New transplant method aims to reduce complications in bone marrow failure patients

NCT ID NCT03047746

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

Summary

This study tests a stem cell transplant method for people with acquired or inherited bone marrow failure. Donor stem cells are specially processed to remove certain immune cells, which may lower the risk of graft rejection and graft-versus-host disease. The goal is to see if this approach improves engraftment and survival.

What this could mean

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

Active substance

TCR alpha/beta T cell-depleted peripheral blood stem cells

What this could lead to

If successful, this approach could provide a safer transplant option for patients with bone marrow failure, reducing risks of graft failure and graft-versus-host disease.

What could go wrong

This is an early pilot study with a small number of participants. Risks include graft failure, infections, and graft-versus-host disease despite the cell depletion.

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.

acquired aplastic anemia bone marrow failure syndrome Congenital Bone Marrow Failure Syndromes dyskeratosis congenita Fanconi anemia Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria Shwachman-Diamond 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