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Recruiting NCT04476563

Checkpoint Inhibitor-induced Liver Injury

Observational Immune-Mediated Hepatitis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Obtaining biological samples.
Who it may be relevant to
Registry conditions: Immune-Mediated Hepatitis. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Official title

Checkpoint Inhibitor-induced Liver Injury Study (ChILI)

Overview

In this multi-center prospective observational study, the investigators plan to identify the incidence and risk factors for checkpoint inhibitor-induced liver injury and characterize biochemical, genetic, immunological, and histological features associated with it.

Detailed description

Checkpoint inhibitor-induced liver injury (ChILI) is a new incompletely understood category of hepatotoxicity which is distinct from other types of drug-induced liver injury (DILI) such as direct or idiosyncratic DILI. The data regarding the incidence and risk factors is lacking. Therefore, 'in-depth phenotyping' together with data from the control group exposed to checkpoint inhibitors (CPI) is necessary to develop refined algorithms incorporating CPI-related factors, host genetic and environmental risk factors that would enable pre-empting ChILI.

The aim of the study is to enroll two deeply phenotyped cohorts (patients who developed ChILI and patients who are starting checkpoint inhibitors) and obtain biological samples at multiple time points.

Interventions

  • Diagnostic test Obtaining biological samples
    Biological samples (blood, urine, stool). Liver tissue will be obtained from ChILI group when clinically indicated

Primary outcome measures

  • Incidence of checkpoint inhibitor-induced liver injury (ChILI) and other immune-mediated adverse reactions [Time frame: 3 years]
  • Identify novel biomarkers associated with the diagnosis of ChILI [Time frame: 3 years]

Eligibility criteria

Inclusion criteria

Both patient groups and control group:

  • Able to give written informed consent OR Potential participants who have developed encephalopathy related to ChILI as a response to checkpoint inhibitor therapy, who lack the capacity to give written informed consent and have a consultee (personal or nominated) - for ChILI patient group only

ChILI group:

Patients who developed checkpoint inhibitor-induced liver injury and meet the following criteria:

  • Meets one of the following analytical thresholds at enrolment (visit 1)
  • Alanine transaminase (ALT) exceeding 5 times the upper limit of normal (ULN) OR
  • ALT exceeding 3 times ULN plus bilirubin exceeding 2 times ULN OR
  • Alkaline phosphatase (ALP) exceeding 2 times ULN with accompanying elevations of gamma-glutamyl transferase in the absence of known bone metastases driving the rise in ALP level
  • Absence of other known causes of liver injury after detailed investigations

Patients who developed ChILI but did not meet the above criteria at enrolment or who were found to have a different cause for their liver injury after further investigations will be excluded from the analysis

Control group:

Consecutive patients with cancer who have a clinical indication to start checkpoint inhibitors. A small proportion of patients will develop ChILI following their checkpoint inhibitor treatment and will be classified as cases.

Exclusion criteria

  • Patients who are treated with cytotoxic chemotherapy concurrently with checkpoint inhibitors.
  • On the judgment of chief investigator that the person has certain alternative explanations to the acute event (rather than ChILI).

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United Kingdom · 1 center
  • University of Nottingham — Nottingham

Publications

  • Ji C, Kumpf S, Qian J, Federspiel JD, Sheehan M, Capunitan D, Atallah E, Astbury S, Arat S, Oziolor E, Ocana MF, Ramaiah SK, Grove J, Aithal GP, Lanz TA. Transcriptomic and proteomic characterization of cell and protein biomarkers of checkpoint inhibitor-induced liver injury. Cancer Immunol Immunother. 2025 May 3;74(6):190. doi: 10.1007/s00262-025-04033-z. PMID 40317333

Identifiers

NCT: NCT04476563 · 20034

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗