A Multiple Ascending Dose Study of Pegozafermin in Participants With Biopsy Confirmed Nonalcoholic Steatohepatitis (NASH) or Nonalcoholic Fatty Liver Disease (NAFLD) and at High Risk of NASH

NCT ID: NCT04048135

Last Updated: 2024-04-02

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

101 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-07-29

Study Completion Date

2022-01-19

Brief Summary

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Part 1: This is a multi-center evaluation of pegozafermin (administered weekly or every other week) in a randomized, double-blind, placebo-controlled study administered for 12 weeks in participants with NASH and NAFLD at high risk of NASH, including a pre-defined number of participants with biopsy confirmed NASH and fibrosis stages F1-F3 to be enrolled.

Part 2: This is a multi-center, open label evaluation of pegozafermin at 27 mg administered weekly for 20 weeks in participants with biopsy-proven NASH (NAS ≥4, fibrosis stage F2 or F3).

Detailed Description

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Conditions

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NASH

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators
Part 1 was blinded, Part 2 was open label

Study Groups

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Part 1: Pegozafermin 3 milligrams (mg) weekly (QW)

Participants were administered 3 mg of pegozafermin QW, via subcutaneous (SC) injection, starting on Day 1 through Day 85.

Group Type EXPERIMENTAL

Pegozafermin

Intervention Type DRUG

Subcutaneous injection

Part 1: Pegozafermin 9 mg QW

Participants were administered 9 mg of pegozafermin QW, via SC injection, starting on Day 1 through Day 85.

Group Type EXPERIMENTAL

Pegozafermin

Intervention Type DRUG

Subcutaneous injection

Part 1: Pegozafermin 18 mg QW

Participants were administered 18 mg of pegozafermin QW, via SC injection, starting on Day 1 through Day 85.

Group Type EXPERIMENTAL

Pegozafermin

Intervention Type DRUG

Subcutaneous injection

Part 1: Pegozafermin 27 mg QW

Participants were administered 27 mg of pegozafermin QW, via SC injection, starting on Day 1 through Day 85.

Group Type EXPERIMENTAL

Pegozafermin

Intervention Type DRUG

Subcutaneous injection

Part 1: Pegozafermin 18 mg Every 2 Weeks (Q2W)

Participants were administered 18 mg of pegozafermin Q2W, via SC injection, starting on Day 1 through Day 85.

Group Type EXPERIMENTAL

Pegozafermin

Intervention Type DRUG

Subcutaneous injection

Part 1: Pegozafermin 36 mg Q2W

Participants were administered 36 mg of pegozafermin Q2W, via SC injection, starting on Day 1 through Day 85.

Group Type EXPERIMENTAL

Pegozafermin

Intervention Type DRUG

Subcutaneous injection

Part 1: Placebo QW or Q2W

Participants were administered placebo matching to pegozafermin QW or Q2W, via SC injection, starting on Day 1 through Day 85.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Subcutaneous injection

Part 2: Pegozafermin 27 mg QW

Participants were administered 27 mg of pegozafermin QW, via SC injection, starting on Day 1 through Day 134.

Group Type EXPERIMENTAL

Pegozafermin

Intervention Type DRUG

Subcutaneous injection

Interventions

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Pegozafermin

Subcutaneous injection

Intervention Type DRUG

Placebo

Subcutaneous injection

Intervention Type OTHER

Other Intervention Names

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BIO89-100

Eligibility Criteria

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Inclusion Criteria

* Participants must be 21 to 75 years of age inclusive, at the time of signing the informed consent form (ICF).
* Evidence of steatosis by Fibroscan and magnetic resonance imaging based proton density fat fraction (MRI-PDFF)
* NASH or NAFLD at high risk for NASH as reflected by AT LEAST ONE of the following:
* Diagnosis of NASH with fibrosis (stages F1, F2 or F3), without cirrhosis, by percutaneous liver biopsy within 24 months prior to screening
* Central obesity WITH type 2 diabetes mellitus (T2DM)
* Central obesity WITH either increased alanine transaminase (ALT) and/or Fibroscan vibration-controlled transient elastography (VCTE) score ≥7 KPa.
* Part 2 only: Biopsy-proven NASH in a liver biopsy obtained within 24 weeks of baseline with fibrosis stage F2 or F3 and NAS ≥4, with a score of at least 1 in each of steatosis, ballooning degeneration, and lobular inflammation. A small number of high risk F1 allowed.

Exclusion Criteria

* Clinically significant disorder or a history of any illness that, in the opinion of the Investigator, might confound the results of the study, or pose additional risk to the participant by participation in the study.
* History of type 1 diabetes.
* Weight loss of more than 5% within 3 months prior to Day -1 or more than 10% within 6 months prior to Day -1 or planning to try to lose weight during conduct of study.
* History of a liver disorder other than NASH or clinical suspicion of a liver disorder other than NASH
* History of cirrhosis or evidence of cirrhosis
Minimum Eligible Age

21 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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ProSciento, Inc.

INDUSTRY

Sponsor Role collaborator

89bio, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Charlton, MD

Role: STUDY_DIRECTOR

89bio, Inc.

Locations

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89bio Clinical Study Site

Madison, Alabama, United States

Site Status

89bio Clinical Study Site

Chandler, Arizona, United States

Site Status

89bio Clinical Study Site

Tucson, Arizona, United States

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89bio Clinical Study Site

Chula Vista, California, United States

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89bio Clinical Study Site

Huntington Beach, California, United States

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89bio Clinical Study Site

Montclair, California, United States

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89bio Clinical Study Site

Miami, Florida, United States

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89bio Clinical Study Site

Miami Lakes, Florida, United States

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89bio Clinical Study Site

Ocala, Florida, United States

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89bio Clinical Study Site

Sarasota, Florida, United States

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89bio Clinical Study Site

Lake Charles, Louisiana, United States

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89bio Clinical Study Site

Berlin, New Jersey, United States

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89bio Clinical Study Site

Florham Park, New Jersey, United States

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89bio Clinical Study Site

East Syracuse, New York, United States

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89bio Clinical Study Site

Concord, North Carolina, United States

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89bio Clinical Study Site

Raleigh, North Carolina, United States

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89bio Clinical Study Site

Greenwood, South Carolina, United States

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89bio Clinical Study Site

Summerville, South Carolina, United States

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89bio Clinical Study Site

Hermitage, Tennessee, United States

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89bio Clinical Study Site

Austin, Texas, United States

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89bio Clinical Study Site

Edinburg, Texas, United States

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89bio Clinical Study Site

Houston, Texas, United States

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89bio Clinical Study Site

San Antonio, Texas, United States

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89bio Clinical Study Site

San Antonio, Texas, United States

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89bio Clinical Study Site

Wichita Falls, Texas, United States

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89bio Clinical Study Site

San Juan, , Puerto Rico

Site Status

Countries

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United States Puerto Rico

References

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Alkhouri N, Lazas D, Loomba R, Frias JP, Feng S, Tseng L, Balic K, Agollah GD, Kwan T, Iyer JS, Morrow L, Mansbach H, Margalit M, Harrison SA. Clinical trial: Effects of pegozafermin on the liver and on metabolic comorbidities in subjects with biopsy-confirmed nonalcoholic steatohepatitis. Aliment Pharmacol Ther. 2023 Nov;58(10):1005-1015. doi: 10.1111/apt.17709. Epub 2023 Sep 18.

Reference Type DERIVED
PMID: 37718721 (View on PubMed)

Tseng CL, Balic K, Charlton RW, Margalit M, Mansbach H, Savic RM. Population Pharmacokinetics and Pharmacodynamics of Pegozafermin in Patients with Nonalcoholic Steatohepatitis. Clin Pharmacol Ther. 2023 Dec;114(6):1323-1331. doi: 10.1002/cpt.3046. Epub 2023 Oct 2.

Reference Type DERIVED
PMID: 37696614 (View on PubMed)

Loomba R, Lawitz EJ, Frias JP, Ortiz-Lasanta G, Johansson L, Franey BB, Morrow L, Rosenstock M, Hartsfield CL, Chen CY, Tseng L, Charlton RW, Mansbach H, Margalit M. Safety, pharmacokinetics, and pharmacodynamics of pegozafermin in patients with non-alcoholic steatohepatitis: a randomised, double-blind, placebo-controlled, phase 1b/2a multiple-ascending-dose study. Lancet Gastroenterol Hepatol. 2023 Feb;8(2):120-132. doi: 10.1016/S2468-1253(22)00347-8. Epub 2022 Dec 12.

Reference Type DERIVED
PMID: 36521501 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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Other Identifiers

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BIO89-100-002

Identifier Type: -

Identifier Source: org_study_id

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