Actively Recruiting

Phase 1
Age: 1Year - 26Years
All Genders
ID07148050

Immunotherapy for Pediatric and Young Adult Solid Tumors Using Interleukin-15 and -21 Enhanced Glypican-3-specific CAR T Cells

Led by Seattle Children's Hospital · Updated on 2026-02-17

21

Participants Needed

1

Research Sites

782 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating an experimental immunotherapy for children and young adults with solid tumors outside the central nervous system that express a molecule called glypican-3 (GPC3). This Phase 1 study aims to assess the safety, feasibility, and effectiveness of special immune cells called CAR T cells that have been genetically modified to target GPC3 and enhanced with genes IL-15 and IL-21 to help them grow and last longer in the body. The study also includes a safety switch gene to control the CAR T cells if needed. Participants will have blood drawn to create personalized CAR T cells programmed to attack their tumors. After confirming eligibility, participants will receive lymphodepleting chemotherapy for three days to prepare the body, followed by a single infusion of the CAR T cells at Seattle Children's Hospital. The study uses a dose escalation approach to determine the safest and most effective dose, with close monitoring for side effects during the first month after treatment. During the study, participants will have frequent visits for assessments including tumor evaluations, safety checks, and laboratory tests. After the initial month of monitoring, participants will continue follow-up visits for up to 15 years with decreasing frequency over time. Researchers will measure the ability to manufacture the CAR T cells, safety by tracking adverse events, dose limits, and changes in tumor burden to understand how the treatment works and its effects over the long term.

CONDITIONS

Brief Title

Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells

Who Can Participate

Age: 1Year - 26Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of a solid tumor expressing glypican-3 (GPC3)
  • Lansky or Karnofsky score of 60% or higher
  • Life expectancy greater than 16 weeks
  • Informed consent obtained and understood by patient or guardian
  • For hepatocellular carcinoma patients: Barcelona Liver Cancer Stage A, B, or C
  • For hepatocellular carcinoma patients: Child-Pugh Turcotte Score less than 7
  • Refractory or relapsed disease after prior treatment and at least one salvage therapy
  • Recovered from acute toxic effects of previous chemotherapy or investigational agents
  • Adequate organ function and laboratory values
  • Sexually active patients willing to use effective birth control for 12 months after infusion
Not Eligible

You will not qualify if you...

  • History of hypersensitivity to murine protein-containing products or presence of human anti-mouse antibodies if previously treated with murine antibodies
  • History of organ transplantation
  • Known HIV positive status
  • Active bacterial, fungal, or viral infection (except Hepatitis B or C)
  • Active autoimmune or inflammatory disorders
  • Live vaccines received within 30 days prior to enrollment
  • Pregnancy or lactation
  • Uncontrolled infections
  • Systemic steroid treatment equivalent to or greater than 0.5 mg/kg/day prednisone within 24 hours before CAR T cell infusion
  • Severe heart conditions such as congestive heart failure (NYHA Class III or IV), unstable angina, serious arrhythmias, recent myocardial infarction within 6 months, or history of myocarditis

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - About 1 month

Participants receive lymphodepleting chemotherapy for 3 days, followed by a single infusion of SC-CAR.GPC3xIL15.21 CAR T cells. Treatment is given at Seattle Children's Hospital with close monitoring for side effects.

Daily visits during chemotherapy and CAR T cell infusion, plus close monitoring visits for about 1 month

Follow-up

Duration - Up to 15 years

Participants are monitored for side effects and response to treatment for 15 years after CAR T cell infusion, with visits becoming less frequent over time.

Regular visits with decreasing frequency over time

Trial Site Locations

Total: 1 location

1

Seattle Children's Hospital

Seattle, Washington, United States, 98105

Actively Recruiting

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Research Team

M

Michelle Choe, MD

A

Andras Heczey, MD

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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