Keytruda takes on childhood liver cancer: new hope for relapsed patients?

NCT ID NCT04134559

Recruiting now Disease control Sponsor: Allison O'Neill, MD Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial is testing the immunotherapy drug pembrolizumab (Keytruda) in children and young adults up to age 30 with relapsed or hard-to-treat liver cancer. The goal is to see if the drug can shrink tumors or slow the cancer's growth. Researchers will also study how the immune system responds to treatment.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Pembrolizumab (Keytruda)
What this could lead to: If it works, this could point toward a new treatment option for children with liver cancer that has come back or not responded to standard therapy.
What could go wrong: This is a small, early-phase trial with only 18 participants, so results may not apply to all patients. Immunotherapy can cause side effects like inflammation in healthy organs.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

fibrolamellar hepatocellular carcinoma hepatoblastoma hepatocellular carcinoma liver cancer Liver Neoplasms pediatric hepatocellular carcinoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FIBROLAMELLAR CARCINOMA HEPATOCELLULAR CARCINOMA, CHILDHOOD LIVER CANCER LIVER CANCER, PEDIATRIC

Contacts and locations

Locations

Baylor College of Medicine
RECRUITING

Houston, Texas, 77030, United States
Children's Hospital Boston
RECRUITING

Boston, Massachusetts, 02115, United States
Cincinnati Children's Medical Center
RECRUITING

Cincinnati, Ohio, 45229, United States
Dana Farber Cancer Institute
RECRUITING

Boston, Massachusetts, 02115, United States
University of California San Francisco
RECRUITING

San Francisco, California, 94143, United States

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