Evaluating varegacestat in individuals with liver impairment

A Phase 1, Open-Label, Single-Dose Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Varegacestat in Participants with or Without Hepatic Impairment

This study is testing how a new drug called varegacestat works in adults with liver problems compared to healthy adults to see if it's safe and how the liver affects the drug.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 44 (estimated)
• Ages: 18 Years to 84 Years
• Sex: All
• Sponsor: Immunome, Inc. (Industry-sponsored)
• Locations: 2 sites (Orlando, Florida and 1 other locations)
• Trial ID: NCT06841315 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the pharmacokinetics, safety, and tolerability of varegacestat in adults with varying degrees of hepatic impairment compared to healthy individuals with normal liver function. It is an open-label, single-dose, parallel study that will enroll participants with mild and moderate hepatic impairment alongside matched healthy participants. The study may also include participants with severe hepatic impairment if deemed necessary based on initial findings. The goal is to understand how liver function affects the drug's behavior in the body.

Who should consider this trial

Good fit: Ideal candidates include healthy adults aged 18 to 84 and adults with mild to severe hepatic impairment who are clinically stable.

Not a fit: Patients with acute liver conditions or those not meeting the specified age and health criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide insights into the safe use of varegacestat in patients with liver impairment, potentially leading to better treatment options.

How similar studies have performed: Other studies evaluating pharmacokinetics in hepatic impairment have shown varying degrees of success, but this specific approach with varegacestat is novel.

Eligibility criteria

Inclusion Criteria (Healthy Participants):

* Healthy, adult, male or female (of nonchildbearing potential only), 18 to 84 years of age, inclusive, at the screening visit. Participant will be matched by age (± 10 years) and sex to a participant with hepatic impairment (HI). Each healthy participant may be matched to more than 1 participant with HI, but cannot be matched to more than 1 participant within the same HI group.
* Medically healthy with no clinically significant medical history, physical examination, clinical laboratory profiles, vital signs, and ECGs as deemed by the PI or designee

Inclusion Criteria (Hepatic Impairment):

* Healthy, apart from HI, adult male or female (of nonchildbearing potential only), 18 to 84 years of age, inclusive, at the screening visit.
* Considered to have HI (of any etiology) that has been clinically stable (no acute episodes of illness due to deterioration in hepatic function) for at least 1 month prior to dosing per the PI and Sponsor and is likely to remain stable throughout the study. To be classified as having hepatic impairment with known medical history of liver disease (with or without a known history of alcohol abuse).
* Has a score on the Child-Pugh scale at screening visit and confirmed at check-in as follows:

Mild: ≥5 and ≤6 \[Category A\]; or

Moderate: ≥7 and ≤9 \[Category B\]; or

Severe: ≥10 and ≤15 \[Category C\].

- Medically healthy, apart from HI, with no clinically significant medical history, physical examination, clinical laboratory profiles, vital signs, and ECGs as deemed by the PI or designee

Exclusion Criteria (Healthy Volunteers):

* Is mentally or legally incapacitated or has significant emotional problems at the time of the screening visit or expected during the conduct of the study.
* History or presence of clinically significant medical or psychiatric condition or disease in the opinion of the PI or designee.
* History of any illness that, in the opinion of the PI or designee, might confound the results of the study or pose an additional risk to the participant by their participation in the study.

Exclusion Criteria (Hepatic Impairment):

Participants must not be enrolled in the study, if they meet any of the following criteria:

* Is mentally or legally incapacitated or has significant emotional problems at the time of the screening visit or expected during the conduct of the study.
* History or presence of clinically significant medical (except for HI) or psychiatric condition or disease in the opinion of the PI or designee.
* History of any illness that, in the opinion of the PI or designee, might confound the results of the study or pose an additional risk to the participant by their participation in the study.

Where this trial is running

Orlando, Florida and 1 other locations

• Orlando Clinical Research Center — Orlando, Florida, United States (Recruiting)
• The Texas Liver Institute — San Antonio, Texas, United States (Recruiting)

Study contacts

• Study coordinator: Thomas Marbury, MD

Conditions Healthy Volunteer; Hepatic Impairment