MN-001 in Non-alcoholic Fatty Liver Disease, Type 2 Diabetes Mellitus, and Hypertriglyceridemia

Study Overview
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Brief Summary
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The design of the Phase 2 clinical trial includes the following elements: Multi-center, two-arm, randomized, double-blind, placebo-controlled trial to evaluate MN-001 (tipelukast) vs. placebo in approximately 40 patients in the U.S. Patients will be randomized 1:1 to receive either 500 mg/day of MN-001 (tipelukast) or placebo for 24 weeks. The co-primary endpoints are (1) change from baseline in liver fat content measured by controlled attenuation parameter (CAP) score at Week 24, and (2) change from baseline in fasting serum triglycerides at Week 24. FebroScan is a non-invasive, quantitative, and accurate measure of liver fat content commonly used in early phase trials to measure treatment response. Secondary endpoints include safety and tolerability and changes in lipid profile (HDL-C, LDL-C, and total cholesterol).

Official Title
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A Phase 2, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Efficacy of MN-001 in Patients Diagnosed With Non-alcoholic Fatty Liver Disease, Type 2 Diabetes Mellitus, and Hypertriglyceridemia

Conditions
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Diabetes Mellitus, Type 2, Hypertriglyceridemia, Non-Alcoholic Fatty Liver Disease

Intervention / Treatment
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* Drug: MN-001
* Drug: MN-001 placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: FibroScan® CAP score ≥ 248 dB/m within 8 weeks of randomization. Diagnosis or history of Type 2 Diabetes mellitus with hemoglobin A1c (HbA1c) >6.5 and ≤10% at Screening. Fasting serum triglycerides (TG) at Screening >150 mg/dL On a stable dose of oral antidiabetic therapy for a minimum of 3 months prior to Screening. Exclusion Criteria: Other causes of chronic liver disease (autoimmune, primary biliary cholangitis, HBV, HCV, Wilson's, α-1-antitrypsin deficiency, hemochromatosis, biopsy-proven cirrhosis, hepatocellular carcinoma); Documented history of advanced liver fibrosis Evidence of cirrhosis, hepatic decompensation, portal hypertension including splenomegaly, ascites, encephalopathy and/or esophageal varices; Diagnosis or history of Diabetes mellitus type 1; Weight change >5% within last 3 months of Screening visit; Active gastrointestinal disease or history of gastric bypass surgery which could interfere with the absorption of oral medication; History of clinically significant acute cardiac event within 6 months of Screening;

Ages Eligible for Study
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Minimum Age: 21 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MN-001<br> | Drug: MN-001<br>* MN-001 is a novel, orally bioavailable small molecule compound<br>|
| Placebo Comparator: MN-001 Placebo<br>The placebo comparator is a tablet identical in appearance to MN-001. | Drug: MN-001 placebo<br>* The placebo tablet is identical in appearance to the MN-001 tablet, and contains excipients of MN-001.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change in controlled attenuation parameter (CAP) score by sound-based elastography at Week 24 |  | Week 24 | 
| Mean change from baseline in fasting serum triglyceride levels at Week 24 |  | Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of MN-001 | Incidence of adverse events, abnormal clinical laboratory results | Baseline to Week 24 | 
| Mean change from baseline in lipids | Changes in lipids (HDL-C, LDL-C, total cholesterol) after MN-001 treatment for 24 weeks | Baseline to Week 24 |