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
Purpose
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
Condition
- Metabolic Dysfunction-Associated SteatoHepatitis (MASH)
Eligibility
- Eligible Ages
- Between 18 Years and 75 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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-CRN 3. NAS score ≥3, as defined in the protocol 4. Meets genotype criteria for study enrollment, as defined in the protocol 5. Has a protocol defined FibroScan®-AST (FAST) score at screening or within approximately 12 weeks of screening
Exclusion Criteria
- 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) Note: Other protocol-defined Inclusion/Exclusion Criteria apply
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental ALN-HSD |
Randomized 1:1 |
|
|
Placebo Comparator Placebo |
Randomized 1:1 |
|
Recruiting Locations
Chandler, Arizona 85224
Peoria, Arizona 85381
Tucson, Arizona 85712
Tucson, Arizona 85712
Tucson, Arizona 85715
Coronado, California 92118
Fountain Valley, California 92708
Lancaster, California 93534
Long Beach, California 90815
Orange, California 92868
Orange, California 92868
Pasadena, California 91105
Rialto, California 92377
Aurora, Colorado 80045
Hialeah Gardens, Florida 33016
Jupiter, Florida 33458
Miami, Florida 33173
Miami, Florida 33186
Orlando, Florida 32803
Indianapolis, Indiana 46202
Metairie, Louisiana 70006
Flowood, Mississippi 39232
St Louis, Missouri 63110
New York, New York 10032
Danville, Pennsylvania 17822
Hershey, Pennsylvania 17033
Philadelphia, Pennsylvania 19104
Austin, Texas 78757
Austin, Texas 78757
Dallas, Texas 75203
Fort Worth, Texas 76104
Georgetown, Texas 78628
Houston, Texas 77099
San Antonio, Texas 78215
San Antonio, Texas 78229
Charlottesville, Virginia 22903
Richmond, Virginia 23249
San Juan, Puerto Rico 00927
More Details
- Status
- Recruiting
- Sponsor
- Regeneron Pharmaceuticals