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

  • UPMC Children's Hospital of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

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