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X-Linked Severe Combined Immunodeficiency (LVXSCID-OC) - Healing Genes
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X-Linked Severe Combined Immunodeficiency (LVXSCID-OC)

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Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency (LVXSCID-OC)

Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency


Phase 1 / 2

DESCRIPTION:

The National Institutes of Health Clinical Center is recruiting patients with X-Linked Severe Combined Immunodeficiency (LVXSCID-OC) to trial a gene-modified stem cell therapy that uses the patient’s own (autologous) blood or bone marrow stem cells, genetically editing them in the lab using a virus, then safely returning them to the patient via a single infusion. These gene-edited stem cells with corrected IL2RG genes may produce the protein necessary for the growth and maturation of immune system cells called lymphocytes.

The treatment will require bone marrow harvest surgery, then after the targeted cells are gene-edited in the lab, then later infused after mild chemotherapy treatment. Participants will be followed frequently first 2 years, and less frequently thereafter. Long-term follow up may last 15 years.


PATIENT MUST:

  • Be 2 to 40 years of age
  • Have a proven mutation in the common gamma chain gene as defined by direct sequencing of patient DNA
  • Not have an available HLA matched sibling donor as determined before enrollment
  • Have an expected survival of at least 120 days.

THE STUDY INVOLVES:

  1. Prescreening tests to confirm eligibility of the patient to participate.
  2. Outpatient surgery to withdraw bone marrow stem cells from the iliac crest (ridge of hip).
  3. No treatment while the cells are gene-edited in the lab.
  4. Patient will admit to the hospital.
  5. Busulfan treatment (chemotherapy) for several days prior to the infusion of the modified stem cells.
  6. Stem cell transfusion with close monitoring.
  7. Participants will be followed frequently first 2 years, and less frequently thereafter. Long-term follow up may last 15 years.

LOCATIONS AND CONTACTS:
The study site is at the National Institutes of Health Clinical Center. Map.

 

Contacts:
Suk See DeRavin, M.D., Ph.D     |     301-496-6772 (301) 443-8628

 

SPONSOR INFORMATION:
National Institute of Allergy and Infectious Diseases (NIAID)

 

Or go online:
https://clinicaltrials.gov/ct2/show/NCT03315078

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