Milder transplant method aims to help young patients with rare blood diseases
NCT ID NCT01962415
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler preparation can reduce deaths and side effects while still allowing donor cells to take hold. About 100 participants will receive a transplant from umbilical cord blood, bone marrow, or blood stem cells and be followed for at least a year.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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UPMC Children's Hospital of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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