AI-Guided First-Line Immunotherapy Selection in Unresectable Hepatocellular Carcinoma
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18+
SponsorTongji Hospital
About this trial
This pragmatic, multicenter, cluster-randomized trial will evaluate whether a locked artificial intelligence (AI) clinical decision-support system can improve outcomes by helping multidisciplinary teams select first-line immune checkpoint inhibitor (ICI)-based systemic treatment for adults with unresectable hepatocellular carcinoma (HCC).
Twenty-six hospitals or independent HCC multidisciplinary teams will be randomly assigned in a 1:1 ratio to AI-assisted treatment selection or usual-care treatment selection. Approximately 1,800 participants will be enrolled. Eligible participants must already be considered suitable for first-line ICI-based systemic therapy; the study does not compare immunotherapy with no immunotherapy.
At AI-assisted sites, the system will use prespecified pretreatment information to estimate and compare expected outcomes across clinically appropriate, locally available, guideline-concordant ICI-based regimens. The AI output is advisory. Treating clinicians and patients retain responsibility for the final treatment decision, and reasons for not following an AI recommendation will be recorded. At usual-care sites, treatment will be selected through the standard multidisciplinary decision-making process without access to the AI output. Both groups will receive approved standard-of-care treatments.
The AI model, input definitions, preprocessing pipeline, decision rules, thresholds, and software version will be locked before enrollment of the first participant and will not be retrained or modified using trial outcome data. Both groups will use the same eligibility criteria, patient-registration time point, imaging schedule, follow-up schedule, and outcome definitions.
The primary outcome is progression-free survival assessed by blinded independent central imaging review. Overall survival is a key secondary outcome. Additional outcomes include tumor response, duration of response, safety, quality of life, treatment delivery, and implementation measures. This trial evaluates the clinical utility of a prespecified AI system rather than developing or optimizing another prediction model.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age 18 years or older.
Hepatocellular carcinoma confirmed by histology or cytology, or diagnosed using accepted noninvasive radiologic criteria.
Unresectable hepatocellular carcinoma for which first-line systemic therapy is indicated, including Barcelona Clinic Liver Cancer stage B disease that is unsuitable for or no longer benefiting from locoregional therapy, or stage C disease.
No prior systemic anticancer therapy for unresectable hepatocellular carcinoma.
Disqualifiers
Known combined hepatocellular-cholangiocarcinoma or another primary liver malignancy other than hepatocellular carcinoma.
A contraindication or clinical condition that makes all protocol-specified immune checkpoint inhibitor-based first-line regimens inappropriate according to applicable prescribing information and routine clinical practice.
Initiation of systemic therapy or completion of the final first-line regimen decision before prospective participant registration.
Exposure of the treating clinical team to the participant-specific AI recommendation before confirmation of eligibility and registration.
Trial design
Design model
Parallel
Treatments tested in this trial
AI-Assisted Clinical Decision Support Strategy
Other interventionA locked AI clinical decision-support system will analyze prespecified pretreatment information and provide the multidisciplinary team with patient-specific estimates and a comparative ranking of clinically appropriate first-line ICI-based treatment options. The recommendation is advisory and does not replace clinical judgment or shared decision-making.
Usual-Care Multidisciplinary Treatment-Selection Strategy
Other interventionFirst-line ICI-based treatment will be selected through the hospital's usual multidisciplinary decision-making process without access to the AI clinical decision-support system. Participants will otherwise undergo the same eligibility assessment, follow-up schedule, and outcome ascertainment as participants in the AI-assisted arm.
Treatment groups
1,800 Participants
are divided into 2 treatment groupsGroup A: AI-Assisted Treatment SelectionExperimental treatment 1 intervention
Group B: Usual-Care Treatment SelectionActive comparator 1 intervention
Trial outcomes
Primary outcomes
1
Progression-Free Survival Assessed by Blinded Independent Central Review Using RECIST Version 1.1
Progression-free survival is defined as the time from prospective participant registration to the first radiographically documented disease progression according to RECIST version 1.1, as determined by blinded independent central review, or death from any cause, whichever occurs first. Participants without either event will be censored at the date of their last adequate radiographic tumor assessment. Imaging assessments will follow the same prespecified schedule in both study groups.
Time frame
From participant registration until radiographic disease progression or death from any cause, whichever occurs first, assessed up to 27 monthsSecondary outcomes
1
Overall Survival
Overall survival is defined as the time from prospective participant registration to death from any cause. Participants who are alive or whose survival status is unknown at the time of analysis will be censored at the date they were last known to be alive. Vital status will be ascertained using harmonized follow-up procedures in both study groups.
Time frame
From participant registration until death from any cause, assessed up to 44 months2
Objective Response Rate Assessed by Blinded Independent Central Review Using RECIST Version 1.1
Objective response rate is defined as the proportion of participants with measurable disease at baseline who achieve a confirmed best overall response of complete response or partial response according to RECIST version 1.1, as determined by blinded independent central review. Responses must be confirmed by a subsequent assessment at least 4 weeks after initial documentation. Participants without an adequate post-baseline tumor assessment or without a confirmed response will be classified as non-responders.
Time frame
From participant registration through the last tumor assessment before disease progression or initiation of new anticancer therapy, assessed up to 27 months3
Duration of Response
Among participants who achieve a confirmed complete response or partial response, duration of response is defined as the time from the first documented response that is subsequently confirmed to the first radiographically documented disease progression according to RECIST version 1.1, as determined by blinded independent central review, or death from any cause, whichever occurs first. Participants without either event will be censored at the date of their last adequate radiographic tumor assessment before initiation of new anticancer therapy.
Time frame
From the first documented response that is subsequently confirmed until radiographic disease progression or death from any cause, assessed up to 27 months4
Disease Control Rate
Disease control rate is defined as the proportion of participants with measurable disease at baseline whose best overall response is a confirmed complete response, confirmed partial response, or stable disease maintained for at least 6 weeks after participant registration according to RECIST version 1.1, as determined by blinded independent central review. Participants without an adequate post-baseline tumor assessment or without documented disease control will be classified as not achieving disease control.
Time frame
From participant registration through the last tumor assessment before disease progression or initiation of new anticancer therapy, assessed up to 27 monthsSponsors and contacts
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Source ClinicalTrials.gov
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