NCT05519475RecruitingPhase 2
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
Source: ClinicalTrials.gov · Last updated 8 Oct 2026
Brief summary (registry, in English)
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
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 120 (estimated)
- First submitted
- 15 Aug 2022
- Start date
- 9 Feb 2023
- Primary completion date
- 27 Apr 2028
- Lead sponsor
- Regeneron Pharmaceuticals
- Diseases
- Fatty liver disease (MASLD/MASH)
- NCT number
- NCT05519475
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 120
- From 9 Feb 2023 to the primary completion date, 27 Apr 2028
- 71 sites in 4 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
Other illness
- 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
Who the study is looking for
- 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
Age in the registry: 18 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (71)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| JCHO Hokkaido Hospital Coordinator of this site? Confirm its status | Sapporo | Japan | Terminated |
| Pusan National University Hospital Coordinator of this site? Confirm its status | Busan | South Korea | Terminated |
| Kyungpook National University Hospital Coordinator of this site? Confirm its status | Daegu | South Korea | Terminated |
| Keimyung University Dongsan Hospital Coordinator of this site? Confirm its status | Daegu | South Korea | Terminated |
| Seoul National University Bundang Hospital Coordinator of this site? Confirm its status | Seongnam-si | South Korea | Terminated |
| Hanyang University Seoul Hospital Coordinator of this site? Confirm its status | Seoul | South Korea | Terminated |
| Isis Clinical Research Center Coordinator of this site? Confirm its status | Guaynabo | Puerto Rico | Terminated |
| Klinical Investigations Coordinator of this site? Confirm its status | San Juan | Puerto Rico | Terminated |
| Fundacion de Investigacion (FDI) Clinical Research Coordinator of this site? Confirm its status | San Juan | Puerto Rico | Recruiting |
| Latin Clinical Trial Center Coordinator of this site? Confirm its status | San Juan | Puerto Rico | Terminated |
| University of Colorado Anschutz Medical Campus Coordinator of this site? Confirm its status | Aurora | United States | Recruiting |
| Pinnacle Clinical Research - Austin Coordinator of this site? Confirm its status | Austin | United States | Recruiting |
| American Research Corporation Coordinator of this site? Confirm its status | Austin | United States | Recruiting |
| Austin Clinical Research, LLC Coordinator of this site? Confirm its status | Austin | United States | Terminated |
| Mercy Medical Center Coordinator of this site? Confirm its status | Baltimore | United States | Terminated |
| Synergy Healthcare LLC Coordinator of this site? Confirm its status | Bradenton | United States | Terminated |
| South Texas Research Institute (STRI) - Brownsville Coordinator of this site? Confirm its status | Brownsville | United States | Terminated |
| San Fernando Valley Health Institute Coordinator of this site? Confirm its status | Canoga Park | United States | Terminated |
| Velocity Clinical Research - Austin Coordinator of this site? Confirm its status | Cedar Park | United States | Terminated |
| University of Virginia Medical Center Coordinator of this site? Confirm its status | Charlottesville | United States | Recruiting |
| Velocity Clinical Research Coordinator of this site? Confirm its status | Chula Vista | United States | Terminated |
| Southern California Research Center Coordinator of this site? Confirm its status | Coronado | United States | Recruiting |
| Methodist Dallas Medical Center Coordinator of this site? Confirm its status | Dallas | United States | Recruiting |
| Geisinger Medical Center Coordinator of this site? Confirm its status | Danville | United States | Recruiting |
| South Texas Research Institute (STRI) - Edinburg Coordinator of this site? Confirm its status | Edinburg | United States | Terminated |
| The Institute for Liver Health II LLC DBA Arizona Clinical Trials - Flagstaff Coordinator of this site? Confirm its status | Flagstaff | United States | Terminated |
| GI Associates Coordinator of this site? Confirm its status | Flowood | United States | Recruiting |
| GI Alliance Research Coordinator of this site? Confirm its status | Fort Worth | United States | Recruiting |
| Allure Health at Mt. Olympus Medical Research Coordinator of this site? Confirm its status | Friendswood | United States | Terminated |
| Velocity Clinical Research Coordinator of this site? Confirm its status | Gardena | United States | Terminated |
| Advanced Medical Trials Coordinator of this site? Confirm its status | Georgetown | United States | Recruiting |
| Penn State Health Milton S. Hershey Medical Center Coordinator of this site? Confirm its status | Hershey | United States | Recruiting |
| Evolution Clinical Trials Coordinator of this site? Confirm its status | Hialeah Gardens | United States | Recruiting |
| Pioneer Research Solutions, Inc. Coordinator of this site? Confirm its status | Houston | United States | Recruiting |
| Velocity Clinical Research-Huntington Park Coordinator of this site? Confirm its status | Huntington Park | United States | Terminated |
| Indiana University School of Medicine Coordinator of this site? Confirm its status | Indianapolis | United States | Recruiting |
| Health Awareness, Inc Coordinator of this site? Confirm its status | Jupiter | United States | Recruiting |
| R&H Clinical Research, Inc. Coordinator of this site? Confirm its status | Katy | United States | Terminated |
| Om Research LLC Coordinator of this site? Confirm its status | Lancaster | United States | Recruiting |
| Ark Clinical Research - Long Beach Coordinator of this site? Confirm its status | Long Beach | United States | Terminated |
| Ark Clinical Research, LLC Coordinator of this site? Confirm its status | Long Beach | United States | Terminated |
| Velocity Clinical Research, Westlake Coordinator of this site? Confirm its status | Los Angeles | United States | Terminated |
| Tandem Clinical Research Coordinator of this site? Confirm its status | Metairie | United States | Recruiting |
| International Medical Investigational Centers (IMIC) Incorporated Coordinator of this site? Confirm its status | Miami | United States | Terminated |
| Med Research of Florida, LLC Coordinator of this site? Confirm its status | Miami | United States | Recruiting |
| Genoma Research Group, Inc. Coordinator of this site? Confirm its status | Miami | United States | Recruiting |
| Miami Clinical Research Coordinator of this site? Confirm its status | Miami | United States | Terminated |
| US Associates in Research, LLC Coordinator of this site? Confirm its status | Miami | United States | Terminated |
| Intermountain Health Coordinator of this site? Confirm its status | Murray | United States | Terminated |
| Deaconess Clinic - GI Specialty Center Coordinator of this site? Confirm its status | Newburgh | United States | Terminated |
| Columbia University Irving Medical Center Coordinator of this site? Confirm its status | New York | United States | Recruiting |
| Knowledge Research Center, St. Joseph's Medical Tower Coordinator of this site? Confirm its status | Orange | United States | Recruiting |
| Clinnova Research Solutions Coordinator of this site? Confirm its status | Orange | United States | Recruiting |
| Charter Research LLC - Orlando Coordinator of this site? Confirm its status | Orlando | United States | Recruiting |
| National Research Institute Coordinator of this site? Confirm its status | Panorama City | United States | Terminated |
| California Liver Research Institute Coordinator of this site? Confirm its status | Pasadena | United States | Recruiting |
| The Institute for Liver Health II LLC DBA Arizona Liver Health - Peoria Coordinator of this site? Confirm its status | Peoria | United States | Recruiting |
| Great Lakes Research Institute Coordinator of this site? Confirm its status | Pharr | United States | Terminated |
| University of Pennsylvania - Perelman Center for Advanced Medicine Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
| Cadena Care Inst. Coordinator of this site? Confirm its status | Poway | United States | Terminated |
| Inland Empire Clinical Trials Coordinator of this site? Confirm its status | Rialto | United States | Recruiting |
| Richmond Veterans Administration Medical Center Coordinator of this site? Confirm its status | Richmond | United States | Recruiting |
| Pinnacle Clinical Research - San Antonio Coordinator of this site? Confirm its status | San Antonio | United States | Recruiting |
| American Research Corporation at the Texas Liver Institute Coordinator of this site? Confirm its status | San Antonio | United States | Recruiting |
| Precision Research Institute - San Diego Coordinator of this site? Confirm its status | San Diego | United States | Terminated |
| R & H Clinical Research Coordinator of this site? Confirm its status | Stafford | United States | Terminated |
| Washington University School of Medicine Coordinator of this site? Confirm its status | St Louis | United States | Recruiting |
| The Institute for Liver Health II LLC dba Arizona Liver Health Coordinator of this site? Confirm its status | Tucson | United States | Recruiting |
| Arizona Liver Health - Tucson Coordinator of this site? Confirm its status | Tucson | United States | Recruiting |
| Del Sol Research Management, LLC Coordinator of this site? Confirm its status | Tucson | United States | Recruiting |
| Adobe Clinical Research Coordinator of this site? Confirm its status | Tucson | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.