Lab research aims to fix immune defects using Patients' own stem cells

NCT ID NCT01212055

Recruiting now Knowledge-focused Sponsor: National Cancer Institute (NCI) Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated Jun 17, 2026 · Updated 26 times

Summary

This study collects blood stem cells from people with DOCK8 deficiency, LAD-1, or GATA2 deficiency to test gene therapy in the lab. Participants receive daily injections to move stem cells into the blood, then donate cells through a procedure called apheresis. The cells are used only for research, not as a treatment. The goal is to learn how to correct the genetic defects in a test tube.

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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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Contacts and locations

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Study contacts

Contact

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

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States

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

Conditions

The condition(s) this trial relates to.

combined immunodeficiency due to DOCK8 deficiency GATA2 deficiency with susceptibility to MDS/AML inborn error of immunity leukocyte adhesion deficiency 1 monocytopenia with susceptibility to infections

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DOCK8 DOCK8 GATA2 DEFICANCY LAD-1