Evaluating lepodisiran in patients with varying liver function

A Phase 1, Multicenter, Sequential-Design, Single-Dose, Open-Label Study of Lepodisiran in Participants With Normal Hepatic Function and Participants With Mild, Moderate, or Severe Hepatic Impairment

This study is testing how well a new injection called lepodisiran works in people with different levels of liver function to see how much of the drug gets into their bloodstream and how long it stays there.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 33 (estimated)
• Ages: 18 Years to 85 Years
• Sex: All
• Sponsor: Eli Lilly and Company (industry)
• Locations: 4 sites (Miami, Florida and 3 other locations)
• Trial ID: NCT06916078 on ClinicalTrials.gov

What this trial studies

This study aims to assess the pharmacokinetics of lepodisiran, a medication administered via subcutaneous injection, in participants with different levels of liver function impairment, including normal, mild, moderate, and severe cases. The trial will measure how much of the drug enters the bloodstream and the duration it remains in the body. Additionally, the study will evaluate the tolerability of lepodisiran and monitor any potential side effects experienced by participants. The duration of the study is approximately 9 weeks, excluding the screening phase.

Who should consider this trial

Good fit: Ideal candidates include individuals weighing at least 55 kg with either normal liver function or classified as having mild to severe liver impairment.

Not a fit: Patients with significant cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, or neurological disorders may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide insights into the safe use of lepodisiran for patients with liver dysfunction.

How similar studies have performed: While this specific approach is novel, similar pharmacokinetic studies have shown promise in evaluating drug safety and efficacy in patients with liver impairment.

Eligibility criteria

Inclusion Criteria:

* Have a body weight of 55 kilogram (kg) or more and body mass index within the range 19.0 to 42.0 kilogram per square meter (kg/m²)
* Additional Inclusion Criteria for Participants Without Hepatic Impairment in Group 1:

  * Healthy participants with clinically normal hepatic function
* For Participants with Mild to Severe Hepatic Impairment in Groups 2 through 4:

  * Participants with hepatic impairment classified as Child-Pugh score A, B, or C (mild, moderate, or severe impairment). Diagnosis of chronic hepatic impairment of greater than 6 months, per physician diagnosis and standard-of-care practice

Exclusion Criteria:

* Have significant history of, or current, cardiovascular (CV), respiratory, hepatic (hepatic applies to Group 1 only), renal, gastrointestinal, endocrine, hematological, or neurological disorders
* Have severe atopy or a history of clinically significant multiple or severe drug allergies
* Have known allergies to lepodisiran, related compounds, or any components of the formulation
* Have a history of, or current, psychiatric disorders
* Have had any malignancy within the past 5 years
* Have estimated glomerular filtration rate (eGFR) less than 60 milliliters per minute per 1.73 m² (mL/min/1.73 m²) using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
* Have participated, within the last 1 month, in a clinical study involving an investigational product

Where this trial is running

Miami, Florida and 3 other locations

• Clinical Pharmacology of Miami — Miami, Florida, United States (RECRUITING)
• Orlando Clinical Research Center — Orlando, Florida, United States (RECRUITING)
• American Research Corporation at Texas Liver Institute — San Antonio, Texas, United States (RECRUITING)
• CRU Early Phase Unit — Kistarcsa, Hungary (RECRUITING)

Study contacts

• Study coordinator: Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or

Conditions: Liver Dysfunction, Healthy