This is a randomized, double-blind, placebo-controlled study that will evaluate the safety, efficacy, tolerability of BIO89-100 in patients with biopsy-confirmed fibrosis stages F2-F3 NASH.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
222
Main Study: Number of Participants With Histological Resolution of Nonalcoholic Steatohepatitis (NASH) Without Worsening of Fibrosis
Nonalcoholic fatty liver disease activity score (NAS) was the sum of the scores of steatosis, inflammation, and ballooning. NAS score ranged from 0 to 8, with higher scores indicating worse disease severity. Resolution of NASH was defined as the total absence of ballooning (score=0) and absent or mild inflammation (score=0 to 1). NASH clinical research system (CRN) fibrosis is staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis). Worsening of fibrosis was defined as progression of fibrosis greater than or equal to (≥) 1 stage in NASH CRN fibrosis score.
Time frame: Week 24
Main Study: Number of Participants Who Achieved Improvement of Fibrosis ≥1 Stage Without Worsening of NASH
Worsening of NASH was defined as increase in nonalcoholic fatty liver disease activity score (NAS) for ballooning, inflammation, or steatosis. NAS was the sum of the scores of steatosis, inflammation, and ballooning. NAS score ranged from 0 to 8, with higher scores indicating worse disease severity. Fibrosis improvement was defined as ≥1-stage decrease in NASH CRN fibrosis score. NASH CRN fibrosis was staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).
Time frame: Week 24
Main Study: Number of Participants With at Least a 2-Point Improvement in NAS and no Worsening of Fibrosis
NAS was the sum of the scores of steatosis, inflammation, and ballooning. NAS score ranged from 0 to 8, with higher scores indicating worse disease severity. Worsening of fibrosis was defined as progression of fibrosis ≥1 stage in NASH CRN fibrosis score. NASH CRN fibrosis is staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).
Time frame: Week 24
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89bio Clinical Study Site
Birmingham, Alabama, United States
89bio Clinical Study Site
Birmingham, Alabama, United States
89bio Clinical Study Site
Dothan, Alabama, United States
89bio Clinical Study Site
Guntersville, Alabama, United States
89bio Clinical Study Site
Madison, Alabama, United States
89bio Clinical Study Site
Chandler, Arizona, United States
89bio Clinical Study Site
Glendale, Arizona, United States
89bio Clinical Study Site
Peoria, Arizona, United States
89bio Clinical Study Site
Tucson, Arizona, United States
89bio Clinical Study Site
Tucson, Arizona, United States
...and 79 more locations
Main Study: Number of Participants With NASH Resolution and Fibrosis Improvement ≥1 Stage
NAS was the sum of the scores of steatosis, inflammation, and ballooning. NAS score ranged from 0 to 8, with higher scores indicating worse disease severity. Resolution of NASH was defined as the total absence of ballooning (score=0) and absent or mild inflammation (score=0 to 1). Fibrosis improvement was defined as ≥1-stage decrease in NASH CRN fibrosis score. NASH CRN fibrosis was staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).
Time frame: Week 24
Main Study: Number of Participants With at Least a 2-point Improvement in NAS Score and Are Magnetic Resonance Imaging - Proton Density Fat Fraction (MRI-PDFF) Responders and Alanine Aminotransferase (ALT) Responders
A responder was defined as achieving ≥2 point improvement in NAS score and are MRI-PDFF responders and ALT responders at Week 24. NAS was the sum of the scores of steatosis, inflammation, and ballooning. NAS score ranged from 0 to 8, with higher scores indicating worse disease severity. MRI-PDFF responder was defined as ≥30% reduction from baseline in liver fat by MRI-PDFF. ALT responder was defined as ≥17 units/liter (U/L) or ≥30% reduction from baseline in ALT.
Time frame: Week 24
Main Study: Percent Change From Baseline in Serum Triglycerides
Time frame: Baseline, Week 24
Main Study: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-c)
Time frame: Baseline, Week 24
Main Study: Percent Change From Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-c)
Time frame: Baseline, Week 24
Main Study: Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-c)
Time frame: Baseline, Week 24
Main Study: Percent Change From Baseline in Adiponectin
Time frame: Baseline, Week 24
Main Study: Percent Change From Baseline in HbA1c (Glycated Hemoglobin)
Time frame: Baseline, Week 24
Main Study: Percent Change From Baseline in Alanine Transaminase
Time frame: Baseline, Week 12 and 24
Main Study: Percent Change From Baseline in Hepatic Fat Fraction By Magnetic Resonance Imaging - (MRI-PDFF)
Time frame: Baseline, Week 12 and 24
Main Study: Percent Change From Baseline in N-Terminal Type III Collagen Propeptide (Pro-C3)
Time frame: Baseline, Week 12 and 24
Main Study: Trough Serum Concentration of Pegozafermin
Serum trough concentration (ng/mL) of pegozafermin taken from pre-dose samples.
Time frame: Predose at Week 12 and 24
Main and Extension Study: Percent Change From Baseline in Alanine Transaminase
Baseline was prior to dosing on Day 1 of the main study.
Time frame: Baseline, Week 48
Main and Extension Study: Percent Change From Baseline in N-Terminal Type III Collagen Propeptide (Pro-C3)
Baseline was prior to dosing on Day 1 of the main study.
Time frame: Baseline, Week 48
Main and Extension Study: Percent Change From Baseline in Hepatic Fat Fraction By Magnetic Resonance Imaging - (MRI-PDFF)
Baseline was prior to dosing on Day 1 of the main study.
Time frame: Baseline, Week 48
Main and Extension Study: Trough Serum Concentration of Pegozafermin
Serum trough concentration (ng/mL) of pegozafermin taken from pre-dose samples.
Time frame: Predose at Week 48
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of IP, whether or not considered related to the IP. TEAEs were defined as AEs that started or worsened on or after the first dose of study IP up to Week 51. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, important medical event or reaction. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Time frame: Baseline up to Week 51