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

RecruitingPhase 3

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

Who can join

Ages 6 to 11 · All sexes

Full eligibility 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

About the study

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).

What is being tested

Sponsor: Novartis Pharmaceuticals · Participants: 60 · Started: Dec 9, 2024

Contact the study team

Official record on ClinicalTrials.gov — NCT06597019

Locations in the U.S.

CaliforniaUC San Francisco Medical Center, San Francisco
UC San Francisco Medical Center, San Francisco
District of ColumbiaChildren's National Hospital, Washington D.C. (Not yet recruiting)
Childrens National Hospital, Washington D.C.
FloridaExcel Medical Clinical Trials LLC, Boca Raton
New YorkIcahn School of Med at Mt Sinai, New York
UtahPrimary Childrens Medical Center, Salt Lake City (Not yet recruiting)
Primary Childrens Medical Center, Salt Lake City
VirginiaVirginia Commonwealth University, Richmond
West VirginiaWest Virginia Childrens Hospital, Morgantown
West Virginia Childrens Hospital, Morgantown

Conditions

From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.