Actively Recruiting
Apheresis of Patients With Immunodeficiency
Led by National Cancer Institute (NCI) · Updated on 2026-05-07
7
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
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 collecting stem cells from the blood of individuals with immunodeficiency diseases in order to use the cells to develop potential gene therapy treatments. Objectives: \- To collect blood stem cells from patients with immunodeficiency diseases tto test our ability to correct the defects of these cells in the test tube. Eligibility: * Individuals between 18 and 40 years of age with immunodeficiency diseases. * Individuals with human immunodeficiency virus (HIV) will not be able to participate in this study. Design: * Participants will provide an initial blood sample for disease screening (such as hepatitis B and C, syphilis, or viruses like the Epstein-Barr virus, herpes simplex virus, or toxoplasmosis) and to check kidney and liver function. * Starting 5 days before blood donation, participants will receive daily injections of a drug called G-CSF (granulocyte colony stimulating factor, or filgrastim), which pushes stem cells out of the bone marrow and into the bloodstream. Participants will receive the injections at the National Institutes of Health Clinical Center. * On day 5, participants will have a single leukapheresis procedure to collect the stem cells from the blood. * No additional treatment will be provided as part of this protocol. The cells that are collected will be used fore experiments in the lab and will not be used to treat individuals with these diseases.
CONDITIONS
Official Title
Apheresis of Patients With Immunodeficiency
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Individuals age 18-40 years
- Diagnosis of DOCK8 deficiency, LAD-1, or GATA2 Deficiency
- Serum creatinine less than 1.5 mg/dL
- Total Bilirubin less than 3 mg/dL, ALT and AST less than 5 times the upper limit of normal
- Ability to give informed consent
- Adequate venous access for peripheral apheresis or consent to use a temporary central venous catheter
- Negative urine pregnancy test within one week prior to starting filgrastim for individuals of childbearing age
You will not qualify if you...
- HIV infection
- Chronic hepatitis B or hepatitis C infection
- Psychiatric disorder affecting compliance or informed consent
- Active infection not responding to antimicrobial therapy
- Pregnant individuals
- Breastfeeding individuals
- Sexually active individuals able to become pregnant who are unwilling or unable to use effective contraception during filgrastim administration and apheresis
- Active malignancy outside the hematopoietic system
- Active pulmonary disease
- Uncontrolled hypertension, stroke, or severe heart disease including symptomatic angina
- Other medical contraindications to stem cell donation such as severe atherosclerosis, autoimmune disease, iritis or episcleritis, deep venous thrombosis, or cerebrovascular accident
- Thrombocytopenia with platelets less than 50,000 per microliter
- Receiving experimental therapy or investigational agents
- Sensitivity to filgrastim, E. Coli derived products, or biosimilar recombinant proteins
- Positive tests for transfusion-transmissible infections including hepatitis B, hepatitis C, or HIV
AI-Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
C
Corina E Gonzalez, M.D.
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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