Liver injury from checkpoint inhibitors

Checkpoint Inhibitor-induced Liver Injury Study (ChILI)

Observational University of Nottingham · NCT04476563

This project will test whether specific blood, genetic, immune, and liver tissue markers can predict who gets liver injury from checkpoint inhibitors by following people starting these drugs and those who develop liver injury.

Quick facts

Study typeObservational
Enrollment160 (estimated)
Ages18 Years and up
SexAll
SponsorUniversity of Nottingham Academic / other
Drugs / interventionschemotherapy
Locations1 site (Nottingham)
Trial IDNCT04476563 on ClinicalTrials.gov

What this trial studies

This multicenter prospective observational project will enroll two deeply phenotyped cohorts: patients who develop checkpoint inhibitor–induced liver injury (ChILI) and patients starting checkpoint inhibitor therapy. Investigators will collect blood, genetic material, immune profiling data, and liver tissue where available at multiple time points and compare features between groups. The study will determine incidence, identify clinical and host risk factors, and characterize biochemical, immunological, genetic, and histological signatures of ChILI. These data aim to support development of algorithms to better predict and manage checkpoint inhibitor–related hepatotoxicity.

Who should consider this trial

Good fit: Ideal participants are adults starting immune checkpoint inhibitor therapy or those who have developed checkpoint inhibitor–related liver injury (meeting lab criteria such as ALT >5×ULN, or ALT >3×ULN with bilirubin >2×ULN, or ALP >2×ULN with GGT rise and no bone cause) who can give consent or have an appropriate consultee if incapacitated.

Not a fit: Patients whose liver abnormalities are explained by other known causes (for example viral hepatitis, metastatic liver disease, or other drug-induced liver injury) or who cannot provide required samples are unlikely to benefit from this study.

Why it matters

Potential benefit: If successful, the project could help predict and prevent severe checkpoint inhibitor–related liver damage by guiding monitoring and earlier treatment.

How similar studies have performed: Previous research on immune-related adverse events has suggested some biomarkers, but prospective, deeply phenotyped cohorts focused specifically on checkpoint inhibitor–induced liver injury remain limited and this approach is relatively novel.

Eligibility criteria

Show full inclusion / exclusion 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:

1. 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
2. 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).

Where this trial is running

Nottingham

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Immune-Mediated HepatitisImmune checkpoints inhibitorsImmune-related adverse eventsHepatotoxicityLiver injury
Last reviewed 2026-06-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.