NCT05519475 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
| NCT ID | NCT05519475 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Regeneron Pharmaceuticals |
| Condition | Metabolic Dysfunction-Associated SteatoHepatitis (MASH) |
| Study Type | INTERVENTIONAL |
| Enrollment | 120 participants |
| Start Date | 2023-02-09 |
| Primary Completion | 2028-04-27 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 120 participants in total. It began in 2023-02-09 with a primary completion date of 2028-04-27.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
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-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 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
Contact & Investigator
Clinical Trial Management
STUDY DIRECTOR
Regeneron Pharmaceuticals
Frequently Asked Questions
Who can join the NCT05519475 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 75 Years, studying Metabolic Dysfunction-Associated SteatoHepatitis (MASH). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05519475 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT05519475 currently recruiting?
Yes, NCT05519475 is actively recruiting participants. Contact the research team at clinicaltrials@regeneron.com for enrollment information.
Where is the NCT05519475 trial being conducted?
This trial is being conducted at Chandler, United States, Flagstaff, United States, Peoria, United States, Tucson, United States and 11 additional locations.
Who is sponsoring the NCT05519475 clinical trial?
NCT05519475 is sponsored by Regeneron Pharmaceuticals. The principal investigator is Clinical Trial Management at Regeneron Pharmaceuticals. The trial plans to enroll 120 participants.