Neoplasms

All Conditions

Digestive System Diseases

Rare Diseases

Carcinoma

Carcinoma, Hepatocellular

Liver Neoplasms

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Adenocarcinoma

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Liver Diseases

Fibrolamellar Carcinoma

Childhood Hepatocellular Carcinoma

Antineoplastic Agents

All Drugs and Chemicals

Hematinics

Pembrolizumab

Liver Extracts

Antineoplastic Agents, Immunological

Immune Checkpoint Inhibitors

Pembrolizumab will be administered every 3 weeks at a dose of 2mg/kg/dose (max: 200mg) with 21 consecutive days defined as a treatment cycle.

Pembrolizumab will be administered every 3 weeks, at predetermined dose with 21 consecutive days defined as a treatment cycle.

DFCI Clinical Trials Hotline

Arun Rangaswami, MD

Allison O'Neill, MD

James Geller, MD

Jennifer Foster, MD, MPH

This research study is studying an immunotherapy drug (pembrolizumab or KEYTRUDA) as a possible treatment for pediatric hepatocellular carcinoma or hepatocellular neoplasm not otherwise specified (HCN NOS).

Patients who fulfill eligibility criteria will be entered into the trial to receive pembrolizumab or KEYTRUDA

This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. "Investigational" means that the drug is being studied. In the case of this trial, the investigators are studying whether pembrolizumab can treat pediatric hepatocellular carcinoma.

The FDA (the U.S. Food and Drug Administration) has not approved pembrolizumab for your specific disease but it has been approved for other uses in adults. Checkpoint inhibitors are in early-phase study in pediatric patients across diagnoses.

In this research study, the investigators plan to investigate whether pediatric patients with hepatocellular carcinoma experience stable disease or response to pembrolizumab. In addition, the investigators would like to explore different biological factors of the tumor and immune system that might help us predict whether pediatric patients with HCC may benefit from treatment with pembrolizumab.

Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma

The Role of Checkpoint Inhibition in Relapsed/Refractory Pediatric Hepatocellular Carcinoma: Clinical Efficacy and Biologic Correlates - A Phase II Study

The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Expression levels of infiltrating immune cells and markers of checkpoint inhibition, as assessed by immunohistochemistry on pre-treatment specimens, quantified using a semi-quantitative scoring system based on percent of cells showing positive staining.

organ affected or laboratory determination, severity (by NCI CTCAE v5.0), and attribution.

Descriptive analysis of gene mutation patterns correlating with disease response to checkpoint inhibition

Spots Global Cancer Trial Database for hepatocellular carcinoma childhood