New hope for rare immune disorder: can a transplant reset the Body's defenses?

NCT ID NCT01861106

Summary

This study is testing whether a stem cell transplant from a matched donor can treat GATA2 deficiency, a rare genetic disorder that causes serious infections and can lead to blood cancers. The transplant aims to replace the patient's faulty bone marrow with healthy donor cells to restore normal blood and immune system function. The trial is enrolling 144 participants aged 6 to 70 who have this condition and a suitable donor.

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Contacts and locations

Study contacts

  • Contact

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

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

Conditions

Explore the condition pages connected to this study.