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Newly Diagnosed, Infant X-Linked Severe Combined Immunodeficiency (LVXSCID-ND) - Healing Genes
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Newly Diagnosed, Infant X-Linked Severe Combined Immunodeficiency (LVXSCID-ND)

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Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants (LVXSCID-ND)

A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells


Phase 1 / 2

DESCRIPTION:

Researchers in San Francisco, CA, Memphis, TN, and Seattle, WA, seek newly diagnosed infants with X-Linked Severe Combined Immunodeficiency (LVXSCID-ND) to trial a gene-modified stem cell therapy that uses the patient’s own (autologous) 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 gamma-chain gene may produce the protein necessary for the growth and maturation of immune system cells called lymphocytes, and thus normal immune function

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 up to 24 months of age and male
  • 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

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 10 years.

LOCATIONS AND CONTACTS:

The study sites are at the University of California-San Francisco in CA, Map, St. Jude Children’s Research Hospital in Memphis, TN, Map, and at Seattle Children’s Research Institute in Seattle, WA, Map.
 
Contacts:
Stephen Gottschalk, MD  |  901-595-2166  |  [email protected]
Contact: Ewelina Mamcarz, MD  |  901-595-8343  |  [email protected]
 
SPONSOR INFORMATION:
St. Jude Children’s Research Hospital
National Heart, Lung, and Blood Institute (NHLBI)
Assisi Foundation
California Institute for Regenerative Medicine (CIRM)
 
Or go online:
https://clinicaltrials.gov/ct2/show/NCT01512888

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