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Clinical Trials in the UK / NCT06597019
Recruiting Phase 3

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

NCT06597019 · tracked via the Priya Life Science UK tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 3
Started
2024-12-09
Last updated
2026-05-15

Condition(s) studied

Familial Hypercholesterolemia - Heterozygous

Investigational drug(s) / intervention(s)

InclisiranPlacebo

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 \<23 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 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).

Eligibility

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

Primary outcome measure(s)

Trial sites (66)

FacilityCityRegionStatus
UC San Francisco Medical Center San Francisco California Recruiting
UC San Francisco Medical Center San Francisco California Recruiting
Children's National Hospital Washington D.C. District of Columbia Not Yet Recruiting
Childrens National Hospital Washington D.C. District of Columbia Recruiting
Excel Medical Clinical Trials LLC Boca Raton Florida Recruiting
Icahn School of Med at Mt Sinai New York New York Recruiting
Primary Childrens Medical Center Salt Lake City Utah Not Yet Recruiting
Primary Childrens Medical Center Salt Lake City Utah Recruiting
Virginia Commonwealth University Richmond Virginia Recruiting
West Virginia Childrens Hospital Morgantown West Virginia Recruiting
West Virginia Childrens Hospital Morgantown West Virginia Recruiting
Novartis Investigative Site Buenos Aires Argentina Recruiting
Novartis Investigative Site CABA Argentina Recruiting
Novartis Investigative Site Salzburg Austria Recruiting
Novartis Investigative Site Vienna Austria Recruiting
Novartis Investigative Site Brussels Belgium Recruiting
Novartis Investigative Site Leuven Belgium Recruiting
Novartis Investigative Site Fortaleza Ceará Recruiting
Novartis Investigative Site Rio de Janeiro Rio de Janeiro Recruiting
Novartis Investigative Site Porto Alegre Rio Grande do Sul Recruiting
Novartis Investigative Site São Paulo São Paulo Recruiting
Novartis Investigative Site Beijing Beijing Municipality Recruiting
Novartis Investigative Site Shanghai China Recruiting
Novartis Investigative Site Prague Czechia Recruiting
Novartis Investigative Site Prague Czechia Recruiting
Novartis Investigative Site Marseille France Recruiting
Novartis Investigative Site Nantes France Recruiting
Novartis Investigative Site Paris France Recruiting
Novartis Investigative Site Freiburg im Breisgau Baden-Wurttemberg Recruiting
Novartis Investigative Site Frankfurt am Main Hesse Recruiting
Novartis Investigative Site Hanover Germany Recruiting
Novartis Investigative Site Athens Greece Recruiting
Novartis Investigative Site Ioannina Greece Recruiting
Novartis Investigative Site Hong Kong Hong Kong Recruiting
Novartis Investigative Site Budapest Hungary Recruiting
Novartis Investigative Site Jerusalem Israel Recruiting
Novartis Investigative Site Ramat Gan Israel Recruiting
Novartis Investigative Site Milan MI Recruiting
Novartis Investigative Site Roma RM Recruiting
Novartis Investigative Site Torino TO Recruiting

+ 26 more sites — see the full list on the official registry below.

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 NCT06597019 on ClinicalTrials.gov ↗ ← All trials in the UK