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.
Key facts
- Who runs it
- Regeneron Pharmaceuticals
- Where
- Guaynabo and San Juan, Puerto RicoSee the 4 sitesAlso in 3 other countries, 67 sites
- Trial code
- 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
- Status
- Active, not recruiting
- Phase
- Phase 2
- People taking part
- 120 people, planned
- Started
- 9 Feb 2023
- Expected to end
- 7 Dec 2028
- Type of study
- Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
- Treatments tested
- ALN-HSD
- Placebo
- Record updated
- 12 November 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with MASH, a liver disease with scarring (fibrosis stage 2 or 3), who also have a genetic risk that meets the protocol rules.
- Age
- 18 to 75 years
- Sex
- Women and men can both join.
- Disease stage
- MASH with fibrosis stage 2 or 3 (NASH-CRN) and an NAS score of 3 or more.
- Earlier treatment
- People who had earlier treatment with an investigational drug aimed at HSD17B13 or PNPLA3 (for example ALN-HSD, ARO-HSD, ALN-PNP, AZD2693) cannot join.
- Biomarker or test result
- The genotype must meet the protocol criteria. A protocol-defined FibroScan-AST (FAST) score is needed at screening or within about 12 weeks of it.
Main reasons for not taking part
- Other known forms of chronic liver disease
- Alcohol or other substance abuse within the last year or during screening
- History of Type 1 diabetes
- Bariatric surgery within about 5 years before the trial or planned during it
- Earlier use of an investigational drug aimed at HSD17B13 or PNPLA3
The registry criteria, word for word
Show all 10 criteriaShow less
Who can take part
- 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
Who cannot take part
- 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
Where in Puerto Rico
Also in 3 other countries, 67 sites
- Isis Clinical Research CenterGuaynabo, Puerto RicoNo longer taking part
- Fundacion de Investigacion (FDI) Clinical ResearchSan Juan, Puerto RicoNo longer taking part
- Klinical InvestigationsSan Juan, Puerto RicoNo longer taking part
- Latin Clinical Trial CenterSan Juan, Puerto RicoNo longer taking part
About the trial
What the trial wants to find out
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
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
The 2 groups in the trial
- ALN-HSDTreatment being tested
Randomized 1:1
- PlaceboPlacebo, for comparison
Randomized 1:1
Data and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- 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
- Plain-language title
- Tests ALN-HSD gene silencing for liver scarring in adults with MASHGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 9 November 2026.
- Record updated
- 12 November 2026