Apheresis of Patients With Immunodeficiency

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Cancer Institute (NCI)
Study ID
NCT01212055
Status
Recruiting
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Conditions

  • DOCK8
  • GATA2 Deficancy
  • LAD-1

Eligibility Criteria

Sex
ALL
Age
18 Years - 40 Years
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Nov 8, 2010
Status verified
Apr 2026

Study Design

Enrollment
7 participants (estimated)

Arms

  • Arm: Cohort 1
    Patients with DOCK8 deficiency, LAD-1, or GATA2 Deficiency

Primary Outcome Measure

To collect blood stem cells from patients with immunodeficiency diseases to test our ability to correct the defects of these cells in the test tube. [ Time Frame: 5 days ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892
Corina Gonzalez, M.D.
[email protected]
202-506-0656

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By research site
National Institutes of Health Clinical Center· Bethesda, MD

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