Trial intelligence / Profile preview

Inclisiran in children with homozygous familial hypercholesterolemia

NL-OMON59348Phase 3Not yet recruiting

Sponsor
Novartis
Start
Jul 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
1
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Overview

Phase 3, randomised, double-blind trial in homozygous familial hypercholesterolemia (HoFH) comparing Inclisiran with Placebo (2 arms). Primary endpoint: Percentage change in LDL-C from baseline to Day 330. Planned start Jul 2025; status: not yet recruiting.

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Drugs

inclisiran
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Conditions

homozygous familial hypercholesterolemia (HoFH)

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