Gentler transplant offers hope for kids with rare, Life-Limiting diseases

NCT ID NCT01962415

Summary

This study is testing a less intense, potentially safer stem cell transplant procedure for children and young adults with serious inherited disorders like sickle cell disease, immune deficiencies, and metabolic diseases. The goal is to see if using lower doses of chemotherapy before the transplant can reduce severe side effects and organ damage while still allowing the donor cells to work. Researchers will track survival, recovery, and long-term health in about 100 participants.

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Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • UPMC Children's Hospital of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

    Contact

    Contact

    Contact

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

Conditions

Explore the condition pages connected to this study.