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.
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT01212055.
Locations matching your search criteria
United States
Maryland
Bethesda
National Institutes of Health Clinical CenterStatus: Active
Contact: National Cancer Institute Referral Office
Phone: 888-624-1937
Background:
Primary immunodeficiency diseases (PID) represent candidate genetic disorders for new
therapeutic approaches. Our laboratory is developing new therapies for individuals with
PID using autologous CD34+ hematopoietic stem cells (HSC). Newer therapies may circumvent
problems with allogeneic HSC transplantation, especially graft rejection and
graft-versus-host-disease. We are particularly interested in three PID: Dedicator of
CytoKinesis-8 (DOCK8) deficiency, Leukocyte Adhesion Deficiency type 1 (LAD-1), and GATA2
Deficiency. For all three diseases the gene has been cloned. Testing new therapies for
these diseases would be considerably enhanced by the acquisition of peripheral blood
CD34+ cells from individuals with these immunodeficiency diseases.
Objectives:
To provide a source of filgrastim or filgrastim biosimilar mobilized peripheral blood
CD34+ hematopoietic stem cells (HSC) for laboratory research studies for DOCK8
deficiency, LAD-1, and GATA2 Deficiency.
Eligibility:
Individuals 18-40 years old with DOCK8 deficiency, LAD-1, and GATA2 Deficiency who meet
the eligibility requirements will be considered for this protocol.
Design:
Individuals 18-40 years old with DOCK8 deficiency, LAD-1, and GATA2 Deficiency will
receive five days of filgrastim or filgrastim biosimilar followed by a single apheresis.
CD34+ cells will be selected and frozen in aliquots by the Cell Processing Section of the
Department of Transfusion Medicine. No treatments, or investigational therapy will be
administered on this protocol.
Trial PhaseNo phase specified
Trial TypeNot provided by clinicaltrials.gov
Lead OrganizationNational Cancer Institute
Principal InvestigatorDennis D. Hickstein