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Engineered immune cells take on childhood cancers in first human trial

NCT ID NCT05650749

First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 35 times

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.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

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.

Conditions

The condition(s) this trial relates to.

neuroblastoma retinoblastoma

As listed by the trial registrant

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