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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

NCT ID: NCT05519475

Last Updated: 2026-01-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

90 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-09

Study Completion Date

2027-09-08

Brief Summary

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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

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Detailed Description

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Conditions

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Metabolic Dysfunction-associated Steatohepatitis (MASH)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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ALN-HSD

Randomized 1:1

Group Type EXPERIMENTAL

ALN-HSD

Intervention Type DRUG

Administered by subcutaneous injection (SC)

Placebo

Randomized 1:1

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Administered by SC injection

Interventions

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ALN-HSD

Administered by subcutaneous injection (SC)

Intervention Type DRUG

Placebo

Administered by SC injection

Intervention Type DRUG

Eligibility Criteria

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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-Clinical Research 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 within approximately 12 weeks of screening

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)
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trial Management

Role: STUDY_DIRECTOR

Regeneron Pharmaceuticals

Locations

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Arizona Liver Health

Chandler, Arizona, United States

Site Status RECRUITING

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

Flagstaff, Arizona, United States

Site Status TERMINATED

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

Peoria, Arizona, United States

Site Status RECRUITING

Adobe Clinical Research

Tucson, Arizona, United States

Site Status RECRUITING

Arizona Liver Health - Tucson

Tucson, Arizona, United States

Site Status RECRUITING

Del Sol Research Management, LLC

Tucson, Arizona, United States

Site Status RECRUITING

San Fernando Valley Health Institute

Canoga Park, California, United States

Site Status TERMINATED

Velocity Clinical Research

Chula Vista, California, United States

Site Status TERMINATED

Southern California Research Center

Coronado, California, United States

Site Status RECRUITING

Ark Clinical Research - Fountain Valley

Fountain Valley, California, United States

Site Status RECRUITING

Velocity Clinical Research

Gardena, California, United States

Site Status TERMINATED

Velocity Clinical Research - Huntington Park

Huntington Park, California, United States

Site Status TERMINATED

Om Research Llc

Lancaster, California, United States

Site Status RECRUITING

Ark Clinical Research - Long Beach

Long Beach, California, United States

Site Status RECRUITING

Velocity Clinical Research - Westlake

Los Angeles, California, United States

Site Status TERMINATED

Clinnova Research Solutions

Orange, California, United States

Site Status RECRUITING

Knowledge Research Center, St. Joseph's Medical Tower

Orange, California, United States

Site Status RECRUITING

National Research Institute

Panorama City, California, United States

Site Status TERMINATED

California Liver Research Institute

Pasadena, California, United States

Site Status RECRUITING

Cadena Care Inst.

Poway, California, United States

Site Status TERMINATED

Inland Empire Liver Foundation

Rialto, California, United States

Site Status RECRUITING

Precision Research Institute - San Diego

San Diego, California, United States

Site Status TERMINATED

University of Colorado Anschutz Medical Campus

Aurora, Colorado, United States

Site Status RECRUITING

Synergy Healthcare, LLC

Bradenton, Florida, United States

Site Status TERMINATED

Evolution Clinical Trials

Hialeah Gardens, Florida, United States

Site Status RECRUITING

Health Awareness, inc.

Jupiter, Florida, United States

Site Status RECRUITING

Miami Clinical Research

Miami, Florida, United States

Site Status TERMINATED

International Medical Investigational Centers (IMIC) Incorporated

Miami, Florida, United States

Site Status TERMINATED

Genoma Research Group, Inc.

Miami, Florida, United States

Site Status RECRUITING

US Associates in Research, LLC

Miami, Florida, United States

Site Status TERMINATED

Med Research of Florida, LLC

Miami, Florida, United States

Site Status RECRUITING

Charter Research LLC - Orlando

Orlando, Florida, United States

Site Status RECRUITING

Indiana University School of Medicine

Indianapolis, Indiana, United States

Site Status RECRUITING

Deaconess Clinic - GI Specialty Center

Newburgh, Indiana, United States

Site Status RECRUITING

Tandem Clinical Research

Metairie, Louisiana, United States

Site Status RECRUITING

Mercy Medical Center

Baltimore, Maryland, United States

Site Status TERMINATED

GI Associates

Flowood, Mississippi, United States

Site Status RECRUITING

Washington University School of Medicine

St Louis, Missouri, United States

Site Status RECRUITING

Columbia University Irving Medical Center

New York, New York, United States

Site Status RECRUITING

Geisinger Medical Center

Danville, Pennsylvania, United States

Site Status RECRUITING

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status RECRUITING

University of Pennsylvania - Perelman Center for Advanced Medicine

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Austin Clinical Research, LLC

Austin, Texas, United States

Site Status TERMINATED

American Research Corporation

Austin, Texas, United States

Site Status RECRUITING

Pinnacle Clinical Research - Austin

Austin, Texas, United States

Site Status RECRUITING

South Texas Research Institute (STRI) - Brownsville

Brownsville, Texas, United States

Site Status RECRUITING

Velocity Clinical Research - Austin

Cedar Park, Texas, United States

Site Status TERMINATED

Methodist Dallas Medical Center

Dallas, Texas, United States

Site Status RECRUITING

South Texas Research Institute (STRI) - Edinburg

Edinburg, Texas, United States

Site Status RECRUITING

GI Alliance Research

Fort Worth, Texas, United States

Site Status RECRUITING

Allure Health at Mt. Olympus Medical Research

Friendswood, Texas, United States

Site Status TERMINATED

Advanced Medical Trials

Georgetown, Texas, United States

Site Status RECRUITING

Pioneer Research Solutions, Inc.

Houston, Texas, United States

Site Status RECRUITING

R & H Clinical Research, Inc.

Katy, Texas, United States

Site Status TERMINATED

Great Lakes Research Institute

Pharr, Texas, United States

Site Status TERMINATED

American Research Corporation at The Texas Liver Institute

San Antonio, Texas, United States

Site Status RECRUITING

Pinnacle Clinical Research - San Antonio

San Antonio, Texas, United States

Site Status RECRUITING

R & H Clinical Research

Stafford, Texas, United States

Site Status TERMINATED

Intermountain Health

Murray, Utah, United States

Site Status RECRUITING

University of Virginia Medical Center

Charlottesville, Virginia, United States

Site Status RECRUITING

Richmond Va Medical Center

Richmond, Virginia, United States

Site Status RECRUITING

JCHO Hokkaido Hospital

Sapporo, Hokkaido, Japan

Site Status TERMINATED

Isis Clinical Research Center

Guaynabo, San Juan, Puerto Rico

Site Status TERMINATED

Klinical Investigations

San Juan, , Puerto Rico

Site Status TERMINATED

Latin Clinical Trial Center

San Juan, , Puerto Rico

Site Status TERMINATED

Fundacion de Investigacion (FDI) Clinical Research

San Juan, , Puerto Rico

Site Status RECRUITING

Seoul National University Bundang Hospital

Seongnam-si, Gyeonggi-do, South Korea

Site Status RECRUITING

Pusan National University Hospital

Busan, , South Korea

Site Status RECRUITING

Kyungpook National University Hospital

Daegu, , South Korea

Site Status TERMINATED

Keimyung University Dongsan Hospital

Daegu, , South Korea

Site Status TERMINATED

Hanyang University Seoul Hospital

Seoul, , South Korea

Site Status RECRUITING

Countries

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United States Japan Puerto Rico South Korea

Central Contacts

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Clinical Trials Administrator

Role: CONTACT

[email protected]
844-734-6643

Related Links

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https://nashgenstudy.com/en-us

Study Informational Website

Other Identifiers

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ALN-HSD-NASH-2130

Identifier Type: -

Identifier Source: org_study_id

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