Last Update: Jul 24, 2026 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 (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol ClinicalTrials.gov Identifier: [NCT06597006](https://clinicaltrials.gov/ct2/show/NCT06597006) Novartis Reference Number:CKJX839C12304 [See if you Pre-qualify](#trial-eligibility "See if you Pre-qualify") All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. ## Study Description 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). This is a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter 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 (LDL-C) on stable standard of care background lipid-lowering therapy. Condition Familial Hypercholesterolemia - Homozygous Phase Phase3 Overall Status Recruiting Number of Participants9 Start Date Feb 28, 2025 Completion Date Apr 15, 2029 Gender All Age(s) 2 Years - 11 Years (Child) ## Interventions Drug ### Inclisiran Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg, inclisiran sodium 180 mg s.c. for participants with body weight \ Drug ### Placebo Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection) ## Eligibility Criteria 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 ≥30 days before screening with no planned medication or dose changes during study participation \* Participants on a documented regimen of LDL-apheresis for ≥ 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 Austria #### Novartis Investigative Site Recruiting Vienna,1090,Austria China #### Novartis Investigative Site Recruiting Beijing,Beijing Municipality,100013,China Germany #### Novartis Investigative Site Recruiting Frankfurt am Main,Hesse,60590,Germany Greece #### Novartis Investigative Site Recruiting Thessaloniki,546 42,Greece #### Novartis Investigative Site Recruiting Ioannina,455 00,Greece Malaysia #### Novartis Investigative Site Recruiting Kota Bharu,Kelantan,16150,Malaysia Netherlands #### Novartis Investigative Site Recruiting Amsterdam,North Holland,1105 az,Netherlands South Africa #### Novartis Investigative Site Recruiting Bloemfontein,Free State,9301,South Africa #### Novartis Investigative Site Recruiting Johannesburg,Gauteng,2193,South Africa Taiwan #### Novartis Investigative Site Recruiting Taichung,407219,Taiwan #### Novartis Investigative Site Recruiting Taipei,111045,Taiwan Turkey (Türkiye) #### Novartis Investigative Site Recruiting Izmir,35100,Turkey (Türkiye) #### Novartis Investigative Site Recruiting Adana,Saricam,01330,Turkey (Türkiye) #### Novartis Investigative Site Recruiting Ankara,Yenimahalle,06500,Turkey (Türkiye) United Kingdom #### Novartis Investigative Site Recruiting Southampton,So16 6yd,United Kingdom United States #### Childrens National Hospital Recruiting Washington D.C.,District of Columbia,20010,United States ###### Sarah Clauss ###### Desiree Tobechukwu Nwanze Phone: [+1 202 476 5000]() Email: #### Washington Univ School Of Medicine Recruiting St Louis,Missouri,63110,United States ###### Jodi Pagano Email: ###### Anne Goldberg #### UC San Francisco Medical Center Recruiting San Francisco,California,94143-0348,United States ###### Luis Gay Phone: [+1 415 476 8338]() Email: ###### Martin Thelin #### Primary Childrens Medical Center Recruiting Salt Lake City,Utah,84113,United States ###### Adam Ware ###### Linda Lambert Email: ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: [](mailto:) #### Novartis Pharmaceuticals Phone: [ 1-888-669-6682](tel:1-888-669-6682) Email: