Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
Doctors at research centers in CA, MD, and MA seek patients with X-linked Chronic Granulomatous Disease caused by mutations in the CYBB gene, to trial a gene-modified stem cell therapy designed to deliver a normal copy of the CYBB gene. Study participants will undergo a blood draw treatment that sorts out specific stem cells and returns the blood to the patient. After the gene modification in lab, the stem cells are re-infused post chemotherapy and the patient will be closely assessed during treatment, with follow-up for 2 years.
- Be 23 months of age or older
- Have a diagnosis of SCID-X1 confirmed by DNA sequencing and supported by laboratory evidence for absent or reduction > 95% of the biochemical activity of the NADPH-oxidase
- Not have a 10/10 HLA-matched donor available after searching of NMDP registries
THE STUDY INVOLVES:
- Screening before the treatment
- Leukapheresis will draw the needed immune cells but return blood cells to the body
- Researchers will use a virus in the lab to deliver the gene to the immune cells
- A dose of chemotherapy will be delivered
- The gene-modified immune cells will be re-infused to the patient
- The patient will be closely assessed during treatment, with follow-up for 2 years.
LOCATIONS AND CONTACTS:
Trials will take place at three research centers:
University of California, Los Angeles (UCLA) in Los Angeles, CA. Map.
The National Institutes of Health in Bethesda, MD. Map.
Boston Children’s Hospital in Boston, MA. Map.
Caroline Y Kuo, MD | 310-794-1940 | [email protected]
Kit L Shaw, MD | 310-267-0584 | [email protected]
University of California, Los Angeles
Boston Children’s Hospital
The National Institute of Allergy and Infectious Diseases (NIAID)
California Institute for Regenerative Medicine (CIRM)
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