Partial match, full hope: new transplant approach aims to cure rare immune disease

NCT ID NCT03910452

First seen Nov 01, 2025 · Last updated May 07, 2026 · Updated 27 times

Summary

This early-phase study tests whether a bone marrow transplant from a partially matched relative can safely cure chronic granulomatous disease (CGD), a condition causing severe infections and inflammation. Up to 4 participants aged 4-65 will receive chemotherapy, radiation, and immune-suppressing drugs before and after the transplant to help their bodies accept the new cells. The goal is to achieve long-term donor cell engraftment without severe graft-versus-host disease.

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Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

Conditions

Explore the condition pages connected to this study.