Apheresis of Patients With Immunodeficiency
Recruiting
Observational Study
LAD-1
DOCK8
GATA2 Deficancy
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 18 – 40
- Sex
- Any
- Study type
- Observational
- Participants needed
- 7 (estimated)
- Sponsor
- National Cancer Institute (NCI) · NIH
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About This Trial
Background:
\- Gene therapy is being investigated as a possible treatment for individuals with immunodeficiency diseases or other conditions that make it difficult to fight off infection. Gene therapy avoids problems with donor identification and possible rejection of bone marrow transplant by using the patient s own modified blood cells to help treat the disease. Researchers are interested in co…
Trial Locations
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Eligibility Criteria
-INCLUSION CRITERIA - PATIENT:
1. Individuals age 18-40 years.
2. Diagnosis of DOCK8 deficiency, LAD-1, or GATA2 Deficiency:
-DOCK8 deficiency
* Homozygous or compound heterozygous mutations in the DOCK8 gene.
-LAD-1
* Less than 10% CD18 expression on the neutrophil surface.
-…
Contacts