NCT05648214 · Regeneron Pharmaceuticals
A Trial to Learn if ALN-PNP is Safe and Well Tolerated in Healthy Adults and Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)
What this study is about
This study is researching an experimental drug called ALN-PNP (called "study drug"). This is a first in human study. The study drug is not approved by any public health agency such as the United States Food and Drug Administration (FDA) for any kind of treatment. This study consists of 3 parts. Part A is focused on healthy participants.
View original scientific description
This study is researching an experimental drug called ALN-PNP (called "study drug"). This is a first in human study. The study drug is not approved by any public health agency such as the United States Food and Drug Administration (FDA) for any kind of treatment. This study consists of 3 parts. Part A is focused on healthy participants. Parts B and C of the study are focused on participants who are known to have MASLD and a specific variant of the PNPLA3 gene. The aim of the study is to see how safe, tolerable and effective the study drug is.
Interventions
DRUG
ALN-PNP
Administered per the protocol
DRUG
Placebo (PB)
Administered per the protocol
Primary outcome measures
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to Day 337
Severity of TEAEs
Time frame: Up to Day 337
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
- Part A (Healthy Adults):
- From 18 to 55 years of age
- For Japanese cohorts ONLY; the Japanese participant must:
- Be Japanese, born in Japan, and have both biologic parents and 4 biologic grandparents who are ethnically Japanese and born in Japan
- Have maintained a Japanese lifestyle, with no significant change since leaving Japan, including having access to Japanese food and adhering to a Japanese diet
- Be living \<10 years outside of Japan
- Has a Body Mass Index (BMI) between 18 and 32 kg/m\^2, inclusive, at the screening visit
- Is judged by the investigator to be in good health, as described in the protocol
- Is in good health based on laboratory safety testing obtained at the screening visit and approximately within 24 hours prior to administration of study drug Part B and Part C (Participants with MASLD):
- Part B: From 18 to 65 years of age
- Part C: From 18 to 75 years of age
- BMI from 23.0 kg/m2 to 40.0 kg/m2, inclusive, for East Asians (including but not limited to South Koreans, Chinese, Taiwanese, and Japanese) and BMI from 27.0 kg/m2 to 40.0 kg/m2, inclusive, for any other ethnicity at screening visit 1
- Liver fat content ≥8.5% as measured by MRI-PDFF at screening visit 3 Key
Exclusion criteria
- History of clinically significant cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, psychiatric, neurological, or dermatologic disease, as assessed by the investigator, that may confound the results of the study or poses an additional risk to the participant by study participation
- Presents any concern to the study investigator that might confound the results of the study or poses an additional risk to the participant by their participation in the study
- Hospitalized for any reason within 30 days of the screening visit
- Using the Modification of Diet in Renal Disease equation, has a glomerular filtration rate as described in the protocol at the screening visit
- Has Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) or total bilirubin above the Upper Limit of Normal (ULN) range
- Is a current smoker or former smoker, including e-cigarettes, who stopped smoking within 3 months prior to the screening visit
- Has a history of alcohol or drug abuse per investigator opinion
- Is positive for hepatitis C antibody and if so, positive for qualitative (ie, detected or not detected) Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) test at the screening visit Part B and Part C:
- Evidence of other forms of known chronic liver disease, as defined in the protocol
- Has a contraindication to MRI examinations, as defined in the protocol
- History of Type 1 diabetes
- Has lost or gained more than 4.0% body weight over the 3 months prior to or during the screening period
- Has known Human Immunodeficiency Virus (HIV) infection, evidence of current or chronic Hepatitis B Virus (HBV) infection, or current or chronic HCV infection, as defined in the protocol
- Bariatric surgery within approximately 5 years (Part B) or 3 years (Part C) prior or planned during the study period NOTE: Other protocol defined inclusion / exclusion criteria apply
Where
- Glendale, California
- Los Angeles, California
- Miami, Florida
- Marrero, Louisiana
- Houston, Texas
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 Apr 16, 2026 · Source of record for eligibility and locations