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

Launched by REGENERON PHARMACEUTICALS · Aug 26, 2022

Keywords

ClinConnect Summary

This clinical trial is exploring a new investigational drug called ALN-HSD to treat a liver condition known as non-alcoholic steatohepatitis (NASH), which is a serious form of fatty liver disease. NASH can lead to liver damage, inflammation, and potentially more severe issues like cirrhosis or liver failure. The study aims to see if ALN-HSD can reduce liver scarring and improve liver function in adults who are at increased genetic risk for this condition. Researchers will also look at how the drug works, possible side effects, and how it is processed in the body.

To be eligible for this trial, participants must be adults aged 18 or older with a diagnosis of NASH that shows certain levels of liver scarring (fibrosis) and inflammation. However, individuals with other types of chronic liver disease, significant alcohol use, or certain medical histories are not eligible. If you choose to participate, you will receive the study drug and have regular check-ups to monitor your health and any effects from the treatment. This research is important because it could lead to better ways to manage NASH and improve the lives of those affected by this condition.

Gender

ALL

Eligibility criteria

• Key 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
• Key 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)
• • Note: Other protocol-defined Inclusion/Exclusion Criteria apply