Stem cells harvested for lab tests in immune disorder gene therapy quest
NCT ID NCT01212055
First seen Jun 27, 2026 · Last updated Jul 17, 2026 · Updated 6 times
Summary
This 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 used only for laboratory research to test gene correction, not for treating patients.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- G-CSF (filgrastim) injections and leukapheresis
- What this could lead to
- If successful, this research could help develop gene therapies for DOCK8 deficiency, LAD-1, and GATA2 deficiency.
- What could go wrong
- This is a very early, small study (7 people) focused only on cell collection for lab experiments, not treatment. It may not lead to a therapy.
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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
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
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