Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma

Recruiting now · Phase 2

Conditions studied: Hepatocellular Carcinoma, Childhood, Fibrolamellar Carcinoma, Liver Cancer, Liver Cancer, Pediatric

In brief

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).

Key facts

Study ID

NCT04134559

Run by

Allison O'Neill, MD

People needed

18

Starts

2020-11-01

Expected to finish

2028-01-01

Last updated by the study team

2026-02-24

Who can join

Age: any, up to 30. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age: Patients must be <30 years of age at the time of study enrollment.
• Diagnosis: Patients must have relapsed/refractory, histologically confirmed HCC to be eligible for enrollment. Patients with hepatocellular neoplasm not otherwise specified (HCN NOS) will also be eligible.
• Disease Status: Participants must have measurable disease by RECIST criteria, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded for non-nodal lesions and short axis for nodal lesions) measured at ≥20 mm with conventional technique or ≥10 mm with spiral CT scan, MRI, or calipers by clinical exam. See Section 10 for the evaluation of measurable disease.
• Performance Level: Karnofsky performance status ≥ 60% for patients ≥ 16 years of age or Lansky ≥ 60% for patients < 16 years of age.
• Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy.
• Patients must not have received standard or targeted treatment regimens within 14 days of initiation of treatment with pembrolizumab.
• Patients must not have received prior radiotherapy within 7 days of initiation of treatment with pembrolizumab. Patients who have experienced radiation-induced adverse events must recover to a grade 1 prior to enrollment.
• Organ Function Requirements: Participants must have normal organ and marrow function as defined below:
• Adequate Bone Marrow Function defined as:
• Peripheral absolute neutrophil count (ANC) ≥ 750/μL
• Platelet count ≥ 75,000/μL (can be transfused)
• Adequate Liver Function defined as:
• Total bilirubin < 1.5 x institutional upper limit of normal (ULN)
• AST(SGOT) ≤ 2.5 x ULN
• ALT(SGPT) ≤ 2.5 x ULN
• If liver function studies are more elevated than the thresholds above, and if if this elevation is felt secondary to tumor, patients may still be eligible for enrollment after discussion with the study PI.
• Adequate Renal and Metabolic Function defined as:
• A serum creatinine based on age/gender as follows:
• Age Maximum Serum Creatinine (mg/dL) Male Female
• 1 to <2 years 0.6 0.6
• 2 to <6 years 0.8 0.8
• 6 to <10 years 1.0 1.0
• 10 to <13 years 1.2 1.2
• 13 to <16 years 1.5 1.4
• >16 years 1.7 1.4

You may not qualify if…

• Participants who are receiving any other investigational agents are not eligible.
• Participants who have received checkpoint inhibitors (PD-1, PD-L1, and CTLA-4 inhibitors) are not eligible.
• Participants who have received antibody-based therapies are not eligible if they are within 3 half-lives of receipt of the last antibody dose.
• Participants who are receiving chronic steroids are not eligible.Chronic steroids are defined as either > or = 2mg/kg/day of body weight or > or = 20mg/day of prednisone or equivalent for persons who weigh > or = 10kg administered for > or = 14 consecutive days.
• Participants who are receiving anti-inflammatory or immunosuppressive medications are not eligible.
• Participants with known autoimmune disease, with the exceptions of childhood asthma or atopic dermatitis, are not eligible.
• Patients with a history of a positive test for human immunodeficiency virus or acquired immunodeficiency syndrome are not eligible.
• Patients with a history of allergic reactions attributed to compounds of similar chemical or biologic composition to pembrolizumab are not eligible. History of severe allergy to monoclonal antibody therapies (i.e. anaphylaxis) are likewise an exclusion.
• Patients with uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, or psychiatric illness/social situations that would limit compliance with study requirements are not eligible.
• Patients with prior solid organ transplantation are not eligible.

Where it is running

• University of California San Francisco — San Francisco, California, United States (enrolling)
• Children's Hospital Boston — Boston, Massachusetts, United States (enrolling)
• Dana Farber Cancer Institute — Boston, Massachusetts, United States (enrolling)
• Cincinnati Children's Medical Center — Cincinnati, Ohio, United States (enrolling)
• Baylor College of Medicine — Houston, Texas, United States (enrolling)

Full record on ClinicalTrials.gov

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