Pembrolizumab for Hepatocellular Carcinoma

University of California San Francisco, San Francisco, CA
Hepatocellular Carcinoma+3 More ConditionsPembrolizumab - Drug
No minimum age - 30
All Sexes
What conditions do you have?

Study Summary

This trial is testing an immunotherapy drug to see if it can treat pediatric liver cancer.

Eligible Conditions
  • Childhood Hepatocellular Carcinoma
  • Fibrolamellar Carcinoma
  • Liver Cancer
  • Pediatric Liver Cancer

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3
Phase 2

Study Objectives

1 Primary · 5 Secondary · Reporting Duration: 2 Years

2 Years
DNA sequencing of specimens
Expression levels of infiltrating immune cells and markers of checkpoint inhibition on pre-treatment specimens
Number of Participants with DLT
Percent change immune cell phenotype, cytokines, and circulating tumor DNA
63 Days
Immune-related best overall response (irBOR)
Month 100
Progression-free survival (PFS)

Trial Safety

Phase-Based Safety

2 of 3
This is further along than 68% of similar trials

Side Effects for

Pembrolizumab Second Course
100%Inappropriate antidiuretic hormone secretion
100%Urinary tract infection
This histogram enumerates side effects from a completed 2021 Phase 3 trial (NCT03066778) in the Pembrolizumab Second Course ARM group. Side effects include: Inappropriate antidiuretic hormone secretion with 100%, Urinary tract infection with 100%, Parkinsonism with 100%.

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

1 Treatment Group

1 of 1

Experimental Treatment

18 Total Participants · 1 Treatment Group

Primary Treatment: Pembrolizumab · No Placebo Group · Phase 2

Experimental Group · 1 Intervention: Pembrolizumab · Intervention Types: Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 2 years

Who is running the clinical trial?

Allison O'NeillLead Sponsor
Allison O'Neill, MDPrincipal Investigator - Dana-Farber Cancer Institute
Dana-Farber Cancer Institute
2 Previous Clinical Trials
150 Total Patients Enrolled

Eligibility Criteria

Age No minimum age - 30 · All Participants · 0 Total Inclusion Criteria

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Frequently Asked Questions

Might I be eligible to take part in this research project?

"This trial is seeking 18 participants who have hepatocellular carcinoma, and who were diagnosed with the condition in childhood. Those that wish to join should be between 0 and 30 years old." - Anonymous Online Contributor

Unverified Answer

What condition has seen the greatest improvement with Pembrolizumab?

"Pembrolizumab is an effective treatment for malignant neoplasms, unresectable melanoma, and microsatellite instability high." - Anonymous Online Contributor

Unverified Answer

At how many different locations is this trial currently taking place?

"Presently, there are 5 sites where this study is taking place: Baylor College of Medicine (Houston), Dana Farber Cancer Institute (Boston), Children's Hospital Boston (Cincinnati), and other locations." - Anonymous Online Contributor

Unverified Answer

What are Pembrolizumab's long-term effects on patients?

"Pembrolizumab has received a Phase 2 rating, meaning that while there is some evidence suggesting it is safe, there is no data to support its efficacy. Consequently, our team at Power have given it a safety score of 2." - Anonymous Online Contributor

Unverified Answer

How many people are allowed to enroll in this research project?

"In order to properly run this trial, 18 willing and eligible participants are required. These individuals can visit either of the two main study locations - Baylor College of Medicine in Houston or Dana-Farber Cancer Institute in Boston - though other recruitment sites may be available." - Anonymous Online Contributor

Unverified Answer

What other studies has Pembrolizumab been a part of in the past?

"City of Hope conducted the first clinical trial for pembrolizumab in 2010 and there are now 249 completed studies. Out of the 1000+ trials currently underway, many are being performed in Houston, Texas." - Anonymous Online Contributor

Unverified Answer

Are there any positions available for participants in this research project?

"Yes, this trial is still recruiting patients according to the most recent update on from September 8th, 2020. The study was first posted on November 1st, 2020." - Anonymous Online Contributor

Unverified Answer

Can patients below the age of 45 years old participate in this research?

"According to the inclusion criteria for this trial, qualified applicants are aged 0 to 30. There are currently 126 studies recruiting patients under 18 and 3813 for people over 65." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.