Actively Recruiting
A Three-Part, Phase 1/2a Study of the Safety, Tolerability, and Pharmacokinetics of ALN-PNP in Healthy Adults and Adults With Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)
Led by Regeneron Pharmaceuticals · Updated on 2026-04-16
172
Participants Needed
6
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying an experimental drug called ALN-PNP in healthy adults and adults with Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD) who have a specific PNPLA3 gene variant. This first-in-human trial aims to evaluate the safety, tolerability, and how the drug is processed in the body across three parts: Part A with healthy participants, and Parts B and C with MASLD participants. The study also explores the drug's impact on liver fat and potential side effects related to the drug. The study involves randomized, double-blind treatment with ALN-PNP or placebo given in single and multiple doses. Part A includes healthy adults aged 18 to 55, including a cohort of Japanese participants, while Parts B and C include adults with MASLD aged up to 65 and 75 respectively. Participants receive the study drug according to protocol and are monitored for drug levels, immune response, and changes in liver fat and blood lipids over time. Participants will be involved in safety assessments including monitoring treatment-emergent adverse events up to day 337 and measuring liver fat by MRI-PDFF up to day 253. Blood samples will be taken to check drug concentration, metabolites, and antibodies. Lipid profiles and other biomarkers will be tracked through the study. The trial includes thorough follow-up over several months to assess safety, drug effects, and tolerability in detail.
CONDITIONS
Brief 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
Eligibility Criteria
You may qualify if you...
- For Part A (Healthy Adults): Age 18 to 55 years
- For Part A Japanese cohorts: Must be ethnically Japanese with Japanese lifestyle and living less than 10 years outside Japan
- Body Mass Index (BMI) between 18 and 32 kg/m2 for Part A
- Judged to be in good health by the investigator and lab tests within 24 hours before study drug
- For Part B (MASLD participants): Age 18 to 65 years
- For Part C (MASLD participants): Age 18 to 75 years
- BMI between 23.0 and 40.0 kg/m2 for East Asians; 27.0 to 40.0 kg/m2 for other ethnicities
- Liver fat content 8.5% or higher measured by MRI-PDFF at screening
You will not qualify if you...
- For Part A: History of significant cardiovascular, respiratory, liver, kidney, gastrointestinal, endocrine, blood, psychiatric, neurological, or skin disease
- Any condition that may confound study results or increase risk as judged by investigator
- Hospitalization within 30 days before screening
- Reduced kidney function as defined by study protocol
- Elevated liver enzymes (ALT, AST) or total bilirubin above normal range
- Current smoker or stopped smoking within 3 months before screening
- History of alcohol or drug abuse
- Positive hepatitis C antibody and RNA test
- For Parts B and C: Other known chronic liver diseases
- Contraindication to MRI
- History of Type 1 diabetes
- Weight change greater than 4% within 3 months before or during screening
- Known HIV infection or chronic Hepatitis B or C infection
- Bariatric surgery within 5 years (Part B) or 3 years (Part C) or planned during study
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 48 weeks
Participants receive ALN-PNP or placebo as part of the study treatment.
Visits according to protocol for dosing and assessments
Duration - Up to Day 337 (approximately 48 weeks) from first dose
Participants are monitored after treatment to assess safety and pharmacokinetics.
Multiple follow-up visits for safety and lab assessments
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
Research Team
C
Clinical Trials Administrator
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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