Actively Recruiting

Phase 1
Phase 2
Age: 18Years - 75Years
All Genders
Healthy Volunteers
NCT05648214

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)

Led by Regeneron Pharmaceuticals · Updated on 2026-04-16

172

Participants Needed

6

Research Sites

241 weeks

Total Duration

On this page

AI-Summary

What this Trial 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. 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. The study is looking at several other research questions, including: * What side effects may happen from taking the study drug (Parts A, B and C) * How much study drug (Parts A, B and C) and study drug metabolites (byproduct of the body breaking down the study drug) (Parts B and C) are in the blood at different times * Whether the body makes antibodies against the study drug (which could make the study drug less effective or could lead to side effects) (Part A, B and C) * Explore impact of Japanese ethnicity on safety and PK (Pharmacokinetics, or study of what the body does to the drug) of single doses of ALN-PNP over time (Part A) * How the study drug works to change liver fat content in MASLD (Part B and C) * Better understanding of the study drug and MASLD (Part B and C)

CONDITIONS

Official Title

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)

Who Can Participate

Age: 18Years - 75Years
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Part A: Ages 18 to 55 years
  • Japanese participants in Part A must be ethnically Japanese, born in Japan, have Japanese parents and grandparents, maintain a Japanese lifestyle and diet, and have lived less than 10 years outside Japan
  • Body Mass Index (BMI) between 18 and 32 kg/m² for Part A
  • Good health based on investigator judgment and lab safety tests for Part A
  • Part B: Ages 18 to 65 years
  • Part C: Ages 18 to 75 years
  • BMI between 23.0 and 40.0 kg/m² for East Asians and between 27.0 and 40.0 kg/m² for other ethnicities in Parts B and C
  • Liver fat content of at least 8.5% measured by MRI-PDFF at screening for Parts B and C
Not Eligible

You will not qualify if you...

  • Part A: History of significant cardiovascular, respiratory, liver, kidney, gastrointestinal, endocrine, hematological, psychiatric, neurological, or dermatologic diseases
  • Part A: Any condition that may affect study results or increase participant risk
  • Part A: Hospitalization within 30 days before screening
  • Part A: Reduced kidney function as defined in protocol
  • Part A: Elevated liver enzymes (ALT, AST) or total bilirubin above normal limits
  • Part A: Current or recent smoker within 3 months
  • Part A: History of alcohol or drug abuse
  • Part A: Positive hepatitis C antibody and RNA test
  • Parts B and C: Other chronic liver diseases
  • Parts B and C: Contraindications to MRI
  • Parts B and C: History of Type 1 diabetes
  • Parts B and C: Weight change over 4% in past 3 months
  • Parts B and C: Known HIV infection, current or chronic hepatitis B or C infection
  • Parts B and C: Bariatric surgery within 5 years (Part B) or 3 years (Part C) or planned during study

AI-Screening

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Trial Site Locations

Total: 6 locations

1

California Clinical Trials Medical Group

Glendale, California, United States, 91206

Completed

2

Velocity Clinical research

Los Angeles, California, United States, 90057

Actively Recruiting

3

Genoma Research Group, Inc

Miami, Florida, United States, 33173

Actively Recruiting

4

Med Research of Florida, LLC

Miami, Florida, United States, 33186

Actively Recruiting

5

Tandem Clinical Research

Marrero, Louisiana, United States, 70072

Actively Recruiting

6

Pioneer Research Solutions

Houston, Texas, United States, 77099

Actively Recruiting

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Research Team

C

Clinical Trials Administrator

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

5

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