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Chronic granulomatous disease - Healing Genes
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Chronic granulomatous disease

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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


Phase 1 / 2

DESCRIPTION:

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.


PATIENT MUST:

  • 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:

  1. Screening before the treatment
  2. Leukapheresis will draw the needed immune cells but return blood cells to the body
  3. Researchers will use a virus in the lab to deliver the gene to the immune cells
  4. A dose of chemotherapy will be delivered
  5. The gene-modified immune cells will be re-infused to the patient
  6. 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.

 

Contacts:

Caroline Y Kuo, MD  |  310-794-1940  |  [email protected]

Kit L Shaw, MD  |  310-267-0584  |  [email protected]

 

 

SPONSOR INFORMATION:

University of California, Los Angeles

Boston Children’s Hospital

The National Institute of Allergy and Infectious Diseases (NIAID)

Genethon

California Institute for Regenerative Medicine (CIRM)

 

Or go online:

https://clinicaltrials.gov/ct2/show/NCT02234934

 

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