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

Last updated: February 2, 2026
Sponsor: Regeneron Pharmaceuticals
Overall Status: Active - Recruiting

Phase

2

Condition

Primary Biliary Cholangitis

Liver Disease

Treatment

ALN-HSD

Placebo

Clinical Study ID

NCT05519475
ALN-HSD-NASH-2130
  • Ages 18-75
  • All Genders

Study 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

Eligibility Criteria

Inclusion

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-ClinicalResearch Network (CRN)

  3. NAS score ≥3, as defined in the protocol

  4. Meets genotype criteria for study enrollment, as defined in the protocol

  5. Has protocol defined FibroScan®-AST (FAST) score at screening or withinapproximately 12 weeks of screening

Exclusion

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 anytime 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 studyperiod

  5. Prior exposure to any investigational drug targeting HSD17B13 or patatin-likephospholipase domain containing 3 (PNPLA3) (eg, ALN-HSD, ARO-HSD, ALN-PNP, AZD2693)

Note: Other protocol-defined Inclusion/Exclusion Criteria apply

Study Design

Total Participants: 90
Treatment Group(s): 2
Primary Treatment: ALN-HSD
Phase: 2
Study Start date:
February 09, 2023
Estimated Completion Date:
September 08, 2027

Connect with a study center

  • JCHO Hokkaido Hospital

    Sapporo, Hokkaido 062-8618
    Japan

    Site Not Available

  • JCHO Hokkaido Hospital

    Sapporo 2128295, Hokkaido 2130037 062-8618
    Japan

    Terminated

  • JCHO Hokkaido Hospital

    Hokkaido, Nakanoshima 1-jo, Toyohira-ku, Sapporo-shi 062 8618
    Japan

    Site Not Available

  • Seoul National University Bundang Hospital

    Seongnam-si, Gyeonggi-do 13620
    Korea, Republic of

    Site Not Available

  • Seoul National University Bundang Hospital (SNUBH)

    Seongnam-si, Gyeonggi-do 13620
    Korea, Republic of

    Active - Recruiting

  • Pusan National University Hospital

    Busan, 49241
    Korea, Republic of

    Site Not Available

  • Keimyung University Dongsan Hospital

    Daegu, 42601
    Korea, Republic of

    Terminated

  • Keimyung University Dongsan Medical Center

    Daegu, 42601
    Korea, Republic of

    Active - Recruiting

  • Kyungpook National University Hospital

    Daegu, 41944
    Korea, Republic of

    Site Not Available

  • Hanyang University Seoul Hospital

    Seoul, 04763
    Korea, Republic of

    Site Not Available

  • Isis Clinical Research Center

    Guaynabo, San Juan 4568138 00969
    Puerto Rico

    Terminated

  • FDI Clinical Research

    San Juan, 00927
    Puerto Rico

    Active - Recruiting

  • Fundacion de Investigacion (FDI) Clinical Research

    San Juan, 00927
    Puerto Rico

    Site Not Available

  • ISIS Clinical Research Center

    San Juan, 00969
    Puerto Rico

    Active - Recruiting

  • Klinical Investigations

    San Juan, 00909
    Puerto Rico

    Suspended

  • Latin Clinical Trial Center

    San Juan, 00909
    Puerto Rico

    Suspended

  • Fundacion de Investigacion (FDI) Clinical Research

    San Juan 4568127, 00927
    Puerto Rico

    Active - Recruiting

  • Klinical Investigations

    San Juan 4568127, 00909
    Puerto Rico

    Terminated

  • Latin Clinical Trial Center

    San Juan 4568127, 00909
    Puerto Rico

    Terminated

  • Seoul National University Bundang Hospital

    Seongnam-si 1897000, Gyeonggi-do 1841610 13620
    South Korea

    Terminated

  • Pusan National University Hospital

    Busan 1838524, 49241
    South Korea

    Terminated

  • Keimyung University Dongsan Hospital

    Daegu 1835329, 42601
    South Korea

    Terminated

  • Kyungpook National University Hospital

    Daegu 1835329, 41944
    South Korea

    Terminated

  • Hanyang University Seoul Hospital

    Seoul 1835848, 04763
    South Korea

    Terminated

  • Arizona Liver Health

    Chandler, Arizona 85224
    United States

    Site Not Available

  • The Institute for Liver Health II LLC dba Arizona Liver Health

    Chandler, Arizona 85224
    United States

    Site Not Available

  • The Institute for Liver Health II LLC DBA Arizona Clinical Trials - Flagstaff

    Flagstaff, Arizona 86001
    United States

    Site Not Available

  • The Institute for Liver Health II LLC DBA Arizona Liver Health - Peoria

    Peoria, Arizona 85381
    United States

    Site Not Available

  • Adobe Clinical Research

    Tucson, Arizona 85712
    United States

    Active - Recruiting

  • Arizona Liver Health - Tucson

    Tucson, Arizona 85712
    United States

    Site Not Available

  • Arizona Liver Health - Tuscon

    Tucson, Arizona 85712
    United States

    Active - Recruiting

  • Del Sol Research Management, LLC

    Tucson, Arizona 85715
    United States

    Active - Recruiting

  • Arizona Liver Health

    Chandler 5289282, Arizona 5551752 85224
    United States

    Active - Recruiting

  • The Institute for Liver Health II LLC DBA Arizona Clinical Trials - Flagstaff

    Flagstaff 5294810, Arizona 5551752 86001
    United States

    Terminated

  • The Institute for Liver Health II LLC DBA Arizona Liver Health - Peoria

    Peoria 5308480, Arizona 5551752 85381
    United States

    Active - Recruiting

  • Adobe Clinical Research

    Tucson 5318313, Arizona 5551752 85712
    United States

    Active - Recruiting

  • Arizona Liver Health - Tucson

    Tucson 5318313, Arizona 5551752 85712
    United States

    Active - Recruiting

  • Del Sol Research Management, LLC

    Tucson 5318313, Arizona 5551752 85715
    United States

    Active - Recruiting

  • San Fernando Valley Health Institute

    Canoga Park, California 91304
    United States

    Site Not Available

  • Velocity Clinical Research

    Chula Vista, California 91911
    United States

    Site Not Available

  • Southern California Research Center

    Coronado, California 92118
    United States

    Site Not Available

  • Ark Clinical Research - Fountain Valley

    Fountain Valley, California 92708
    United States

    Site Not Available

  • Velocity Clinical Research

    Gardena, California 90247
    United States

    Site Not Available

  • Velocity Clinical Research - Huntington Park

    Huntington Park, California 90255
    United States

    Site Not Available

  • Om Research Llc

    Lancaster, California 93534
    United States

    Site Not Available

  • Ark Clinical Research - Long Beach

    Long Beach, California 90815
    United States

    Site Not Available

  • National Research Institute

    Los Angeles, California 90057
    United States

    Active - Recruiting

  • Velocity Clinical Research - Westlake

    Los Angeles, California 90057
    United States

    Site Not Available

  • Clinnova Research Solutions

    Orange, California 92868
    United States

    Active - Recruiting

  • Knowledge Research Center, St. Joseph's Medical Tower

    Orange, California 92868
    United States

    Site Not Available

  • National Research Institute

    Panorama City, California 91402
    United States

    Site Not Available

  • California Liver Research Institute

    Pasadena, California 91105
    United States

    Site Not Available

  • Cadena Care Inst.

    Poway, California 92064
    United States

    Site Not Available

  • Inland Empire Liver Foundation

    Rialto, California 92377
    United States

    Site Not Available

  • Precision Research Institute - San Diego

    San Diego, California 92114
    United States

    Site Not Available

  • Precision Research Institute, Llc

    San Diego, California 92114
    United States

    Active - Recruiting

  • San Fernando Valley Health Institute

    Canoga Park 5333913, California 5332921 91304
    United States

    Terminated

  • Velocity Clinical Research

    Chula Vista 5336899, California 5332921 91911
    United States

    Terminated

  • Southern California Research Center

    Coronado 5339663, California 5332921 92118
    United States

    Active - Recruiting

  • Ark Clinical Research - Fountain Valley

    Fountain Valley 5350207, California 5332921 92708
    United States

    Active - Recruiting

  • Velocity Clinical Research

    Gardena 5351549, California 5332921 90247
    United States

    Terminated

  • Velocity Clinical Research - Huntington Park

    Huntington Park 5358736, California 5332921 90255
    United States

    Terminated

  • Om Research Llc

    Lancaster 5364940, California 5332921 93534
    United States

    Active - Recruiting

  • Ark Clinical Research - Long Beach

    Long Beach 5367929, California 5332921 90815
    United States

    Active - Recruiting

  • Velocity Clinical Research - Westlake

    Los Angeles 5368361, California 5332921 90057
    United States

    Terminated

  • Clinnova Research Solutions

    Orange 5379513, California 5332921 92868
    United States

    Active - Recruiting

  • Knowledge Research Center, St. Joseph's Medical Tower

    Orange 5379513, California 5332921 92868
    United States

    Active - Recruiting

  • National Research Institute

    Panorama City 5380932, California 5332921 91402
    United States

    Terminated

  • California Liver Research Institute

    Pasadena 5381396, California 5332921 91105
    United States

    Active - Recruiting

  • Cadena Care Inst.

    Poway 5384690, California 5332921 92064
    United States

    Terminated

  • Inland Empire Liver Foundation

    Rialto 5387288, California 5332921 92377
    United States

    Active - Recruiting

  • Precision Research Institute - San Diego

    San Diego 5391811, California 5332921 92114
    United States

    Terminated

  • University of Colorado Anschutz Medical Campus

    Aurora, Colorado 80045
    United States

    Site Not Available

  • University of Colorado Anschutz Medical Campus

    Aurora 5412347, Colorado 5417618 80045
    United States

    Active - Recruiting

  • Synergy Healthcare, LLC

    Bradenton, Florida 34208
    United States

    Site Not Available

  • Evolution Clinical Trials

    Hialeah Gardens, Florida 33016
    United States

    Site Not Available

  • Health Awareness, inc.

    Jupiter, Florida 33458
    United States

    Site Not Available

  • Genoma Research Group, Inc.

    Miami, Florida 33173
    United States

    Active - Recruiting

  • IMIC, Inc

    Miami, Florida 33157
    United States

    Terminated

  • Med Research Of Florida, LLC

    Miami, Florida 33186
    United States

    Active - Recruiting

  • Med Research of Florida, LLC

    Miami, Florida 33186
    United States

    Active - Recruiting

  • Miami Clinical Research Center

    Miami, Florida 33155
    United States

    Site Not Available

  • US Associates in Research, LLC

    Miami, Florida 33175
    United States

    Suspended

  • Charter Research LLC - Orlando

    Orlando, Florida 32803
    United States

    Site Not Available

  • Synergy Healthcare LLC

    Bradenton 4148708, Florida 4155751 34209
    United States

    Terminated

  • Synergy Healthcare, LLC

    Bradenton 4148708, Florida 4155751 34208
    United States

    Terminated

  • Evolution Clinical Trials

    Hialeah Gardens 4158482, Florida 4155751 33016
    United States

    Active - Recruiting

  • Health Awareness, inc.

    Jupiter 4160610, Florida 4155751 33458
    United States

    Active - Recruiting

  • Genoma Research Group, Inc.

    Miami 4164138, Florida 4155751 33173
    United States

    Active - Recruiting

  • IMIC, Inc

    Miami 4164138, Florida 4155751 33157
    United States

    Terminated

  • International Medical Investigational Centers (IMIC) Incorporated

    Miami 4164138, Florida 4155751 33157
    United States

    Terminated

  • Med Research of Florida, LLC

    Miami 4164138, Florida 4155751 33186
    United States

    Active - Recruiting

  • Miami Clinical Research

    Miami 4164138, Florida 4155751 33155
    United States

    Terminated

  • Miami Clinical Research Center

    Miami 4164138, Florida 4155751 33155
    United States

    Terminated

  • US Associates in Research, LLC

    Miami 4164138, Florida 4155751 33175
    United States

    Terminated

  • Charter Research LLC - Orlando

    Orlando 4167147, Florida 4155751 32803
    United States

    Active - Recruiting

  • Indiana University School of Medicine

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • Deaconess Clinic - GI Specialty Center

    Newburgh, Indiana 47630
    United States

    Site Not Available

  • Indiana University School of Medicine

    Indianapolis 4259418, Indiana 4921868 46202
    United States

    Active - Recruiting

  • Deaconess Clinic - GI Specialty Center

    Newburgh 4262171, Indiana 4921868 47630
    United States

    Active - Recruiting

  • Legacy Clinical Solutions: Tandem Clinical Research, LLC

    Marrero, Louisiana 70072
    United States

    Active - Recruiting

  • Tandem Clinical Research

    Marrero, Louisiana 70072
    United States

    Site Not Available

  • Tandem Clinical Research

    Metairie, Louisiana 70006
    United States

    Site Not Available

  • Tandem Clinical Research

    Metairie 4333177, Louisiana 4331987 70006
    United States

    Active - Recruiting

  • Mercy Medical Center

    Baltimore, Maryland 21202
    United States

    Site Not Available

  • Mercy Medical Center, Inc

    Baltimore, Maryland 21202-2102
    United States

    Site Not Available

  • Mercy Medical Center

    Baltimore 4347778, Maryland 4361885 21202
    United States

    Terminated

  • GI Associates

    Flowood, Mississippi 39232
    United States

    Site Not Available

  • GI Associates

    Flowood 4426822, Mississippi 4436296 39232
    United States

    Active - Recruiting

  • Washington University School of Medicine

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Washington University School of Medicine

    St Louis 4407066, Missouri 4398678 63110
    United States

    Active - Recruiting

  • Columbia University Irving Medical Center

    New York, New York 10032
    United States

    Site Not Available

  • Columbia University Irving Medical Center

    New York 5128581, New York 5128638 10032
    United States

    Active - Recruiting

  • Geisinger Medical Center

    Danville, Pennsylvania 17822
    United States

    Site Not Available

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania 17033
    United States

    Site Not Available

  • University of Pennsylvania - Perelman Center for Advanced Medicine

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • University of Pennsylvania - Perelman School of Medicine - Gene Therapy Program

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Geisinger Medical Center

    Danville 5186327, Pennsylvania 6254927 17822
    United States

    Active - Recruiting

  • Penn State Health Milton S. Hershey Medical Center

    Hershey 5193342, Pennsylvania 6254927 17033
    United States

    Active - Recruiting

  • University of Pennsylvania - Perelman Center for Advanced Medicine

    Philadelphia 4560349, Pennsylvania 6254927 19104
    United States

    Active - Recruiting

  • American Research Corporation

    Austin, Texas 78757
    United States

    Active - Recruiting

  • IMA Clinical Research Austin

    Austin, Texas 78745
    United States

    Active - Recruiting

  • Pinnacle Clinical Research - Austin

    Austin, Texas 78757
    United States

    Site Not Available

  • South Texas Research Institute (STRI) - Brownsville

    Brownsville, Texas 78520
    United States

    Site Not Available

  • Velocity Clinical Research

    Cedar Park, Texas 78613
    United States

    Active - Recruiting

  • Velocity Clinical Research - Austin

    Cedar Park, Texas 78613
    United States

    Site Not Available

  • Methodist Dallas Medical Center

    Dallas, Texas 75203
    United States

    Site Not Available

  • South Texas Research Institute (STRI) - Edinburg

    Edinburg, Texas 78520
    United States

    Site Not Available

  • GI Alliance Research

    Fort Worth, Texas 76104
    United States

    Site Not Available

  • Allure Health

    Friendswood, Texas 77546-5700
    United States

    Active - Recruiting

  • Allure Health at Mt. Olympus Medical Research

    Friendswood, Texas 77546
    United States

    Site Not Available

  • Advanced Medical Trials

    Georgetown, Texas 78628
    United States

    Site Not Available

  • Pioneer Research Solutions Inc.

    Houston, Texas 77099
    United States

    Active - Recruiting

  • Pioneer Research Solutions, Inc.

    Houston, Texas 77099
    United States

    Site Not Available

  • R & H Clinical Research

    Katy, Texas 77494
    United States

    Active - Recruiting

  • R & H Clinical Research, Inc.

    Katy, Texas 77494
    United States

    Site Not Available

  • GLRI - McAllen Research

    Pharr, Texas 78577
    United States

    Active - Recruiting

  • Great Lakes Research Institute

    Pharr, Texas 78577
    United States

    Site Not Available

  • American Research Corporation at The Texas Liver Institute

    San Antonio, Texas 78215
    United States

    Active - Recruiting

  • Pinnacle Clinical Research - San Antonio

    San Antonio, Texas 78229
    United States

    Site Not Available

  • R & H Clinical Research

    Stafford, Texas 77477
    United States

    Site Not Available

  • American Research Corporation

    Austin 4671654, Texas 4736286 78757
    United States

    Active - Recruiting

  • Austin Clinical Research, LLC

    Austin 4671654, Texas 4736286 78745
    United States

    Terminated

  • Pinnacle Clinical Research - Austin

    Austin 4671654, Texas 4736286 78757
    United States

    Active - Recruiting

  • South Texas Research Institute (STRI) - Brownsville

    Brownsville 4676740, Texas 4736286 78520
    United States

    Active - Recruiting

  • Velocity Clinical Research - Austin

    Cedar Park 4679867, Texas 4736286 78613
    United States

    Terminated

  • Methodist Dallas Medical Center

    Dallas 4684888, Texas 4736286 75203
    United States

    Active - Recruiting

  • South Texas Research Institute (STRI) - Edinburg

    Edinburg 4688275, Texas 4736286 78539
    United States

    Active - Recruiting

  • GI Alliance Research

    Fort Worth 4691930, Texas 4736286 76104
    United States

    Active - Recruiting

  • Allure Health at Mt. Olympus Medical Research

    Friendswood 4692521, Texas 4736286 77546
    United States

    Terminated

  • Advanced Medical Trials

    Georgetown 4693342, Texas 4736286 78628
    United States

    Active - Recruiting

  • Pioneer Research Solutions, Inc.

    Houston 4699066, Texas 4736286 77099
    United States

    Active - Recruiting

  • R & H Clinical Research, Inc.

    Katy 4702732, Texas 4736286 77494
    United States

    Terminated

  • Great Lakes Research Institute

    Pharr 4718721, Texas 4736286 78577
    United States

    Terminated

  • American Research Corporation at The Texas Liver Institute

    San Antonio 4726206, Texas 4736286 78215
    United States

    Active - Recruiting

  • Pinnacle Clinical Research - San Antonio

    San Antonio 4726206, Texas 4736286 78229
    United States

    Active - Recruiting

  • R & H Clinical Research

    Stafford 4734005, Texas 4736286 77477
    United States

    Terminated

  • Intermountain Health

    Murray 5778755, Utah 5549030 84107
    United States

    Active - Recruiting

  • University of Virginia Medical Center

    Charlottesville, Virginia 22903
    United States

    Site Not Available

  • Richmond Va Medical Center

    Richmond, Virginia 23249
    United States

    Site Not Available

  • University of Virginia Medical Center

    Charlottesville 4752031, Virginia 6254928 22903
    United States

    Active - Recruiting

  • Richmond Va Medical Center

    Richmond 4781708, Virginia 6254928 23249
    United States

    Active - Recruiting

  • Richmond Veterans Administration Medical Center

    Richmond 4781708, Virginia 6254928 23249
    United States

    Active - Recruiting

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