Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

Recruitment Status
RECRUITING
(See Contacts and Locations)Verified January 2026 by Novartis Pharmaceuticals
Sponsor
Novartis Pharmaceuticals
Information Provided by (Responsible Party)
Novartis Pharmaceuticals
Clinicaltrials.gov Identifier
NCT06597006
Other Study ID Numbers:
CKJX839C12304
First Submitted
September 10, 2024
First Posted
September 18, 2024
Last Update Posted
February 19, 2026
Last Verified
January 2026

ClinicalTrials.gov processed this data on February 2026. 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 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 or DiseaseIntervention/Treatment
Familial Hypercholesterolemia - Homozygous
Drug: InclisiranDrug: Placebo

Study Design

Study TypeInterventional
Actual Enrollment9 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 (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol
Study Start DateFebruary 27, 2025
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, inclisiran sodium 180 mg s.c. for participants with body weight \&lt;23 kg to &ge;16 kg, or inclisiran sodium 100 mg s.c. for participants with body weight \&lt;16 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)
    Evaluate the effect of inclisiran compared to placebo on 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)
    Evaluate the effect of inclisiran compared to placebo on reducing LDL-C \[time-adjusted percent change\] over Year 1
  2. Percent change in LDL-C, total cholesterol, non-HDL-C, 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
  3. 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
  4. 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
  5. Absolute change in LDL-C, total cholesterol, non-HDL-C, 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
  6. 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
  7. 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
  8. 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
  9. 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, 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

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, 94143Childrens National Hospital | Washington D.C. District of Columbia, United States, 20010Washington Univ School Of Medicine | St Louis Missouri, United States, 63110Novartis Investigative Site | Vienna , Austria, 1090Novartis Investigative Site | Beijing Beijing Municipality, China, 100013Novartis Investigative Site | Frankfurt am Main Hesse, Germany, 60590Novartis Investigative Site | Ioannina , Greece, 455 00Novartis Investigative Site | Thessaloniki , Greece, 546 42Novartis Investigative Site | Kota Bharu Kelantan, Malaysia, 16150Novartis Investigative Site | Amsterdam North Holland, Netherlands, 1105 AZNovartis Investigative Site | Bloemfontein Free State, South Africa, 9301Novartis Investigative Site | Taichung , Taiwan, 407219Novartis Investigative Site | Taipei , Taiwan, 111045Novartis Investigative Site | Adana Saricam, Turkey (Türkiye), 01330Novartis Investigative Site | Ankara Yenimahalle, Turkey (Türkiye), 06500Novartis Investigative Site | Izmir , Turkey (Türkiye), 35100Novartis Investigative Site | Southampton , United Kingdom, SO16 6YD
Investigators
Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals