Last Update: May 15, 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 (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol ClinicalTrials.gov Identifier: [NCT06597019](https://clinicaltrials.gov/ct2/show/NCT06597019) Novartis Reference Number:CKJX839C12303 [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 6 to \\<12 years) with heterozygous familial hypercholesterolemia (HeFH) 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 6 to \\<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy. Condition Familial Hypercholesterolemia - Heterozygous Phase Phase3 Overall Status Recruiting Number of Participants60 Start Date Dec 09, 2024 Completion Date Apr 15, 2029 Gender All Age(s) 6 Years - 11 Years (Child) ## Interventions Drug ### 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 \ Drug ### Placebo Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection) ## 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 Argentina #### Novartis Investigative Site Recruiting Buenos Aires,C1245aam,Argentina #### Novartis Investigative Site Recruiting CABA,C1181ach,Argentina Austria #### Novartis Investigative Site Recruiting Salzburg,5020,Austria #### Novartis Investigative Site Recruiting Vienna,1090,Austria Belgium #### Novartis Investigative Site Recruiting Leuven,3000,Belgium #### Novartis Investigative Site Recruiting Brussels,1200,Belgium Brazil #### Novartis Investigative Site Recruiting São Paulo,São Paulo,05403 000,Brazil #### Novartis Investigative Site Recruiting Porto Alegre,Rio Grande do Sul,90020-020,Brazil #### Novartis Investigative Site Recruiting Rio de Janeiro,Rio de Janeiro,20211-340,Brazil #### Novartis Investigative Site Recruiting Fortaleza,Ceará,60430-275,Brazil China #### Novartis Investigative Site Recruiting Shanghai,200127,China #### Novartis Investigative Site Recruiting Beijing,Beijing Municipality,100013,China Czechia #### Novartis Investigative Site Recruiting Prague,128 08,Czechia #### Novartis Investigative Site Recruiting Prague,150 06,Czechia France #### Novartis Investigative Site Recruiting Nantes,44093,France #### Novartis Investigative Site Recruiting Marseille,13885,France #### Novartis Investigative Site Recruiting Paris,75012,France Germany #### Novartis Investigative Site Recruiting Hanover,30173,Germany #### Novartis Investigative Site Recruiting Freiburg im Breisgau,Baden-Wurttemberg,79106,Germany #### Novartis Investigative Site Recruiting Frankfurt am Main,Hesse,60590,Germany Greece #### Novartis Investigative Site Recruiting Ioannina,455 00,Greece #### Novartis Investigative Site Recruiting Athens,115 27,Greece Hong Kong #### Novartis Investigative Site Recruiting Hong Kong,999077,Hong Kong Hungary #### Novartis Investigative Site Recruiting Budapest,1026,Hungary Israel #### Novartis Investigative Site Recruiting Jerusalem,9112001,Israel #### Novartis Investigative Site Recruiting Ramat Gan,5265601,Israel Italy #### Novartis Investigative Site Recruiting Torino,TO,10126,Italy #### Novartis Investigative Site Recruiting Verona,VR,37126,Italy #### Novartis Investigative Site Recruiting Milan,MI,20162,Italy #### Novartis Investigative Site Recruiting Roma,RM,00165,Italy Malaysia #### Novartis Investigative Site Recruiting Kuala Lumpur,Kuala Lumpur,50586,Malaysia Netherlands #### Novartis Investigative Site Recruiting Amsterdam,North Holland,1105 az,Netherlands Poland #### Novartis Investigative Site Recruiting Gdansk,80 952,Poland #### Novartis Investigative Site Recruiting Bialystok,15-274,Poland #### Novartis Investigative Site Recruiting Lodz,Łódź Voivodeship,93-338,Poland Portugal #### Novartis Investigative Site Recruiting Coimbra,3000-602,Portugal #### Novartis Investigative Site Recruiting Lisbon,1649-035,Portugal #### Novartis Investigative Site Recruiting Porto,4050-651,Portugal #### Novartis Investigative Site Recruiting Porto,4200 319,Portugal South Africa #### Novartis Investigative Site Recruiting Bloemfontein,Free State,9301,South Africa Spain #### Novartis Investigative Site Recruiting Esplugues,Barcelona,08950,Spain #### Novartis Investigative Site Recruiting Málaga,29011,Spain #### Novartis Investigative Site Recruiting Seville,41013,Spain #### Novartis Investigative Site Recruiting Badalona,Barcelona,08916,Spain #### Novartis Investigative Site Recruiting Pamplona,Navarre,31008,Spain #### Novartis Investigative Site Recruiting Barcelona,08041,Spain #### Novartis Investigative Site Recruiting Cadiz,Andalusia,11009,Spain #### Novartis Investigative Site Recruiting Elche,Alicante,03203,Spain Taiwan #### Novartis Investigative Site Recruiting Taipei,11217,Taiwan #### Novartis Investigative Site Recruiting Taipei,111045,Taiwan Turkey (Türkiye) #### Novartis Investigative Site Recruiting Ankara,Yenimahalle,06500,Turkey (Türkiye) #### Novartis Investigative Site Recruiting Adana,Saricam,01330,Turkey (Türkiye) #### Novartis Investigative Site Recruiting Izmir,35100,Turkey (Türkiye) United Kingdom #### Novartis Investigative Site Recruiting London,Nw3 2qg,United Kingdom #### Novartis Investigative Site Recruiting West Midlands,Birmingham,B4 6nh,United Kingdom United States #### UC San Francisco Medical Center Recruiting San Francisco,California,94143-0348,United States ###### Luis Gay Phone: [+1 415 476 8338]() Email: ###### Martin Thelin #### Excel Medical Clinical Trials LLC Recruiting Boca Raton,Florida,33434,United States ###### Rasha Youssef ###### Mariana Sanchez Villa Phone: [+1 561 756 8206]() Email: #### UC San Francisco Medical Center Recruiting San Francisco,California,94143,United States ###### Luis Gay Phone: [+1 415 990 7296]() Email: ###### Martin Thelin #### Icahn School of Med at Mt Sinai Recruiting New York,New York,10029,United States ###### Jay Krishna Katragadda Phone: [+1 212 659 9174]() Email: ###### Joseph Mahgerefteh #### West Virginia Childrens Hospital Recruiting Morgantown,West Virginia,26506,United States ###### Lee Pyles ###### Robin Hoffer Phone: [+1 724-972-9170]() Email: ###### Robin Hoffer Phone: [+1 878 379 4121]() Email: #### Childrens National Hospital Recruiting Washington D.C.,District of Columbia,20010,United States ###### Carlos Carhuas Phone: [+1 202 476 3578]() Email: ###### Carlos Jesus Carhuas Phone: [+1 202 476 5000]() Email: ###### Sarah Clauss #### Virginia Commonwealth University Recruiting Richmond,Virginia,23298,United States ###### Megan Scott Email: ###### Bradford McQuilkin #### Primary Childrens Medical Center Recruiting Salt Lake City,Utah,84113,United States ###### Linda Lambert Email: ###### Linda Lambert Phone: [+1 801 587 9153]() Email: ###### Adam Ware ## 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: