Status:
SUSPENDED
Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
Lead Sponsor:
St. Jude Children's Research Hospital
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Assisi Foundation
Conditions:
Severe Combined Immunodeficiency Disease, X-linked
Eligibility:
MALE
Up to 24 years
Phase:
PHASE1
PHASE2
Brief Summary
SCID-X1 is a genetic disorder of blood cells caused by DNA changes in a gene that is required for the normal development of the human immune system. The purpose of this study is to determine if a new ...
Detailed Description
Bone marrow CD34+ cells will be obtained in the operating room, transduced with the lentiviral vector that contains a normal copy of the γc gene, and reinfused without any myeloreductive conditioning....
Eligibility Criteria
Inclusion
- \* 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
- 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.
Key Trial Info
Start Date :
August 17 2016
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2034
Estimated Enrollment :
28 Patients enrolled
Trial Details
Trial ID
NCT01512888
Start Date
August 17 2016
End Date
August 1 2034
Last Update
November 10 2025
Active Locations (3)
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1
University of California-San Francisco
San Francisco, California, United States, 94158
2
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
3
Seattle Children's Research Institute
Seattle, Washington, United States, 98101