EUCTR2018-000673-68-GB进行中（未招募）1 期
Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1
Great Ormond Street Hospital for Children NHS Trust0 个研究点目标入组 5 人开始时间: 2018年7月27日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •All subjects must fulfill the following criteria to be included in the study:
- •1.Diagnosis of SCID-X1 based on immunophenotype and lack of T cell function (proliferation to PHA <10% of the lower limit of normal for the laboratory) AND confirmed by a mutation in IL2RG
- •2.Lack of an HLA identical (A, B, C, DR, DQ) related donor
- •3.Age <5 years
- •4.Signed informed consent
- •5.Documentation of willingness to follow up for 15 years post-infusion
- •6.If the patient has previously undergone allogeneic transplant, lack of donor T cell engraftment must be documented.
- •7.Age at least 8 weeks of age by the time of busulfan administration
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 5
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range 0
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •Subjects will not be eligible for the study if any of the following criteria is fulfilled:
- •1.Patients with an active, therapy-resistant infection. Infections that are known to be highly morbid in SCID patients will be considered active and therapy-resistant if the infectious agent is repeatedly isolated despite a minimum of 2 weeks of appropriate therapy and is associated with significant organ dysfunction (including but not limited to abnormalities listed below).
- •a.Mechanical ventilation including continuous positive airway pressure
- •b.Abnormal liver function defined by AST and ALT >10 times the upper range of normal OR Bilirubin >2 mg/dL
- •c.Shortening fraction on echocardiogram <25% or ejection fraction <50%
- •d.Renal failure defined as glomerular filtration rate <30 ml/min/1.73 m2 or dialysis dependence
- •2.Uncontrolled seizure disorder
- •3.Encephalopathy
- •4.Documented coexistence of any disorder known to affect DNA repair
- •5.Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease
- •6.Patients with evidence of infection with HIV-1
- •7.Previous allogeneic transplant with cytoreductive chemotherapy
- •8.Major (life-threatening) congenital anomalies. Examples of major (life-threatening) congenital anomalies” include, but are not limited to: unrepaired cyanotic heart disease, hypoplastic lungs, anencephaly or other major central nervous system malformations, other severe non-repairable malformations of the gastrointestinal or genitourinary tracts that significantly impair organ function.
- •9.Other conditions which in the opinion of the P.I. or Co-investigators, contra-indicate collection and/or infusion of transduced cells or indicate patient’s inability to follow the protocol. These may include for example clinical ineligibility to receive anaesthesia, severe deterioration of clinical condition of the patient after collection of bone marrow but before infusion of transduced cells, or documented refusal or inability of the family to return for scheduled visits. There may be other unforeseen rare circumstances that would result in exclusion of the patient, such as sudden loss of legal guardianship.
研究者
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