Study design & results
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Trial intelligence / Profile preview
ORION-19 inclisiran study in children with homozygous familial hypercholesterolemia
2024-514595-41-00Phase 3Authorised
- Sponsor
- Novartis
- Start
- Aug 2025
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 13
01
Overview
Phase 3, randomised, double-blind trial in homozygous familial hypercholesterolemia (HoFH) comparing Inclisiran then open-label inclisiran with Placebo then open-label inclisiran (2 arms). Primary endpoint: Percentage change in LDL-C from baseline to Day 330. Planned start Aug 2025; status: authorised.
02
Drugs
inclisiran
03
Conditions
homozygous familial hypercholesterolemia (HoFH)
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