Phase
Condition
Liver Disease
Primary Biliary Cholangitis
Treatment
denifanstat
Placebo
Clinical Study ID
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Willing and able to participate in the study and provide written informed consent.
Adults between 18 and 75 years of age.
Body mass index (BMI) ≥23 kg/m2 for Asian patients and ≥25 kg/m2 for patients ofother races.
Presence of metabolic risk factor(s), as follows:
T2DM OR
At least 2 out of 4 of the following:
BMI ≥30 kg/m2,
Hypertension, or on active antihypertensive treatment
Elevated fasting serum TGs or on active treatment for hypertriglyceridemia
Reduced fasting serum HDL-c, or on active treatment for dyslipidemia.
For patients with T2DM:
HbA1c ≤9.5%
If treatment naive: patients must have been diagnosed for at least 12 weeks prior toscreening
Suspected or confirmed diagnosis of MASH or MASLD or non-invasively diagnosed MASHor MASLD
Stable ALT and AST levels
Exclusion
Exclusion Criteria:
Previous intake of an approved MASH medication
Exclusionary laboratory values:
ALT and/or AST >5 × ULN.
ALP ≥2 × ULN.
Total serum bilirubin concentration >1.3 mg/dL.
Serum albumin concentration <3.5 g/dL.
International normalized ratio (INR) >1.3, except for patients receivinganticoagulant treatment.
Platelet count <140,000/μL.
Fasting TG level ≥500 mg/dL.
eGFR <45 mL/min/1.73 m2.
History of excessive alcohol intake for a period of more than 3 consecutive monthswithin 1 year prior to screening.
Presence of cirrhosis on liver histology and/or cross-sectional imaging evidenceconsistent with cirrhosis and/or portal hypertension.
Current or historical clinically evident hepatic decompensation.
Evidence of another form of active liver disease.
Positive serologic evidence of current infectious liver disease.
MELD score ≥12.
Planned or history of liver transplantation.
Prior or planned bariatric surgery.
Gain or loss of >5% of body weight in the 3 months or >10% of body weight in the 6months prior to screening, qualifying liver biopsy, and the baseline visit (V1).
Any of the following conditions or procedures within 6 months prior to the baselinevisit (V1):
Myocardial infarction
Cardiac revascularization surgery
Unstable angina
Transient ischemic attack, stroke, or cerebrovascular disease
Unstable or undiagnosed arrhythmias.
Uncontrolled high BP.
Malignancy with a complete remission date within 5 years prior to the baseline visit (V1).
Any current or history of hepatocellular carcinoma.
Diabetes other than T2DM
Uncontrolled hypothyroidism.
Any other known serious disease or other disease which in the Investigator's opinionwould exclude the patient from participating in the study.
Use of a nonpermitted concomitant medication within 30 days or 5 half-lives prior toscreening.