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
Recruiting · NCT05519475 · Interventional (participants receive a specific treatment) · Lead sponsor: Regeneron Pharmaceuticals
View the official record on ClinicalTrials.gov →Interventions studied
ALN-HSDPlacebo
What this trial is about
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
Who can take part
Age range
18 Years to 75 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 2
Study type
Interventional (participants receive a specific treatment)
Inclusion criteria
- 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
- Key
Exclusion criteria
- 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
- 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
Where it is running
60 locations listed across 14 US states.
- Arizona Liver Health - Chandler, Arizona, United States
- The Institute for Liver Health II LLC DBA Arizona Clinical Trials - Flagstaff - Flagstaff, Arizona, United States
- The Institute for Liver Health II LLC DBA Arizona Liver Health - Peoria - Peoria, Arizona, United States
- Adobe Clinical Research - Tucson, Arizona, United States
- Arizona Liver Health - Tucson - Tucson, Arizona, United States
- Del Sol Research Management, LLC - Tucson, Arizona, United States
- San Fernando Valley Health Institute - Canoga Park, California, United States
- Velocity Clinical Research - Chula Vista, California, United States
- Southern California Research Center - Coronado, California, United States
- Ark Clinical Research - Fountain Valley - Fountain Valley, California, United States
- Velocity Clinical Research - Gardena, California, United States
- Velocity Clinical Research - Huntington Park - Huntington Park, California, United States
- Om Research Llc - Lancaster, California, United States
- Ark Clinical Research - Long Beach - Long Beach, California, United States
- Velocity Clinical Research - Westlake - Los Angeles, California, United States
- Clinnova Research Solutions - Orange, California, United States
- Knowledge Research Center, St. Joseph's Medical Tower - Orange, California, United States
- National Research Institute - Panorama City, California, United States
- California Liver Research Institute - Pasadena, California, United States
- Cadena Care Inst. - Poway, California, United States
- Inland Empire Liver Foundation - Rialto, California, United States
- Precision Research Institute - San Diego - San Diego, California, United States
- University of Colorado Anschutz Medical Campus - Aurora, Colorado, United States
- Synergy Healthcare LLC - Bradenton, Florida, United States
- Evolution Clinical Trials - Hialeah Gardens, Florida, United States
- Health Awareness, inc. - Jupiter, Florida, United States
- Miami Clinical Research - Miami, Florida, United States
- International Medical Investigational Centers (IMIC) Incorporated - Miami, Florida, United States
- Genoma Research Group, Inc. - Miami, Florida, United States
- US Associates in Research, LLC - Miami, Florida, United States
- Med Research of Florida, LLC - Miami, Florida, United States
- Charter Research LLC - Orlando - Orlando, Florida, United States
- Indiana University School of Medicine - Indianapolis, Indiana, United States
- Deaconess Clinic - GI Specialty Center - Newburgh, Indiana, United States
- Tandem Clinical Research - Metairie, Louisiana, United States
- Mercy Medical Center - Baltimore, Maryland, United States
- GI Associates - Flowood, Mississippi, United States
- Washington University School of Medicine - St Louis, Missouri, United States
- Columbia University Irving Medical Center - New York, New York, United States
- Geisinger Medical Center - Danville, Pennsylvania, United States
- Penn State Health Milton S. Hershey Medical Center - Hershey, Pennsylvania, United States
- University of Pennsylvania - Perelman Center for Advanced Medicine - Philadelphia, Pennsylvania, United States
- Austin Clinical Research, LLC - Austin, Texas, United States
- American Research Corporation - Austin, Texas, United States
- Pinnacle Clinical Research - Austin - Austin, Texas, United States
- South Texas Research Institute (STRI) - Brownsville - Brownsville, Texas, United States
- Velocity Clinical Research - Austin - Cedar Park, Texas, United States
- Methodist Dallas Medical Center - Dallas, Texas, United States
- South Texas Research Institute (STRI) - Edinburg - Edinburg, Texas, United States
- GI Alliance Research - Fort Worth, Texas, United States
- Allure Health at Mt. Olympus Medical Research - Friendswood, Texas, United States
- Advanced Medical Trials - Georgetown, Texas, United States
- Pioneer Research Solutions, Inc. - Houston, Texas, United States
- R & H Clinical Research, Inc. - Katy, Texas, United States
- Great Lakes Research Institute - Pharr, Texas, United States
- American Research Corporation at The Texas Liver Institute - San Antonio, Texas, United States
- Pinnacle Clinical Research - San Antonio - San Antonio, Texas, United States
- R & H Clinical Research - Stafford, Texas, United States
- Intermountain Health - Murray, Utah, United States
- University of Virginia Medical Center - Charlottesville, Virginia, United States
Showing 60 of 71 listed sites. See the official record for the complete list.
Read the full protocol, contacts and eligibility on ClinicalTrials.gov →