Safety and Tolerability of VK2809 in Patients With Primary Hypercholesterolemia and Non-Alcoholic Fatty Liver Disease

Study Overview
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Brief Summary
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This study will investigate the efficacy, safety, and tolerability of VK2809 in lowering LDL-C and liver fat content in patients with primary hypercholesterolemia and fatty liver disease. The primary efficacy endpoint is percent change from baseline LDL-C at the end of the treatment period (Week 12). Secondary endpoints include effects on liver fat content and other liver and lipid markers, as well as effects on safety and tolerability, and pharmacokinetic (PK) measurements.

Official Title
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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Assess The Efficacy, Safety, and Tolerability of VK2809 Administered for 12 Weeks Followed by a 4-Week Off-Drug Phase in Patients With Primary Hypercholesterolemia and Non-Alcoholic Fatty Liver Disease

Conditions
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Hyperlipidemia, NAFLD

Intervention / Treatment
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* Drug: VK2809
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Minimum of 10% liver fat as assessed by MRI - Proton Density Fat Fraction Fasting serum LDL-C >130 mg/dL at screening, >110 mg/dL on lipid lowering medications Any one of the following: Triglycerides ≥150 mg/dL or receiving prescription medication for elevated triglycerides. Systolic blood pressure >130 mmHg or diastolic blood pressure ≥85 mmHg or receiving prescription medication for hypertension. Waist circumference >40 inches (men) or >35 inches (women) Body mass index (BMI) 18.50 - 40.00 kg/m2 inclusive at screening Provide a personally-signed and dated informed consent document Exclusion Criteria: Females of childbearing potential and males unwilling to use barrier birth control method (condom) throughout the study Resting 12-lead ECG showing QTc >450 msec, any tachyarrhythmia or morphology change, or any other clinically significant abnormality Cardiovascular event requiring hospitalization in the past year History or presence of thyroid disorder History of malignancy in past 5 years LDL-C ≥190 mg/dL or familial hypercholesterolemia Significant hepatic or renal function test abnormalities

Ages Eligible for Study
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Minimum Age: 18 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 |
| --- | --- |
| Placebo Comparator: Placebo<br>Placebo capsule | Drug: Placebo<br> <br> |
| Experimental: VK2809 (5mg)<br>5mg VK2809 capsule | Drug: VK2809<br> <br> |
| Experimental: VK2809 (10mg)<br>10mg VK2809 capsule | Drug: VK2809<br> <br> |
| Experimental: VK2809 (10mg QOD)<br>10mg VK2809 capsule | Drug: VK2809<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in LDL-C in patients receiving VK2809 compared to placebo |  | 12 weeks |