph Mahgerefteh

Primary Childrens Medical Center

Recruiting

Salt Lake City, Utah, United States, 84113

Contacts

Linda Lambert

+1 801 587 9153Linda.lambert@hsc.utah.edu

Principal Investigator:

Adam Ware

Virginia Commonwealth University

Recruiting

Richmond, Virginia, United States, 23298

Contacts

Megan Scott

megan.scott@vcuhealth.org

Principal Investigator:

Bradford McQuilkin

West Virginia Childrens Hospital

Recruiting

Morgantown, West Virginia, United States, 26506

Contacts

Robin Hoffer

+1 724-972-9170Robin.Hoffer1@hsc.wvu.edu

Principal Investigator:

Lee Pyles

West Virginia Childrens Hospital

Recruiting

Morgantown, West Virginia, United States, 26506

Contacts

Robin Hoffer

+1 878 379 4121robin.hoffer1@hsc.wvu.edu

Principal Investigator:

Lee Pyles

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

May 15, 2026

Last verified

May, 2026

Keywords

  • Heterozygous familial hypercholesterolemia (HeFH),
  • LDL-cholesterol (LDL-C),
  • Children,
  • pediatric,
  • small interfering ribonucleic acid (siRNA),
  • inclisiran,
  • Familial Hypercholesterolemia,
  • Heterozygous FH,
  • Hypercholesterolemia,
  • Lipoprotein(a),
  • Hyperlipidemia,
  • Dyslipidemia,
  • Heart Failure,
  • Cardiovascular Diseases,
  • Cholesterol,
  • Aortic Stenosis

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-05-15.