Gentler transplant shows promise for kids with blood diseases

NCT ID NCT01050855

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

First seen Jun 26, 2026 · Last updated Jul 22, 2026 · Updated 3 times

Summary

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer side effects than standard transplants. Researchers are tracking how well the donor cells take and overall survival.

What this could mean

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

Active substance: Reduced intensity conditioning regimen (Campath, Fludarabine, Melphalan, Busulfan, Cyclosporine, Cellcept)
What this could lead to: If successful, this could offer a safer transplant option for children with non-malignant blood disorders, reducing side effects and improving long-term survival.
What could go wrong: This is a mid-stage trial with only 75 participants, so results may not apply to everyone. The regimen still carries risks like graft failure or infection.

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Conditions

The condition(s) this trial relates to.

beta thalassemia bone marrow failure syndrome common variable immunodeficiency disease hemoglobinopathy hemophagocytic syndrome immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome immunodeficiency disease inborn error of immunity inherited hemoglobinopathy Langerhans cell histiocytosis macrophage activation syndrome severe combined immunodeficiency severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency sickle cell disease Wiskott-Aldrich syndrome

As listed by the trial registrant

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

HEMOGLOBINOPATHIES IMMUNODEFICIENCIES NON MALIGNANT DISEASES

Contacts and locations

Locations

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

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