Stem cells harvested for lab tests in immune disorder gene therapy quest
NCT ID NCT01212055
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••