204 Participants NeededMy employer runs this trial

ALN-PNP + Tirzepatide for Fatty Liver Disease

Recruiting at 5 trial locations
CT
Overseen ByClinical Trials Administrator
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: Regeneron Pharmaceuticals
Must be taking: Tirzepatide
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment option for people with fatty liver disease, specifically examining how the experimental drug ALN-PNP works alone and in combination with tirzepatide, a medication typically used for managing blood sugar and weight. The goal is to determine if these treatments can reduce liver fat and to understand their effects on the body. Individuals diagnosed with fatty liver disease, possessing a specific genetic profile related to liver fat, and experiencing significant liver fat (15% or more) might be suitable candidates for this study. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

In a previous study, ALN-PNP was well tolerated at all tested dose levels. Researchers found that it helped reduce liver fat, a major issue in nonalcoholic fatty liver disease (NAFLD). No severe side effects were reported for ALN-PNP, indicating a favorable safety profile.

Tirzepatide, the other drug in the study, is already approved for controlling blood sugar, appetite, and weight. Earlier trials have shown it to be safe and well-tolerated. The most common side effects were stomach-related, such as nausea, while serious side effects were rare.

Overall, both ALN-PNP and tirzepatide have demonstrated good safety records in these studies, suggesting they are generally safe for human use.12345

Why are researchers excited about this trial's treatments?

Researchers are excited about ALN-PNP combined with Tirzepatide for treating fatty liver disease because it offers a novel approach compared to current treatments. While most existing therapies focus on lifestyle changes and medications that reduce fat in the liver indirectly, ALN-PNP targets the liver more directly with a unique mechanism, potentially reducing fat accumulation with higher specificity. Additionally, Tirzepatide is known for its dual action on glucose and weight management, which could enhance the overall effectiveness of the treatment by addressing underlying metabolic issues linked to fatty liver disease. This innovative combination could lead to more efficient and targeted results, making it a promising option for patients.

What evidence suggests that this trial's treatments could be effective for fatty liver disease?

Research has shown that ALN-PNP, a treatment tested in this trial, can help reduce liver fat, a major issue in fatty liver disease. Early results indicate its safety and effectiveness in lowering liver fat levels. Another treatment in this trial, Tirzepatide, has also shown promising results. It can significantly reduce liver fat and improve liver health, with some studies showing up to a 55% reduction over a year. Together, these treatments focus on reducing liver fat and may offer new hope for people with fatty liver disease.13678

Who Is on the Research Team?

CT

Clinical Trial Management

Principal Investigator

Regeneron Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for adults with fatty liver disease who have two copies of a specific PNPLA3 gene change, have significant liver fat (≥15%), and a BMI between 30 and less than 45. Their liver enzymes must not be too high, and if taking tirzepatide, the dose should be stable.

Inclusion Criteria

I have two copies of the PNPLA3 p.I148M gene variant.
2. Liver fat by Magnetic Resonance Imaging-Proton Density Fat Fraction (MRI-PDFF) ≥15% at visit 3
3. Has a Body Mass Index (BMI) ≥30 to \<45 kg/m\^2 at visit 2
See 3 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ALN-PNP with or without tirzepatide to assess its effect on liver fat reduction

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • ALN-PNP
  • Tirzepatide

Trial Overview

The study tests ALN-PNP alone or combined with tirzepatide (a drug used for blood sugar and weight control) versus placebo to see how well they reduce liver fat in people with this genetic form of fatty liver disease.

How Is the Trial Designed?

6

Treatment groups

Experimental Treatment

Placebo Group

Group I: Part B: B3Experimental Treatment1 Intervention
Group II: Part B: B2Experimental Treatment2 Interventions
Group III: Part B: B1Experimental Treatment2 Interventions
Group IV: Part A: A2Experimental Treatment2 Interventions
Group V: Part A: A1Experimental Treatment2 Interventions
Group VI: Part B: B4Placebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Regeneron Pharmaceuticals

Lead Sponsor

Trials
690
Recruited
948,000+
Founded
1988
Headquarters
Tarrytown, USA
Known For
Precision medicine
Top Products
Dupixent, EYLEA, Libtayo, Praluent
Leonard Schleifer profile image

Leonard Schleifer

Regeneron Pharmaceuticals

Chief Executive Officer since 1988

MD and PhD in Medicine

George Yancopoulos profile image

George Yancopoulos

Regeneron Pharmaceuticals

Chief Medical Officer since 1997

MD from Harvard Medical School

Citations

NCT06024408 | A Trial to Learn if Receiving ALN-PNP ...

This study is researching an experimental drug called ALN-PNP. The aim of this study is to see how safe, tolerable, and effective the study drug is.

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Particularly in this study, pioglitazone showed significant improvements in NAS scores and resolution of NASH without worsening fibrosis, demonstrating its ...

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Initial findings suggest that ALN-PNP might help reduce liver fat, a major issue in nonalcoholic fatty liver disease (NAFLD).

Study Details | NCT05648214 | A Trial to Learn if ALN-PNP ...

This study is researching an experimental drug called ALN-PNP … works to change liver fat content in MASLD (Part B and C) Is positive for hepatitis

Nonalcoholic Fatty Liver Disease Clinical Trials

The purpose of this study is to identify risk factors and potential predictors for NAFLD and NASH recurrence after liver transplantation.

A Trial to Learn if ALN-PNP is Safe and Well Tolerated in ...

A Trial to Learn if ALN-PNP is Safe and Well Tolerated in Healthy Adults and Adult Participants With Metabolic Dysfunction-Associated Steatotic ...

ALN-PNP / Alnylam, Regeneron

ALN-PNP was well tolerated at all dose levels tested and reduced liver fat clinicaltrials.gov) Trial primary completion date:

Basic science to clinical trials in non-alcoholic fatty liver ... - PMC

both can lead to similar complications including end-stage liver disease, increased risk of cardiovascular disease (3), and hepatocellular carcinoma (HCC) (3,4 ...