NL-OMON54570招募中2 期
PHASE I/II CLINICAL TRIAL OF AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY FOR RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY - Phase I/II clinical trial on RAG1 gene therapy in SCID
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 1（—）
入选标准
- •- RAG1 deficient SCID as confirmed by genetic analysis
- •- Peripheral blood T cells < 300/µL and/or naïve T cells < 1/µL
- •- lack of an available HLA-matched donor (i.c. HLA-identical sibling or 10/10
- •(A, B, C, DR, DQ) allele-matched (un)related donor)
- •- Age < 2 years
- •- Age at least 8 weeks by the time of busulfan and fludarabine administration
- •- Signed consent form (parental or guardian)
- •- Able to return to the local HSCT centre for follow-up (per protocol) during
- •the 2-year study and the 15 year long-term off study review
排除标准
- •- availability of a HLA-matched donor (i.c. HLA-identical sibling or 10/10 (A,
- •B, C, DR, DQ) allele-matched (un)related donor)
- •- RAG 1 deficiency with peripheral blood T cells > 300/µL and/or naïve T cells
- •- Previous allogeneic stem cell transplantation
- •- Significant organ dysfunction/co-morbidity (including but not limited to the
- •ones listed below)
- •a. Mechanical ventilation
- •b. Shortening fraction on echocardiogram <25%
- •c. Renal failure defined as dialysis dependence
- •d. Uncontrolled seizure disorder
- •- Omenn syndrome
- •- Any other condition that the investigator considers is a contraindication
- •to collection and/or infusion of transduced cells for that individual or
- •indicate patient's inability to follow the protocol, for example
- •contraindication f to busulfan, major congenital abnormalities, ineligible to
- •receive anaesthesia, or documented refusal or inability of the family to return
- •for scheduled visits.
- •- Human immunodeficiency virus (HIV) infection or Human T-ceel Leukemia Virus
- •(HTLV) infection).
研究者
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