RecruitingNCT01212055

Apheresis of Patients With Immunodeficiency

Apheresis and CD34+ Selection of Mobilized Peripheral Blood CD34+ Cells From Patients With DOCK8 Deficiency, LAD-1, and GATA2 Deficiency


Sponsor

National Cancer Institute (NCI)

Enrollment

7 participants

Start Date

Nov 8, 2010

Study Type

OBSERVATIONAL

Conditions

Summary

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.


Eligibility

Min Age: 18 YearsMax Age: 40 Years

Plain Language Summary

Simplified for easier understanding

This study collects stem cells from people with certain rare immune deficiency diseases — DOCK8 deficiency, Leukocyte Adhesion Deficiency type 1 (LAD-1), and GATA2 deficiency — using a process called apheresis. Participants receive a growth factor injection (filgrastim) to mobilize stem cells from the bone marrow into the bloodstream before collection. These stem cells are used for research to better understand and potentially treat these conditions. You may be eligible if... - You are between 18 and 40 years old - You have a confirmed diagnosis of DOCK8 deficiency, LAD-1, or GATA2 deficiency - Your kidney and liver function are within acceptable levels - You do not have HIV, active hepatitis B or C, or a history of serious heart conditions - You are not pregnant and agree to use effective contraception You may NOT be eligible if... - You have HIV infection, hepatitis B or C - You have an active infection not responding to treatment - You are pregnant or breastfeeding - You have active pulmonary disease or uncontrolled hypertension - Your platelet count is below 50,000/microliter - You are sensitive to filgrastim (G-CSF) or its components Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.


Locations(1)

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT01212055