NCT05519475From ClinicalTrials.govRecruiting

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

Metabolic Dysfunction-Associated SteatoHepatitis (MASH)

At a glance

Phase: Phase 2
Study type: Interventional
Sponsor: Regeneron Pharmaceuticals
Enrolment target: 120
Started: 9 February 2023
Main results due: 27 April 2028
Study sites: 71
Registry updated: 25 September 2026

Can you take part?

Ages 18 years to 75 years.
Open to any sex.
You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full eligibility criteria

Key Inclusion Criteria:

1. Adult male or female ≥18 years (or country's legal age of adulthood)
2. A diagnosis of MASH with Fibrosis (F) stage 2 or 3, according to the NASH-CRN
3. NAS score ≥3, as defined in the protocol
4. Meets genotype criteria for study enrollment, as defined in the protocol
5. Has a protocol defined FibroScan®-AST (FAST) score at screening or within approximately 12 weeks of screening

Key Exclusion Criteria:

1. Evidence of other forms of known chronic liver disease, as defined in the protocol
2. Known history of alcohol or other substance abuse within the last year or at any time during screening, as defined in the protocol
3. History of Type 1 diabetes
4. Bariatric surgery within approximately 5 years prior to or planned during the study period
5. 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

What this study is about

In the sponsor’s own words, from the registry.

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

Study sites(71)

Arizona Liver Health
Chandler, Arizona, United States
Recruiting
The Institute for Liver Health II LLC DBA Arizona Clinical Trials - Flagstaff
Flagstaff, Arizona, United States
Terminated
The Institute for Liver Health II LLC DBA Arizona Liver Health - Peoria
Peoria, Arizona, United States
Recruiting
Adobe Clinical Research
Tucson, Arizona, United States
Recruiting
Arizona Liver Health - Tucson
Tucson, Arizona, United States
Recruiting
Del Sol Research Management, LLC
Tucson, Arizona, United States
Recruiting
San Fernando Valley Health Institute
Canoga Park, California, United States
Terminated
Velocity Clinical Research
Chula Vista, California, United States
Terminated
Southern California Research Center
Coronado, California, United States
Recruiting
Velocity Clinical Research
Gardena, California, United States
Terminated
Velocity Clinical Research - Huntington Park
Huntington Park, California, United States
Terminated
Om Research Llc
Lancaster, California, United States
Recruiting
Ark Clinical Research - Long Beach
Long Beach, California, United States
Completed
Ark Clinical Research, LLC
Long Beach, California, United States
Completed
Velocity Clinical Research - Westlake
Los Angeles, California, United States
Terminated
Clinnova Research Solutions
Orange, California, United States
Recruiting
Knowledge Research Center, St. Joseph's Medical Tower
Orange, California, United States
Recruiting
National Research Institute
Panorama City, California, United States
Terminated
California Liver Research Institute
Pasadena, California, United States
Recruiting
Cadena Care Inst.
Poway, California, United States
Terminated
Inland Empire Clinical Trials
Rialto, California, United States
Recruiting
Precision Research Institute - San Diego
San Diego, California, United States
Terminated
University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States
Recruiting
Synergy Healthcare LLC
Bradenton, Florida, United States
Terminated
Evolution Clinical Trials
Hialeah Gardens, Florida, United States
Recruiting
Health Awareness, inc.
Jupiter, Florida, United States
Recruiting
Miami Clinical Research
Miami, Florida, United States
Terminated
International Medical Investigational Centers (IMIC) Incorporated
Miami, Florida, United States
Terminated
Genoma Research Group, Inc.
Miami, Florida, United States
Recruiting
US Associates in Research, LLC
Miami, Florida, United States
Terminated
Med Research of Florida, LLC
Miami, Florida, United States
Recruiting
Charter Research LLC - Orlando
Orlando, Florida, United States
Recruiting
Indiana University School of Medicine
Indianapolis, Indiana, United States
Recruiting
Deaconess Clinic - GI Specialty Center
Newburgh, Indiana, United States
Terminated
Tandem Clinical Research
Metairie, Louisiana, United States
Recruiting
Mercy Medical Center
Baltimore, Maryland, United States
Terminated
GI Associates
Flowood, Mississippi, United States
Recruiting
Washington University School of Medicine
St Louis, Missouri, United States
Recruiting
Columbia University Irving Medical Center
New York, New York, United States
Recruiting
Geisinger Medical Center
Danville, Pennsylvania, United States
Recruiting
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Recruiting
University of Pennsylvania - Perelman Center for Advanced Medicine
Philadelphia, Pennsylvania, United States
Recruiting
Austin Clinical Research, LLC
Austin, Texas, United States
Terminated
American Research Corporation
Austin, Texas, United States
Recruiting
Pinnacle Clinical Research - Austin
Austin, Texas, United States
Recruiting
South Texas Research Institute (STRI) - Brownsville
Brownsville, Texas, United States
Terminated
Velocity Clinical Research - Austin
Cedar Park, Texas, United States
Terminated
Methodist Dallas Medical Center
Dallas, Texas, United States
Recruiting
South Texas Research Institute (STRI) - Edinburg
Edinburg, Texas, United States
Terminated
GI Alliance Research
Fort Worth, Texas, United States
Recruiting
Allure Health at Mt. Olympus Medical Research
Friendswood, Texas, United States
Terminated
Advanced Medical Trials
Georgetown, Texas, United States
Recruiting
Pioneer Research Solutions, Inc.
Houston, Texas, United States
Recruiting
R & H Clinical Research, Inc.
Katy, Texas, United States
Terminated
Great Lakes Research Institute
Pharr, Texas, United States
Terminated
American Research Corporation at The Texas Liver Institute
San Antonio, Texas, United States
Recruiting
Pinnacle Clinical Research - San Antonio
San Antonio, Texas, United States
Recruiting
R & H Clinical Research
Stafford, Texas, United States
Terminated
Intermountain Health
Murray, Utah, United States
Terminated
University of Virginia Medical Center
Charlottesville, Virginia, United States
Recruiting

Showing 60 of 71 sites — filter to narrow it down. 36 of the 71 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT05519475. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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