Pegozafermin for Liver Cirrhosis

Recruiting at 255 trial locations

Overseen By ENLIGHTEN clinical trial

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: 89bio, Inc.

Disqualifiers: Liver disorder, Hepatic decompensation, Hepatocellular carcinoma, Diabetes, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

762 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness and safety of pegozafermin for individuals with liver cirrhosis caused by MASH, a severe form of liver disease formerly known as NASH. Participants will receive either pegozafermin, an experimental treatment, or a placebo to determine if the treatment can improve their liver condition. This trial suits individuals with type 2 diabetes or certain metabolic risk factors who have been diagnosed with cirrhosis from MASH. As a Phase 3 trial, it represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, if you are taking vitamin E at more than 400 IU per day, you must have been on a stable dose for at least 6 months before joining the trial.

Is there any evidence suggesting that pegozafermin is likely to be safe for humans?

Research has shown that pegozafermin has promising safety results from earlier studies. In one study, most patients handled the treatment well, with 19 out of 20 participants completing the trial. Another study on safety and tolerability found that patients generally accepted the treatment well. While some side effects were reported, they were usually mild and did not cause participants to leave the study. These findings suggest that pegozafermin is generally safe for humans, based on earlier research. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for cirrhosis?

Most treatments for liver cirrhosis focus on managing symptoms or slowing disease progression. Pegozafermin is unique because it targets the fibroblast growth factor 21 (FGF21) pathway, which plays a role in reducing liver fat and inflammation. Researchers are excited about this treatment because it could directly address underlying metabolic issues in liver cirrhosis, potentially offering a more effective way to improve liver function compared to current options. Additionally, the promise of pegozafermin to reduce liver fat and inflammation could lead to better outcomes for patients, which is a significant improvement over existing treatments.

What evidence suggests that pegozafermin might be an effective treatment for liver cirrhosis?

Research has shown that pegozafermin, which participants in this trial may receive, may help treat liver problems like cirrhosis. In earlier studies, 26% of patients experienced less liver scarring without worsening their NASH (a type of fatty liver disease). Additionally, 32% of patients saw their NASH improve without increased scarring. Another study found that pegozafermin helped prevent the liver from worsening and developing into cirrhosis, which is severe liver damage. These results suggest that pegozafermin could be useful for managing liver cirrhosis related to metabolic issues. ^{1 3 5 6 7}

Who Is on the Research Team?

Maya Margalit, MD

Principal Investigator

89bio, Inc.

Are You a Good Fit for This Trial?

Adults aged 18-75 with compensated cirrhosis due to MASH, confirmed by a liver biopsy showing stage F4 fibrosis. Participants must have a BMI of ≥25 (≥23 for Asians) and <50 kg/m² and at least one metabolic risk factor. Pregnant individuals or those not meeting the specific criteria are excluded.

Inclusion Criteria

I have severe liver scarring confirmed by a biopsy.

Body mass index (BMI) at screening ≥25.0 (≥23.0 for Asian participants) and <50.0 kg/m^2

At least 1 metabolic risk factor

Exclusion Criteria

My liver condition is not MASH.

My liver is not working properly.

I have or had liver cancer.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive pegozafermin or placebo for the duration of the study

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Pegozafermin

Trial Overview The trial is testing Pegozafermin's effectiveness and safety against a placebo in treating compensated cirrhosis caused by MASH. It involves administering Pegozafermin to see if it improves liver health compared to no active treatment.

How Is the Trial Designed?

Group I: Pegozafermin Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Matched placebo

Who Is Running the Clinical Trial?

89bio, Inc.

Lead Sponsor

Trials

7

Recruited

2,600+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06318169;

A Study Evaluating the Efficacy and Safety of Pegozafermin ... An unfavorable change in the health of a participant, including abnormal laboratory findings, that happens during a clinical study or within a certain amount of ...

2. ir.89bio.com ir.89bio.com/news-releases/news-release-details/89bio-presents-new-analyses-evaluating-pegozafermin-and

89bio Presents New Analyses Evaluating Pegozafermin ... Pegozafermin reduced progression to cirrhosis: A post-hoc analysis from the Phase 2b ENLIVEN study (Publication 1563). Biomarker response in ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37718721/

Clinical trial: Effects of pegozafermin on the liver and on ... Improvement of fibrosis without worsening of NASH was observed in 26% of subjects, and NASH resolution without worsening of fibrosis in 32%.

4. clinicaltrials.cedars-sinai.edu clinicaltrials.cedars-sinai.edu/view/BIO89-100-132

Pegozafermin in Compensated Cirrhosis Due to Metabolic ... The purpose of this study is to learn about the safety, effectiveness, and long-term outcomes of an experimental drug called pegozafermin in people.

5. pharmaceutical-technology.com pharmaceutical-technology.com/data-insights/pegozafermin-89bio-liver-cirrhosis-likelihood-of-approval/

Pegozafermin by 89Bio for Liver Cirrhosis: Likelihood of ... According to GlobalData, Phase III drugs for Liver Cirrhosis have a 44% phase transition success rate (PTSR) indication benchmark for progressing into Pre- ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04929483

NCT04929483 | Study Evaluating the Safety, Efficacy and ... This is a randomized, double-blind, placebo-controlled study that will evaluate the safety, efficacy, tolerability of BIO89-100 in patients with ...

7. ir.89bio.com ir.89bio.com/node/9271/pdf

November 27, 2023 “Notably, we observed consistent and robust benefits in F2-F3 NASH patients, as well as in subgroups of patients receiving concurrent GLP-1.