Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Primary outcome measure(s)
- Percentage change in LDL-C from baseline to Day 330 (Year 1) — Baseline and Day 330
Evaluate the effect of inclisiran compared to placebo on reducing LDL-C \[percent change\] at Day 330
Trial sites (19)
| Facility | City | Region | Status |
|---|---|---|---|
| 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
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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