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

Recruitment Status
RECRUITING
(See Contacts and Locations)Verified October 2025 by Novartis Pharmaceuticals
Sponsor
Novartis Pharmaceuticals
Information Provided by (Responsible Party)
Novartis Pharmaceuticals
Clinicaltrials.gov Identifier
NCT06597019
Other Study ID Numbers:
CKJX839C12303
First Submitted
September 10, 2024
First Posted
September 18, 2024
Last Update Posted
November 20, 2025
Last Verified
October 2025

ClinicalTrials.gov processed this data on November 2025. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

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 or DiseaseIntervention/Treatment
Familial Hypercholesterolemia - Heterozygous
Drug: InclisiranDrug: Placebo

Study Design

Study TypeInterventional
Actual Enrollment51 participants
Design AllocationRandomized
Interventional ModelParallel Assignment
MaskingDouble
Primary PurposeTreatment
Official TitleTwo 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
Study Start DateDecember 8, 2024
Actual Primary Completion Date1yr 10mos from now
Actual Study Completion Date2yrs 11mos from now

Groups and Cohorts

Group/CohortIntervention/Treatment
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)
Drug: Inclisiran
Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight &ge;23 kg and inclisiran sodium 180 mg s.c. for participants with body weight \&lt;23 kg. The dose level is based on the participant&#039;s body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.
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)
Drug: Placebo
Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Outcome Measures

Primary Outcome Measures
  1. Percentage change in LDL-C from baseline to Day 330 (Year 1)
    Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[percent change\] at Day 330
Secondary Outcome Measures
  1. Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330 (Year 1)
    Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[time-adjusted percent change\] over Year 1
  2. Absolute change in LDL-C from baseline to Day 330 (Year 1)
    Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[absolute change\] at Day 330 (Year 1)
  3. Percent change in PCSK9 from baseline to Day 330 (Year 1)
    Demonstrate superiority of inclisiran compared to placebo in reducing PCSK9, total cholesterol, Apo B, and non-HDL-C \[percent change\] at Day 330 (Year 1)
  4. Percent change in total cholesterol, non-HDL-C from baseline to Day 330 (Year 1)
    Demonstrate superiority of inclisiran compared to placebo in reducing PCSK9, total cholesterol, Apo B, and non-HDL-C \[percent change\] at Day 330 (Year 1)
  5. Percent change in Apo B from baseline to Day 330 (Year 1)
    Demonstrate superiority of inclisiran compared to placebo in reducing PCSK9, total cholesterol, Apo B, and non-HDL-C \[percent change\] at Day 330 (Year 1)
  6. Percent change in LDLC, total cholesterol, non-HDLC, triglycerides, HDL-C, VLDL-C from baseline to each assessment time up to Day 720 (Year 2)
    Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
  7. Percent change in PCSK9 from baseline to each assessment time up to Day 720 (Year 2)
    Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
  8. Percent change in Apo B, Apo A1 from baseline to each assessment time up to Day 720 (Year 2)
    Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
  9. Absolute change in LDLC, total cholesterol, non-HDLC, triglycerides, HDL-C, VLDL-C from baseline to each assessment time up to Day 720 (Year 2)
    Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
  10. Absolute change in PCSK9 from baseline to each assessment time up to Day 720 (Year 2)
    Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
  11. Absolute change in Apo B, Apo A1 from baseline to each assessment time up to Day 720 (Year 2)
    Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
  12. Percent change in Lp(a) from baseline to each assessment time up to Day 720 (Year 2)
    Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
  13. Absolute change in Lp(a) from baseline to each assessment time up to Day 720 (Year 2)
    Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time

Eligibility Criteria

Ages Eligible for Study(Child)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
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

Contacts and Locations

Sponsors and CollaboratorsNovartis Pharmaceuticals
Locations
UC San Francisco Medical Center | San Francisco California, United States, 94143UC San Francisco Medical Center | San Francisco California, United States, 94143Children's National Hospital | Washington D.C. District of Columbia, United States, 20010Childrens National Hospital | Washington D.C. District of Columbia, United States, 20010Excel Medical Clinical Trials LLC | Boca Raton Florida, United States, 33434Icahn School of Med at Mt Sinai | New York New York, United States, 10029Primary Childrens Medical Center | Salt Lake City Utah, United States, 84113Primary Childrens Medical Center | Salt Lake City Utah, United States, 84113Virginia Commonwealth University | Richmond Virginia, United States, 23298West Virginia Childrens Hospital | Morgantown West Virginia, United States, 26506West Virginia Childrens Hospital | Morgantown West Virginia, United States, 26506Novartis Investigative Site | Buenos Aires , Argentina, C1245AAMNovartis Investigative Site | CABA , Argentina, C1181ACHNovartis Investigative Site | Salzburg , Austria, 5020Novartis Investigative Site | Vienna , Austria, 1090Novartis Investigative Site | Brussels , Belgium, 1200Novartis Investigative Site | Leuven , Belgium, 3000Novartis Investigative Site | Fortaleza Ceará, Brazil, 60430-275Novartis Investigative Site | Rio de Janeiro Rio de Janeiro, Brazil, 20211-340Novartis Investigative Site | Porto Alegre Rio Grande do Sul, Brazil, 90020-020Novartis Investigative Site | São Paulo São Paulo, Brazil, 05403 000Novartis Investigative Site | Beijing Beijing Municipality, China, 100013Novartis Investigative Site | Shanghai , China, 200127Novartis Investigative Site | Prague , Czechia, 128 08Novartis Investigative Site | Prague , Czechia, 150 06Novartis Investigative Site | Marseille , France, 13885Novartis Investigative Site | Nantes , France, 44093Novartis Investigative Site | Paris , France, 75012Novartis Investigative Site | Freiburg im Breisgau Baden-Wurttemberg, Germany, 79106Novartis Investigative Site | Frankfurt am Main Hesse, Germany, 60590Novartis Investigative Site | Hanover , Germany, 30173Novartis Investigative Site | Athens , Greece, 115 27Novartis Investigative Site | Ioannina , Greece, 455 00Novartis Investigative Site | Hong Kong , Hong Kong, 999077Novartis Investigative Site | Budapest , Hungary, 1026Novartis Investigative Site | Jerusalem , Israel, 9112001Novartis Investigative Site | Ramat Gan , Israel, 5265601Novartis Investigative Site | Milan MI, Italy, 20162Novartis Investigative Site | Roma RM, Italy, 00165Novartis Investigative Site | Torino TO, Italy, 10126Novartis Investigative Site | Verona VR, Italy, 3712Novartis Investigative Site | Kuala Lumpur Kuala Lumpur, Malaysia, 50586Novartis Investigative Site | Amsterdam North Holland, Netherlands, 1105 AZNovartis Investigative Site | Bialystok , Poland, 15-274Novartis Investigative Site | Gdansk , Poland, 80 952Novartis Investigative Site | Lodz Łódź Voivodeship, Poland, 93-338Novartis Investigative Site | Coimbra , Portugal, 3000-602Novartis Investigative Site | Lisbon , Portugal, 1649-035Novartis Investigative Site | Porto , Portugal, 4050-651Novartis Investigative Site | Porto , Portugal, 4200 319Novartis Investigative Site | Bloemfontein Free State, South Africa, 9301Novartis Investigative Site | Elche Alicante, Spain, 03203Novartis Investigative Site | Cadiz Andalusia, Spain, 11009Novartis Investigative Site | Esplugues Barcelona, Spain, 08950Novartis Investigative Site | Badalona Catalonia, Spain, 08916Novartis Investigative Site | Pamplona Navarre, Spain, 31008Novartis Investigative Site | Barcelona , Spain, 08041Novartis Investigative Site | Málaga , Spain, 29011Novartis Investigative Site | Seville , Spain, 41013Novartis Investigative Site | Taipei , Taiwan, 111045Novartis Investigative Site | Taipei , Taiwan, 11217Novartis Investigative Site | Adana Saricam, Turkey (Türkiye), 01330Novartis Investigative Site | Ankara Yenimahalle, Turkey (Türkiye), 06500Novartis Investigative Site | Izmir , Turkey (Türkiye), 35100Novartis Investigative Site | West Midlands Birmingham, United Kingdom, B4 6NHNovartis Investigative Site | London , United Kingdom, NW3 2QG
Investigators
Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals