A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of BFKB8488A Compared with Placebo in Patients with Non-Alcoholic Steatohepatitis

A PHASE II, RANDOMIZED, PARALLEL-GROUP, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TOEVALUATE THE EFFICACY, SAFETY, AND PHARMACOKINETICS OF BFKB8488A COMPAREDWITH PLACEBO IN PATIENTS WITH NON-ALCOHOLIC STEATOHEPATITIS.

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-001897-27-FR

Enrollment

260

Registered

2019-10-07

Start date

2020-01-07

Completion date

Unknown

Last updated

2023-02-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Non-alcoholic steatohepatitis (NASH) MedDRA version: 22.0 Level: PT Classification code 10053219 Term: Non-alcoholic steatohepatitis System Organ Class: 10019805 - Hepatobiliary disorders

Interventions

Product Name: BFKB8488A Product Code: RO7040551 Pharmaceutical Form: Solution for injection INN or Proposed INN: BFKB8488A Current Sponsor code: BFKB8488A - RO7040551 Concentration unit: mg/ml milligr

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Age >= 18 and = 8% average liver PDFF) prior to randomization - Confirmed diagnosis of NASH as documented through liver biopsy performed no more than 6 months before randomization, defined according to NASH CRN criteria along with a NASH CRN fibrosis score between F2 and F3 - Use of highly effective contraception as defined by the protocol Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 215 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 45

Exclusion criteria

Exclusion criteria: - Patients with Type 1 diabetes, or on treatment with glucagon-like peptide-1 receptor agonists and/or thiazolidinediones - Treatment with drugs historically associated with NAFLD for more than 2 weeks within the year prior to randomization - History of endocrine diseases defined in the protocol including but not limited to Cushing’s disease, Addison’s disease, and hyper- or hypo-thyroidism - History of any liver disease other than NASH, except for resolved, self-limited illnesses such as hepatitis A or E, and previous Hepatitis C and liver transplantation - Estimated glomerular filtration rate (eGFR) = 5 × upper limit of normal (ULN), Alkaline phosphatase >= 2 × ULN and total bilirubin > ULN at screening - Actively involved in a structured weight loss, dietary program, treatment with medications for the purpose of weight loss, or planned medical procedure or surgery during the study - Weight gain or loss > 5% within 3 months prior to randomization - Patients with osteoporosis, other bone diseases/conditions, or have a history or are current on bone active treatments prohibited by the protocol - Current or history of significant alcohol consumption - Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study

Design outcomes

Primary

Measure	Time frame
Main Objective: •To evaluate the efficacy of BFKB8488A compared with placebo on the basis of resolution of NASH without worsening of fibrosis;Secondary Objective: •To evaluate the efficacy of BFKB8488A compared with placebo on the basis of hepatic fat fraction assessed by magnetic resonance imaging- derived proton density fat fraction (MRI-PDFF), proportion of patients with improvement in liver histology in non-alcoholic fatty liver disease (NAFLD) activity score [NAS], in liver fibrosis greater than or equal to one stage and no worsening of steatohepatitis •To evaluate the safety of BFKB8488A compared with placebo on the basis of incidence and severity of adverse events, changes in vital signs, clinical laboratory test results •To evaluate the BFKB8488A pharmacokinetic (PK) profile on the basis of the serum concentration of BFKB8488A •To evaluate the immune response to BFKB8488A on the basis of incidence and titer of anti-drug antibodies (ADAs) ;Primary end point(s): 1. Proportion of patients with resolution of NASH on overall histopathological reading, without worsening of fibrosis at Week 52;Timepoint(s) of evaluation of this end point: 1. At Week 52	—

Measure

Time frame

Main Objective: •To evaluate the efficacy of BFKB8488A compared with placebo on the basis of resolution of NASH without worsening of fibrosis;Secondary Objective: •To evaluate the efficacy of BFKB8488A compared with placebo on the basis of hepatic fat fraction assessed by magnetic resonance imaging- derived proton density fat fraction (MRI-PDFF), proportion of patients with improvement in liver histology in non-alcoholic fatty liver disease (NAFLD) activity score [NAS], in liver fibrosis greater than or equal to one stage and no worsening of steatohepatitis •To evaluate the safety of BFKB8488A compared with placebo on the basis of incidence and severity of adverse events, changes in vital signs, clinical laboratory test results •To evaluate the BFKB8488A pharmacokinetic (PK) profile on the basis of the serum concentration of BFKB8488A •To evaluate the immune response to BFKB8488A on the basis of incidence and titer of anti-drug antibodies (ADAs) ;Primary end point(s): 1. Proportion of patients with resolution of NASH on overall histopathological reading, without worsening of fibrosis at Week 52;Timepoint(s) of evaluation of this end point: 1. At Week 52

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Secondary

Measure	Time frame
Secondary end point(s): 1.Change from baseline in hepatic fat fraction as assessed by MRI-PDFF at Week 52 2.Proportion of patients improvement in fibrosis of at least 1 stage from baseline with no worsening of NASH at Week 52 3.Proportion of patients improvement in NAS score by at least 2 points from baseline at Week 52 4.Incidence and severity of adverse events, with severity determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) grading scale 5.Change from baseline in targeted vital signs 6.Change from baseline in targeted clinical laboratory test results 7.Maximum serum concentration observed (Cmax) of BFKB8488A 8.Minimum serum concentration observed (Cmin) of BFKB8488A 9.Incidence and titer of ADAs during the study relative to the prevalence of ADAs at baseline;Timepoint(s) of evaluation of this end point: 1. At Week 52 2-3. At Week 52 4. Up to Week 58 (or up to 6 weeks after the final dose of study drug) 5-6. Baseline to Week 58 7-8. Week 0, Week 4, Week 8, Week 12, Week 16, Week 28, Week 29, Week 40, Week 52, Week 58 and unscheduled visit 9. Week 0, Week 4, Week 8, Week 12, Week 16, Week 28, Week 40, Week 52, Week 58 and unscheduled visit	—

Measure

Time frame

Secondary end point(s): 1.Change from baseline in hepatic fat fraction as assessed by MRI-PDFF at Week 52 2.Proportion of patients improvement in fibrosis of at least 1 stage from baseline with no worsening of NASH at Week 52 3.Proportion of patients improvement in NAS score by at least 2 points from baseline at Week 52 4.Incidence and severity of adverse events, with severity determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) grading scale 5.Change from baseline in targeted vital signs 6.Change from baseline in targeted clinical laboratory test results 7.Maximum serum concentration observed (Cmax) of BFKB8488A 8.Minimum serum concentration observed (Cmin) of BFKB8488A 9.Incidence and titer of ADAs during the study relative to the prevalence of ADAs at baseline;Timepoint(s) of evaluation of this end point: 1. At Week 52 2-3. At Week 52 4. Up to Week 58 (or up to 6 weeks after the final dose of study drug) 5-6. Baseline to Week 58 7-8. Week 0, Week 4, Week 8, Week 12, Week 16, Week 28, Week 29, Week 40, Week 52, Week 58 and unscheduled visit 9. Week 0, Week 4, Week 8, Week 12, Week 16, Week 28, Week 40, Week 52, Week 58 and unscheduled visit

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Countries

Belgium, France, United States

Contacts

Public ContactTrial Information Support Line-TISL

F. Hoffman-La Roche Ltd

global.rochegenentechtrials@roche.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026