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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental Group | Experimental | 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）. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Lentiviral Vector Gene Therapy | Device | Lentiviral vector to transfer IL2RG complementary DNA to patients'bone marrow stem cells |
|
| Measure | Description | Time Frame |
|---|---|---|
| 1-year survival rate 1-year survival rate | 1-year survival rate of 10 recruited patients | one year after gene therapy of last recruited patient |
| 3-year survival rate | 3-year survival rate of 10 recruited patients | three years after gene therapy of last recruited patient |
| 5-year survival rate | 5-year survival rate of 10 recruited patients | five years after gene therapy of last recruited patient |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | through study completion, an average of 2 year |
| Vector marking (vector copy number per cell) in blood and bone marrow cells |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Xiaodong Zhao, PHD | Contact | 18623070626 | zhaoxd530@aliyun.com | |
| Qiling Xu, MD | Contact | 18581059910 | 272864835@qq.com |
| Name | Affiliation | Role |
|---|---|---|
| Xiaodong Zhao, PHD | Assistant President of Children's Hospital of Chongqing Medical University | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Chongqing Medical University | Recruiting | Chongqing | Chongqing Municipality | 400014 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30995372 | Background | Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408. | |
| 20660403 |
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| ID | Term |
|---|---|
| D053632 | X-Linked Combined Immunodeficiency Diseases |
| ID | Term |
|---|---|
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016511 | Severe Combined Immunodeficiency |
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vector marking in T cells, B cells, NK cells, myeloid cells, and bone marrow progenitors. |
| 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 | 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) | 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 | 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 | 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 | 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 | through study completion, an average of 2 year |
| Background |
| Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, Martinache C, Rieux-Laucat F, Latour S, Belohradsky BH, Leiva L, Sorensen R, Debre M, Casanova JL, Blanche S, Durandy A, Bushman FD, Fischer A, Cavazzana-Calvo M. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2010 Jul 22;363(4):355-64. doi: 10.1056/NEJMoa1000164. |
| 27099176 | Background | De Ravin SS, Wu X, Moir S, Anaya-O'Brien S, Kwatemaa N, Littel P, Theobald N, Choi U, Su L, Marquesen M, Hilligoss D, Lee J, Buckner CM, Zarember KA, O'Connor G, McVicar D, Kuhns D, Throm RE, Zhou S, Notarangelo LD, Hanson IC, Cowan MJ, Kang E, Hadigan C, Meagher M, Gray JT, Sorrentino BP, Malech HL, Kardava L. Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency. Sci Transl Med. 2016 Apr 20;8(335):335ra57. doi: 10.1126/scitranslmed.aad8856. |
| D000081207 | Primary Immunodeficiency Diseases |
| D007232 | Infant, Newborn, Diseases |
| D007153 | Immunologic Deficiency Syndromes |
| D007154 | Immune System Diseases |