Engineered immune cells take on childhood brain cancer in first human trial

NCT ID NCT04903080

Ongoing Disease control Sponsor: Children's Oncology Group Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests a new treatment for children whose ependymoma brain tumor has come back or not responded to standard therapy. The treatment uses the patient's own immune cells, modified in a lab to recognize and attack tumor cells that carry a protein called HER2. The main goals are to check if the treatment is safe and if it can be delivered across multiple hospitals.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: HER2 CAR T cells (a type of immune cell therapy made from the patient's own blood, engineered to target HER2 on tumor cells)
What this could lead to: If this treatment proves safe and effective, it could offer a new option for children with ependymoma that has not responded to standard treatments.
What could go wrong: This is an early Phase 1 trial with only 50 participants, focused mainly on safety. The treatment may not shrink tumors or may cause serious side effects. It is too early to know if it will work.

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Conditions

The condition(s) this trial relates to.

ependymoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

EPENDYMOMA

Contacts and locations

Locations

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
Children's Healthcare of Atlanta

Atlanta, Georgia, 30329, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital Los Angeles

Los Angeles, California, 90026, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Pittsburgh Children's Hospital of UPMC

Pittsburgh, Pennsylvania, 15224, United States
Texas Children's Cancer Center

Houston, Texas, 77030, United States
The Hospital for Sick Children

Toronto, Ontario, M5G1X8, Canada
University of Florida

Gainesville, Florida, 32608, United States

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