RecruitingHepatocellular Carcinoma, ChildhoodFibrolamellar CarcinomaLiver Cancer
Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma
Eligible age
0–30 yrs
Accepts
All genders
Locations
4 states
Healthy volunteers
No
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About this study
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).
Sponsor: Allison O'Neill, MD
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:
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.
Where it's recruiting
Source: ClinicalTrials.gov · NCT04134559 · last updated 2026-02-24