New stem cell approach could fix immune systems in kids

NCT ID NCT02990819

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase II trial tests a stem cell transplant from unrelated or partially matched family donors for children and young adults with severe immune deficiencies. The stem cells are specially processed to remove certain immune cells, aiming to reduce complications. The goal is to see if this approach leads to stable donor cell engraftment and improved survival.

What this could mean

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

Active substance

stem cell transplant with alpha/beta T and CD19+ cell depletion

What this could lead to

If successful, this could provide a safer way to restore immune function in children with life-threatening immune disorders using donor stem cells.

What could go wrong

This is an early Phase II trial with only 60 participants, so results may not apply to everyone. Risks include graft failure, graft-versus-host disease, and infection.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

chronic granulomatous disease hemophagocytic syndrome hyper-IgM syndrome immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome immunodeficiency disease inborn error of immunity Langerhans cell histiocytosis macrophage activation syndrome severe combined immunodeficiency Wiskott-Aldrich syndrome X-linked lymphoproliferative syndrome

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