Tests ALN-HSD gene silencing for liver scarring in adults with MASH
Tests ALN-HSD against placebo in adults with fatty liver disease and genetic risk.
Plain-language title generated from the registry record
Registry title: 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
Key facts
- Where
- Busan, Daegu, Seongnam-si, and Seoul, South KoreaSee the 5 sites
- Phase
- Phase 2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 75 years old?
Source: NCT05519475, who can take part
Where in South Korea
- Pusan National University HospitalBusan, South KoreaNo longer taking part
- Keimyung University Dongsan HospitalDaegu, South KoreaNo longer taking part
- Kyungpook National University HospitalDaegu, South KoreaNo longer taking part
- Seoul National University Bundang HospitalSeongnam-si, South KoreaNo longer taking part
- Hanyang University Seoul HospitalSeoul, South KoreaNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Regeneron Pharmaceuticals
- Trial code
- NCT05519475
- Record updated
- 8 October 2026
- Original description
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 related to MASH.Read full original descriptionShow less
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
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 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
Inclusion: Adult male or female ≥18 years (or country's legal age of adulthood); A diagnosis of MASH with Fibrosis (F) stage 2 or 3, according to the NASH-CRN; NAS score ≥3, as defined in the protocol; Meets genotype criteria for study enrollment, as defined in the protocol; Has a protocol defined FibroScan®-AST (FAST) score at screening or within approximately 12 weeks of screening.
Exclusion: Bariatric surgery within approximately 5 years prior to or planned during the study period; Prior exposure to any investigational drug targeting HSD17B13 or patatin-like phospholipase domain containing 3 (PNPLA3) (eg, ALN-HSD, ARO-HSD, ALN-PNP, AZD2693); Note: Other protocol-defined Inclusion/Exclusion Criteria apply; Evidence of other forms of known chronic liver disease, as defined in the protocol; Known history of alcohol or other substance abuse within the last year or at any time during screening, as defined in the protocol; History of Type 1 diabetes.