Actively Recruiting
Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma
Led by Allison O'Neill, MD · Updated on 2026-02-24
18
Participants Needed
5
Research Sites
373 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be younger than 30 years old at enrollment.
- Must have relapsed or refractory, histologically confirmed hepatocellular carcinoma or hepatocellular neoplasm not otherwise specified.
- Must have measurable disease by RECIST criteria with at least one lesion measurable at 20 mm by conventional methods or 10 mm by CT, MRI, or clinical exam.
- Karnofsky performance status of 60% or higher for patients 16 years or older, or Lansky score of 60% or higher for patients under 16 years.
- Must have fully recovered from acute toxic effects of all prior cancer therapies.
- No standard or targeted treatments within 14 days before starting pembrolizumab.
- No radiotherapy within 7 days before starting pembrolizumab, and radiation side effects must have resolved to grade 1.
- Must have adequate bone marrow, liver, kidney, metabolic, and thyroid function as defined by specific laboratory criteria.
- Must have insurance pre-authorization approval for pembrolizumab.
- Must be able to understand and sign informed consent; assent required for participants under 18.
- Patients with childbearing potential must agree to use effective contraception during treatment and for 4 months after.
- Women of childbearing potential need a negative pregnancy test within 24 hours before each treatment.
- A tumor sample must be available for central laboratory submission.
You will not qualify if you...
- Receiving other investigational agents.
- Previous treatment with checkpoint inhibitors such as PD-1, PD-L1, or CTLA-4 inhibitors.
- Receipt of antibody-based therapies within 3 half-lives of last dose.
- Receiving chronic steroids defined as 2 mg/kg/day or 20 mg/day prednisone or equivalent for 14 or more consecutive days.
- Receiving anti-inflammatory or immunosuppressive medications.
- Known autoimmune diseases except childhood asthma or atopic dermatitis.
- History of HIV or AIDS.
- History of allergic reactions to pembrolizumab or similar monoclonal antibody therapies, including anaphylaxis.
- Uncontrolled illness including active infections, symptomatic heart failure, or psychiatric/social conditions limiting compliance.
- Prior solid organ transplantation.
AI-Screening
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Trial Site Locations
Total: 5 locations
1
University of California San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
2
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
Actively Recruiting
3
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Actively Recruiting
4
Cincinnati Children's Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
5
Baylor College of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
D
DFCI Clinical Trials Hotline
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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