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

Description

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

Conditions

Metabolic Dysfunction-associated Steatohepatitis (MASH)

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Study Overview

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Study Details

Study overview

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

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

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

Condition
Metabolic Dysfunction-associated Steatohepatitis (MASH)
Intervention / Treatment

-

Contacts and Locations

Chandler

Arizona Liver Health, Chandler, Arizona, United States, 85224

Flagstaff

The Institute for Liver Health II LLC DBA Arizona Clinical Trials - Flagstaff, Flagstaff, Arizona, United States, 86001

Peoria

The Institute for Liver Health II LLC DBA Arizona Liver Health - Peoria, Peoria, Arizona, United States, 85381

Tucson

Adobe Clinical Research, Tucson, Arizona, United States, 85712

Tucson

Arizona Liver Health - Tucson, Tucson, Arizona, United States, 85712

Tucson

Del Sol Research Management, LLC, Tucson, Arizona, United States, 85715

Canoga Park

San Fernando Valley Health Institute, Canoga Park, California, United States, 91304

Chula Vista

Velocity Clinical Research, Chula Vista, California, United States, 91911

Coronado

Southern California Research Center, Coronado, California, United States, 92118

Fountain Valley

Ark Clinical Research - Fountain Valley, Fountain Valley, California, United States, 92708

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • 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)

Ages Eligible for Study

18 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Regeneron Pharmaceuticals,

Clinical Trial Management, STUDY_DIRECTOR, Regeneron Pharmaceuticals

Study Record Dates

2027-09-08