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Clinical Trials in the UK / NCT06597006
Recruiting Phase 3

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

NCT06597006 · tracked via the Priya Life Science UK tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 3
Started
2025-02-28
Last updated
2026-07-24

Condition(s) studied

Familial Hypercholesterolemia - Homozygous

Investigational drug(s) / intervention(s)

InclisiranPlacebo

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 \<23 kg to ≥16 kg, or inclisiran sodium 100 mg s.c. for participants with body weight \<16 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.

Placebo: Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Study summary

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).

Eligibility

Sex
ALL
Min age
2 Years
Max age
11 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (19)

FacilityCityRegionStatus
UC San Francisco Medical Center San Francisco California Recruiting
Childrens National Hospital Washington D.C. District of Columbia Recruiting
Washington Univ School Of Medicine St Louis Missouri Recruiting
Primary Childrens Medical Center Salt Lake City Utah Recruiting
Novartis Investigative Site Vienna Austria Recruiting
Novartis Investigative Site Beijing Beijing Municipality Recruiting
Novartis Investigative Site Frankfurt am Main Hesse Recruiting
Novartis Investigative Site Ioannina Greece Recruiting
Novartis Investigative Site Thessaloniki Greece Recruiting
Novartis Investigative Site Kota Bharu Kelantan Recruiting
Novartis Investigative Site Amsterdam North Holland Recruiting
Novartis Investigative Site Bloemfontein Free State Recruiting
Novartis Investigative Site Johannesburg Gauteng Recruiting
Novartis Investigative Site Taichung Taiwan Recruiting
Novartis Investigative Site Taipei Taiwan Recruiting
Novartis Investigative Site Adana Saricam Recruiting
Novartis Investigative Site Ankara Yenimahalle Recruiting
Novartis Investigative Site Izmir Turkey (Türkiye) Recruiting
Novartis Investigative Site Southampton United Kingdom Recruiting

On this site

📄 Leqvio (inclisiran) drug profile →

More Novartis Pharmaceuticals trials in the UK

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06597006 on ClinicalTrials.gov ↗ ← All trials in the UK