RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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

Official Title

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

Quick Facts

Study Start:2023-02-09
Study Completion:2027-09-08
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05519475

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.

Ages Eligible for Study:18 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Adult male or female ≥18 years (or country's legal age of adulthood)
  2. 2. A diagnosis of MASH with fibrosis (F) stage 2 or 3, according to the NASH-Clinical Research Network (CRN)
  3. 3. NAS score ≥3, as defined in the protocol
  4. 4. Meets genotype criteria for study enrollment, as defined in the protocol
  5. 5. Has protocol defined FibroScan®-AST (FAST) score at screening or within approximately 12 weeks of screening
  1. 1. Evidence of other forms of known chronic liver disease, as defined in the protocol
  2. 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. 3. History of Type 1 diabetes
  4. 4. Bariatric surgery within approximately 5 years prior to or planned during the study period
  5. 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)

Contacts and Locations

Study Contact

Clinical Trials Administrator
CONTACT
844-734-6643
clinicaltrials@regeneron.com

Principal Investigator

Clinical Trial Management
STUDY_DIRECTOR
Regeneron Pharmaceuticals

Study Locations (Sites)

Arizona Liver Health
Chandler, Arizona, 85224
United States
The Institute for Liver Health II LLC DBA Arizona Clinical Trials - Flagstaff
Flagstaff, Arizona, 86001
United States
The Institute for Liver Health II LLC DBA Arizona Liver Health - Peoria
Peoria, Arizona, 85381
United States
Adobe Clinical Research
Tucson, Arizona, 85712
United States
Arizona Liver Health - Tucson
Tucson, Arizona, 85712
United States
Del Sol Research Management, LLC
Tucson, Arizona, 85715
United States
San Fernando Valley Health Institute
Canoga Park, California, 91304
United States
Velocity Clinical Research
Chula Vista, California, 91911
United States
Southern California Research Center
Coronado, California, 92118
United States
Ark Clinical Research - Fountain Valley
Fountain Valley, California, 92708
United States
Velocity Clinical Research
Gardena, California, 90247
United States
Velocity Clinical Research - Huntington Park
Huntington Park, California, 90255
United States
Ark Clinical Research - Long Beach
Long Beach, California, 90815
United States
Velocity Clinical Research - Westlake
Los Angeles, California, 90057
United States
Clinnova Research Solutions
Orange, California, 92868
United States
National Research Institute
Panorama City, California, 91402
United States
California Liver Research Institute
Pasadena, California, 91105
United States
Cadena Care Inst.
Poway, California, 92064
United States
Inland Empire Liver Foundation
Rialto, California, 92377
United States
Precision Research Institute - San Diego
San Diego, California, 92114
United States
University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
United States
Synergy Healthcare, LLC
Bradenton, Florida, 34208
United States
Evolution Clinical Trials
Hialeah Gardens, Florida, 33016
United States
Miami Clinical Research Center
Miami, Florida, 33155
United States
IMIC, Inc
Miami, Florida, 33157
United States
Genoma Research Group, Inc.
Miami, Florida, 33173
United States
US Associates in Research, LLC
Miami, Florida, 33175
United States
Med Research of Florida, LLC
Miami, Florida, 33186
United States
Indiana University School of Medicine
Indianapolis, Indiana, 46202
United States
Tandem Clinical Research
Metairie, Louisiana, 70006
United States
Mercy Medical Center
Baltimore, Maryland, 21202
United States
GI Associates
Flowood, Mississippi, 39232
United States
Washington University School of Medicine
Saint Louis, Missouri, 63110
United States
Columbia University Irving Medical Center
New York, New York, 10032
United States
Geisinger Medical Center
Danville, Pennsylvania, 17822
United States
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
United States
University of Pennsylvania - Perelman Center for Advanced Medicine
Philadelphia, Pennsylvania, 19104
United States
IMA Clinical Research Austin
Austin, Texas, 78745
United States
American Research Corporation
Austin, Texas, 78757
United States
Pinnacle Clinical Research - Austin
Austin, Texas, 78757
United States
South Texas Research Institute (STRI) - Brownsville
Brownsville, Texas, 78520
United States
Velocity Clinical Research - Austin
Cedar Park, Texas, 78613
United States
South Texas Research Institute (STRI) - Edinburg
Edinburg, Texas, 78520
United States
GI Alliance Research
Fort Worth, Texas, 76104
United States
Allure Health at Mt. Olympus Medical Research
Friendswood, Texas, 77546
United States
Advanced Medical Trials
Georgetown, Texas, 78628
United States
Pioneer Research Solutions, Inc.
Houston, Texas, 77099
United States
R & H Clinical Research, Inc.
Katy, Texas, 77494
United States
Great Lakes Research Institute
Pharr, Texas, 78577
United States
American Research Corporation at The Texas Liver Institute
San Antonio, Texas, 78215
United States
Pinnacle Clinical Research - San Antonio
San Antonio, Texas, 78229
United States
R & H Clinical Research
Stafford, Texas, 77477
United States
Richmond Va Medical Center
Richmond, Virginia, 23249
United States

Collaborators and Investigators

Sponsor: Regeneron Pharmaceuticals

  • Clinical Trial Management, STUDY_DIRECTOR, Regeneron Pharmaceuticals

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-02-09
Study Completion Date2027-09-08

Study Record Updates

Study Start Date2023-02-09
Study Completion Date2027-09-08

Terms related to this study

Keywords Provided by Researchers

  • Metabolic dysfunction-associated steatotic liver disease (MASLD)
  • Histologically confirmed MASH
  • Genetic risk alleles for MASH at the HSD17B13 locus
  • Non-alcoholic steatohepatitis (NASH)

Additional Relevant MeSH Terms

  • Metabolic Dysfunction-associated Steatohepatitis (MASH)