Last updated on February 2019

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants


Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: X-linked | Severe Combined Immunodeficiency Disease
  • Age: - 1 Years
  • Gender: Male

Inclusion Criteria:

  • Treatment Eligibility Criteria:
    • Age <2 years at the time of enrollment.
    • No prior therapy with allogeneic stem cell transplantation.
    • A clinical diagnosis of SCID-X1 documented in the medical record.
    • A proven mutation in the common gamma chain gene as defined by direct sequencing of patient DNA.
    • Age > 2 months to < 1 year of age at the time of busulfan administration.
    • Less than 300 CD3+ T-cells by flow cytometry or higher if evidence of maternal engraftment as supported by peripheral blood FISH analysis for XY and XX.
    • Lymphocyte proliferation to phytohemagglutinin (PHA) <10% of the lower limit of normal for the laboratory.

Treatment Exclusion Criteria:

  • Availability of a HLA matched sibling for allogeneic transplantation
  • Prior therapy with allogeneic stem cell transplantation
  • Positive for HIV infection by genome PCR
  • Presence of a medical condition indicating that survival will be less than 16 weeks such as the requirement for mechanical ventilation, severe failure of a major organ system, or evidence of a serious, progressive infection that is refractory to medical therapy.
  • The presence of any medical contraindications to general anesthesia and bone marrow harvest by aspiration
  • A social situation indicating that the family may not be able to comply with protocol procedures and recommended medical care.

Recruitment Status: Open


Brief Description Eligibility Contact Research Team


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