What side effects are associated with this type of intervention?

Common treatments for Non-alcoholic Fatty Liver Disease (NAFLD) include lifestyle modifications, lipid-lowering agents, and investigational drugs. Lifestyle changes like diet and exercise are generally safe but may cause temporary discomfort or fatigue. Lipid-lowering agents such as statins can cause muscle pain, liver enzyme abnormalities, and, in rare cases, severe liver damage. Investigational drugs, including those targeting genetic variants like PNPLA3, are still under study, but potential side effects may include gastrointestinal issues, liver enzyme elevations, and immune reactions such as antibody formation against the drug.

What prior FDA approvals exist?

As of now, there are no FDA-approved medications specifically for the treatment of Non-alcoholic Fatty Liver Disease (NAFLD). Current management primarily focuses on lifestyle modifications such as diet, exercise, and weight loss. Some investigational drugs, like ALN-PNP, are being studied for their potential to reduce liver fat content and improve liver health. Other drugs under investigation include lipid-lowering agents and anti-diabetic medications, which have shown some promise in clinical trials but are not yet approved specifically for NAFLD.

What other types of research exist?

Promising alternatives to ALN-PNP for NAFLD treatment include: 1) Tolvaptan, which has shown efficacy in reducing liver fat content and is being evaluated for safety and tolerability. 2) Lipid-lowering agents, which have demonstrated safety and efficacy in reducing hepatic steatosis in NAFLD/NASH patients. 3) Dipeptidyl peptidase-4 inhibitors like alogliptin, which are being studied for their impact on liver enzymes and overall liver health in NAFLD patients with type 2 diabetes. These alternatives are supported by ongoing research and clinical trials aimed at improving liver health in NAFLD patients.

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RNAi Therapeutics

ALN-PNP for Healthy Subjects

Verified Trial

Phase 1

Recruiting

Research Sponsored by Regeneron Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to day 253

Awards & highlights

Summary

This trial is testing a new drug called ALN-PNP on healthy people and those with a specific liver condition (NAFLD) and gene variant. The goal is to see if the drug is safe, tolerable, and effective in reducing liver fat. Researchers are also studying how the body processes the drug.

Who is the study for?

This trial is for healthy adults, including specific criteria for Japanese participants such as being born in Japan with Japanese parents and grandparents, living a Japanese lifestyle, and not having lived outside of Japan for more than 10 years. Participants must have a BMI between 18-32 and be in good health based on lab tests. Smokers or recent smokers, those with significant diseases or abnormal lab results are excluded.

What is being tested?

The study is testing the safety and how well people tolerate ALN-PNP when given once to healthy adults compared to a placebo. It will also look at how the body processes ALN-PNP, its effects on immune response, and any changes it might cause in blood lipid levels.

What are the potential side effects?

Since this is an early-stage trial to determine safety and tolerability, potential side effects are not fully known yet but may include typical drug-related reactions like injection site discomfort, allergic reactions, fatigue or changes in blood test results.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to day 253

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to day 253

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to day 253 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of TEAEs by severity

Incidence of treatment-emergent adverse events (TEAEs)

Secondary study objectives

Change in apolipoprotein A1 (ApoA1) over time

Change in apolipoprotein B (ApoB) over time

Change in high-density lipoprotein (HDL) over time

+9 more

Trial Design

12Treatment groups

Experimental Treatment

Placebo Group

Group I: Part B: ALN-PNP Dose 5Experimental Treatment1 Intervention

Up to 32 Participants Randomized 1:1:1:1 resulting in 8 participants per arm

Group II: Part B: ALN-PNP Dose 4Experimental Treatment1 Intervention

Up to 32 Participants Randomized 1:1:1:1 resulting in 8 participants per arm

Group III: Part B: ALN-PNP Dose 3Experimental Treatment1 Intervention

Up to 32 Participants Randomized 1:1:1:1 resulting in 8 participants per arm

Group IV: Part A: Optional ALN-PNP Dose 5 or PBExperimental Treatment2 Interventions

8 Participants, Randomized 6:2 This is an optional cohort, if there is a need for additional dose characterization of ALN-PNP.

Group V: Part A: JPN ALN-PNP Dose 5 or PBExperimental Treatment2 Interventions

8 Japanese Participants only, Randomized 6:2

Group VI: Part A: JPN ALN-PNP Dose 4 or PBExperimental Treatment2 Interventions

8 Japanese Participants only, Randomized 6:2

Group VII: Part A: ALN-PNP Dose 5 or PBExperimental Treatment2 Interventions