A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID(severe combined immune deficiency ).The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number per cell) in blood and bone marrow cells, immune reconstitution vector insertion-site patterns and so on.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Lentiviral vector to transfer IL2RG complementary DNA to patients'bone marrow stem cells
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, China
RECRUITING1-year survival rate 1-year survival rate
1-year survival rate of 10 recruited patients
Time frame: one year after gene therapy of last recruited patient
3-year survival rate
3-year survival rate of 10 recruited patients
Time frame: three years after gene therapy of last recruited patient
5-year survival rate
5-year survival rate of 10 recruited patients
Time frame: five years after gene therapy of last recruited patient
Growth velocity after gene therapy,weight in kilograms, height in meters
Body weight and height of patients will be assessed prior to (month 0) and post gene therapy,weight in kilograms, height in meters
Time frame: through study completion, an average of 2 year
Vector marking (vector copy number per cell) in blood and bone marrow cells
vector marking in T cells, B cells, NK cells, myeloid cells, and bone marrow progenitors.
Time frame: through study completion, an average of 1 year
Absolute numbers of peripheral-blood immune-cell subsets
Absolute numbers of peripheral-blood immune-cell subsets,as determined by means of standard flow cytometry
Time frame: through study completion, an average of 1 year
Quantity of DNA T-cell-receptor excision circles (TRECs) in peripheral-blood mononuclear cells
Quantity of DNA T-cell-receptor excision circles (TRECs) in peripheral-blood ,as determined by means of quantitative polymerase chain reaction (PCR)
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Time frame: through study completion, an average of 1 year
Serum immunoglobulins levels
Serum immunoglobulins levels will be reported IgM(immunoglobulin M) in mg/dL Serum immunoglobulins levels will be reported IgM in mg/dL
Time frame: through study completion, an average of 2 year
Number of patients without intravenous immune globulin supplementation
Number of patients without intravenous immune globulin supplementation after gene therapy
Time frame: through study completion, an average of 2 year
Number of patients who has a response to vaccines
Number of patients who has a response to vaccines after gene therapy
Time frame: through study completion, an average of 2 year
Number of patients who recovers from previous infection(virus and bacteria)
Number of patients who recovers from previous infection(virus and bacteria)after gene therapy
Time frame: through study completion, an average of 2 year