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).
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.
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.
Single group
Pembrolizumab
DrugPembrolizumab will be administered every 3 weeks, at predetermined dose with 21 consecutive days defined as a treatment cycle.
Immune-related best overall response (irBOR)
irRECIST criteria
Progression-free survival (PFS)
irRECIST criteria
Expression levels of infiltrating immune cells and markers of checkpoint inhibition on pre-treatment specimens
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.
Percent change immune cell phenotype, cytokines, and circulating tumor DNA
summarized using descriptive statistics.
Number of Participants with DLT
organ affected or laboratory determination, severity (by NCI CTCAE v5.0), and attribution.
Click on the lead sponsor to view all of their trials.
Allison O'Neill, MD
Lead sponsor
Dana-Farber Cancer Institute
Sponsor institution
