Engineered immune cells take on childhood cancers in first human trial
NCT ID NCT05650749
First seen Jun 27, 2026 · Last updated Aug 14, 2026 · Updated 1 time
Summary
This early-stage trial is testing a new treatment called GPC2 CAR T cells for children with neuroblastoma or retinoblastoma that has come back or not responded to standard treatments. The therapy uses a patient's own immune cells, which are modified in a lab to better recognize and attack cancer cells. The main goal is to find a safe dose and understand side effects in up to 45 participants.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- GPC2 CAR T cells (a type of immune cell therapy)
- What this could lead to
- If this works, it could point toward a new treatment option for children with advanced neuroblastoma or retinoblastoma that has not responded to standard therapies.
- What could go wrong
- This is a very early, first-in-human Phase 1 trial with only 45 participants, so safety and effectiveness are not yet known. There is a risk of serious side effects, and the therapy may not work for everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Newborn screening study aims to catch rare diseases at birth