Part B- Phase I/II, Non-Randomized, Single Site Study, Open-label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

Summary

Background: X-Linked Chronic Granulomatous Disease (X-CGD) is caused by a gene mutation that makes the immune system to not work properly. Researchers want to see if a lentiviral gene transfer treatment will have the ability to make the patient s immune system more normal, in particular reduce the risk of CGD related infections. The gene transfer takes a person s own stem cells, cultures them to put the normal gene in, then gives the cells back to the person. Objective: To test a gene transfer treatment for X-CGD. Eligibility: Participants aged 3-60 with X-CGD Design: Participants will be screened under protocol 05-I-0123. They will undergo: Medical history Physical exam Heart tests Imaging tests, as needed Blood tests Lung function tests, as needed Dental and audiology exams, if needed Quality of life questionnaire Bone marrow aspiration. A needle will be inserted into the hip bone or breastbone to collect bone marrow. Some screening tests will be repeated during the study. Participants will have an apheresis procedure under protocol 94-I-0073. Stem cells will be collected. Participants will get a series of drugs to prepare them for the gene transfer. Participants will stay at the NIH Clinical Center for a little over a month. They will get a central line. It is a large intravenous (IV) catheter that is placed into a vein of the neck, chest, or arm. They will get chemotherapy and their corrected stem cells through their IV line. Participants will have 12 follow-up outpatient visits in the 2 years after their gene transfer, as well as visits with their local doctor. Then they will enroll in another study for long-term follow-up visits that will last for 13 years.

Description