Clinical Trial: Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency
Brief Summary:
Background:
- X-linked severe combined immunodeficiency (XSCID) is caused by a genetic abnormality in the IL2RG gene that affects the growth and development of immune cells such as white blood cells. Individuals with XSCID have difficulty fighting infections, which may lead to chronic or severe illness and death. The primary treatment for XSCID is replacement of the patient s immune system with a normal immune system through a bone marrow transplant. The best outcomes in transplant patients are achieved when the bone marrow comes from a sibling, but parents and matching unrelated donors can provide bone marrow for transplant as well. However, because these transplant procedures are not always effective, researchers are studying gene transfer treatment as an approach to treating XSCID.
- Lentiviral gene transfer treatment uses good genes to replace defective genes. A lentivirus is a virus that has been modified to carry corrected genes into the blood through corrected stem cells. By collecting an individual s stem cells and modifying them with a lentivirus, the gene-corrected cells can be returned into the blood to help produce normal healthy immune cells. Gene transfer treatment with lentivirus vector has been used in humans but has never been studied in patients with XSCID.
Objectives:
- To determine the safety and effectiveness of lentiviral gene transfer as a treatment for children and adolescents with X-linked severe combined immunodeficiency.
Eligibility:
- Children and adolescents between 2 and 20 years of age who have XSCID related to a defect in the IL2RG gene and who are not currently under treatme
