GATA2 DEFICIENCY
Clinical trials for GATA2 DEFICIENCY explained in plain language.
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Can a stem cell transplant fix a rare genetic immune disorder?
Disease control Recruiting nowThis study tests whether a stem cell transplant from a healthy donor can treat GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of leukemia. Participants aged 6 to 70 with GATA2 mutations will receive chemotherapy or radiation before t…
Matched conditions: GATA2 DEFICIENCY
Phase: PHASE2 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Scientists track rare genetic disorder to unlock its secrets
Knowledge-focused Recruiting nowThis study follows 600 people with GATA2 deficiency, a genetic disorder affecting the immune system and other body systems. Researchers aim to understand the full range of health problems it causes and why it affects people differently. Participants will have yearly check-ups for…
Matched conditions: GATA2 DEFICIENCY
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated Jul 02, 2026 00:00 UTC
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Stem cells harvested for lab tests in immune disorder gene therapy quest
Knowledge-focused Recruiting nowThis study collects blood stem cells from adults aged 18-40 with DOCK8 deficiency, LAD-1, or GATA2 deficiency. Participants receive daily injections of G-CSF for 5 days to move stem cells into the blood, then undergo a single leukapheresis procedure to collect them. The cells are…
Matched conditions: GATA2 DEFICIENCY
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:02 UTC