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Immunodeficiency Diseases Clinical Trials


St. Jude Clinical Trials

GENEFU: Long-Term Follow-Up of Patients who have Received Gene Therapy/Gene Marked Products


This is a non-therapeutic clinical trial that is only open to St. Jude patients.

  • Participant received an integrating retroviral or lentiviral vector based gene transfer or gene marked product at St. Jude Children’s Research Hospital within the past 15 year time period.
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HSESID: Database for HLH Immunodeficiency Disease


  • Diagnosis of a primary immunodeficiency predisposed to hemophagocytic lymphohistiocytosis
  • Meets at least 4 of 8 diagnostic criteria for HLH as defined by the Histiocyte Society and is receiving HLH-directed therapy
  • High suspicion of isolated Central Nervous System-HLH and is receiving HLH-directed therapy
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LVXSCID-ND: Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) in Newly Diagnosed Infants

Diseases Treated:

X-linked Severe Combined Immunodeficiency (SCID-X1)


  • Diagnosis of Severe Combined Immunodeficiency, X-linked (SCID-X1)
  • Newborn to 2 years of age
  • No prior therapy with allogeneic stem cell transplantation
  • No HIV infection
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