A Pilot, Dose Escalating Study on VLX103 in Moderate Alcoholic Steatohepatitis

Hepatic Steatosis Clinical Trial

Official title: A Pilot, Exploratory Dose Escalating Study on the Safety, Pharmacodynamics and Preliminary Efficacy of VLX103 in the Treatment of Moderate Alcoholic Steatohepatitis

Status Withdrawn

Clinical Trial Summary

The study drug (VLX103) is being developed for the treatment of Alcoholic Steatohepatitis and other liver diseases. Alcoholic Steatohepatitis is an inflammatory (associated with irritation, swelling and cell damage) disease that affects the liver. It is associated with heavy and chronic intake of alcohol and presence of fat in the liver. Signs and symptoms often include fever, yellowing of the skin, nausea and impairment of liver function.

The main objective of this study is to evaluate the safety, pharmacodynamics (what the drug does to the body) and pharmacokinetics (how the drug is handled by the human body, like absorption and elimination) of increasing doses of VLX103 in subjects with moderate Alcoholic Steatohepatitis. In other words, we will evaluate how your body tolerates VLX103 at a specific dose and the effects that this VLX103 dose has on your liver and your body in general. The secondary objectives of this study are to evaluate if VLX103 has the potential to treat Alcoholic Steatohepatitis patients, to determine the maximum dose that can be tolerated, and to measure the levels of VLX103 in your blood at different time points during the study.

VLX103 is an experimental drug. Experimental means that the drug has not been approved by the Food and Drug Administration (FDA) for the treatment of Alcoholic Steatohepatitis. The active ingredient in VLX103, pentamidine, is approved for treating parasitic (microorganisms) infections. Pentamidine is currently approved and marketed in about 20 countries, including the United States, for use by injection (administered by a syringe) and by inhalation (administered by a nebulizer) for other health conditions. However, VLX103 is the first oral form of pentamidine being developed, and is administered by mouth as an oral tablet.

Clinical Trial Description

This is an open label, multiple cohorts, dose escalation Phase Ib study, in which up to 3 doses of VLX103 will be assessed for safety, pharmacodynamics and pharmacokinetics in well defined moderate ASH patients cohorts receiving increasing doses of VLX103. The overall study design is characteristic for early phase, first in patient clinical evaluation of safety and pharmacodynamics, especially when prudent dose escalation is recommended. A maximum of 18 moderate ASH patients will be enrolled and treated in at least 4 clinical sites throughout the US. The open label nature of the study design will allow to efficiently monitor the safety of VLX103 throughout the trial, and taking rapid decisions about dosing adjustments (dose reduction or discontinuation within each dosing cohort, for each patient).

After an adequate screening period (Day -7 to -1), all eligible patients of the first dosing cohort will receive the initial, low dose of VLX103 150 mg per day (QD) for 14 consecutive days. Subjects will be evaluated only for safety, pharmacodynamics and pharmacokinetics during this period. Pre-established safety criteria will be used to decide upon dose escalation, for each subject. If patients tolerate well VLX103 at 150 mg QD , a second cohort will then receive a 300 mg QD regimen for 14 days. At the end of this period, safety, pharmacodynamics and pharmacokinetics will be assessed as well. The next dose will be 450 mg QD for 14 days, with the same monitoring process. Long term safety will also be assessed, at all 3 doses, at Day 30 and 90 post treatment initiation. ;

Related Conditions & MeSH terms

• Fatty Liver
• Fatty Liver, Alcoholic
• Hepatic Steatosis

• NCT number: NCT03201159
• Study type: Interventional
• Source: University of Massachusetts, Worcester
• Status: Withdrawn
• Phase: Phase 1
• Start date: June 25, 2017
• Completion date: February 27, 2018