NCT05519475

A Precision Medicine Approach Using Gene Silencing to Treat a Chronic Liver Disease Called Metabolic Dysfunction-Associated Steatohepatitis (MASH) in Adult Participants at Increased Genetic Risk for This Condition

A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of siRNA Gene Silencing for the Treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH) in Participants With Genetic Risk Factors

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 90 (estimated)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This study is researching an investigational drug, ALN-HSD called "study drug". This study is focused on participants who are known to have metabolic dysfunction-associated steatohepatitis (MASH). MASH is a form of metabolic dysfunction-associated steatotic liver disease (MASLD). MASH occurs when fat builds up in liver cells, damaging them, and making the liver inflamed and stiff from fibrosis (scar tissue). MASH can progress to cirrhosis (long term scarring) and liver failure (when the liver cannot perform its job). The aim of the study is to see the effect of the study drug on lessening liver scarring side effects related to MASH. The study is looking at several other research questions, including: * How ALN-HSD works to improve liver function and lessen MASH-related inflammation in the liver * What side effects may happen from receiving the study drug * How much study drug and study drug metabolites (byproduct of the body breaking down the study drug) are in the blood at different times * Better understanding of the study drug and MASH

Conditions

• Metabolic Dysfunction-associated Steatohepatitis (MASH)

Interventions

Type	Name	Description
DRUG	ALN-HSD	Administered by subcutaneous injection (SC)
DRUG	Placebo	Administered by SC injection

Timeline

• Start date: 2023-02-09
• Primary completion: 2027-09-08
• Completion: 2027-09-08
• First posted: 2022-08-29
• Last updated: 2026-02-03

Locations

71 sites across 4 countries: United States, Japan, Puerto Rico, South Korea

Regulatory

• FDA-regulated drug study