New stem cell processing method offers hope for patients without a perfect donor match

NCT ID NCT03145545

AVAILABLE Disease control Sponsor: Children's Hospital of Philadelphia Source: ClinicalTrials.gov ↗

First seen Jul 08, 2026 · Last updated Jul 09, 2026 · Updated 1 time

Summary

This expanded access protocol provides stem cell transplants for people with serious blood diseases like leukemia, bone marrow failure, or immune deficiencies who do not have a fully matched sibling donor. The donated stem cells are processed to remove certain immune cells (alpha/beta T cells and CD19+ cells) before transplant, which may reduce the risk of graft-versus-host disease. The goal is to offer a potentially life-saving treatment option to patients who otherwise have few alternatives.

What this could mean

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

Active substance: donor stem cells processed to remove certain immune cells
What this could lead to: If successful, this approach could provide a life-saving transplant option for patients with leukemia, bone marrow failure, or immune deficiencies who have no matched sibling donor.
What could go wrong: This is an expanded access protocol, not a formal trial, so data on safety and effectiveness are limited. Risks include graft failure, infection, and graft-versus-host disease.

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Conditions

The condition(s) this trial relates to.

acute leukemia Bone Marrow Failure Disorders bone marrow failure syndrome chronic leukemia immunodeficiency disease inborn error of immunity leukemia lymphoma myelodysplastic syndrome sickle cell disease thalassemia

As listed by the trial registrant

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

BONE MARROW FAILURE SYNDROME IMMUNODEFICIENCIES LEUKEMIA

Contacts and locations

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Study contacts

Contact

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Contact

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Locations

Children's Hospital of Philadelphia
AVAILABLE

Philadelphia, Pennsylvania, 19104, United States

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

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

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