NCT05519475 · Regeneron Pharmaceuticals
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
(NASHGEN-2)
What this study is about
This study is researching an experimental 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).
View original scientific 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).
Interventions
DRUG
ALN-HSD
Administered per the protocol
DRUG
Placebo
Administered per the protocol
Primary outcome measures
Change in quantitative liver Fibrosis (qFibrosis)
Time frame: Baseline to week 52
Change in the continuous qFibrosis score measured by second harmonic generation/two-photon excitation microscopy
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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-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
- 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-define
Where
- Chandler, Arizona
- Flagstaff, Arizona
- Peoria, Arizona
- Tucson, Arizona
- Canoga Park, California
- Chula Vista, California
- Coronado, California
- Fountain Valley, California
- Gardena, California
- Huntington Park, California
- Lancaster, California
- Long Beach, California
And 39 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 8, 2026 · Source of record for eligibility and locations