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RECRUITING
NCT05648214
PHASE1

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)

Sponsor: Regeneron Pharmaceuticals

View on ClinicalTrials.gov

Summary

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)

Official title: A Three-Part, Phase 1, Randomized, Double-blind, Placebo-Controlled, Single and Multiple Dose Study of the Safety, Tolerability, and Pharmacokinetics of ALN-PNP, an siRNA Targeting PNPLA3, in Healthy Adults and Adult Participants With MASLD

Key Details

Gender

All

Age Range

18 Years - 75 Years

Study Type

INTERVENTIONAL

Enrollment

156

Start Date

2022-12-27

Completion Date

2027-08-10

Last Updated

2025-12-22

Healthy Volunteers

Yes

Interventions

DRUG

ALN-PNP

Administered per the protocol

DRUG

Placebo (PB)

Administered per the protocol

Locations (6)

California Clinical Trials Medical Group

Glendale, California, United States

Velocity Clinical research

Los Angeles, California, United States

Genoma Research Group, Inc

Miami, Florida, United States

Med Research of Florida, LLC

Miami, Florida, United States

Tandem Clinical Research

Marrero, Louisiana, United States

Pioneer Research Solutions

Houston, Texas, United States