Long Term Safety Follow up of Patients Enrolled in the Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (GTG002-07 and GTG003-08).
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Genethon
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Hematological reconstitution
研究概览
简要总结
An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Patients enrolled in the initial phase I/II WAS conducted in France and United Kingdom (GTG002.07 and GTG003.08).
- •Parents, guardians or patient signed informed consent, guardians or patient signed informed consent
排除标准
- •Parents, guardians, patients unwilling to return for the follow up study period.
结局指标
主要结局
Hematological reconstitution
时间窗: yearly from 3 years to 10 years
CBC including platelets count and size
Lentiviral integration sites
时间窗: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Presence of lentiviral integration sites in different cells sub-populations
Vector copy numbers
时间窗: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Quantification of vector copy numbers on sorted cells population by q-PCR
Incidence and type of SAEs
时间窗: yearly from 3 years to 15 years
Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality
Replication competent lentivirus (RCL)
时间窗: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Presence of RCL
Change in medical conditions
时间窗: yearly from 3 years to 10 years
Weight and complete clinical exam
Key medical events related to WAS
时间窗: yearly from 3 years to 10 years
Eczema status, infections, bleeding symptoms, autoimmune manifestation
Reconstitution of cell mediated and humoral immunity
时间窗: yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )
Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene
次要结局
- Bone marrow content(yearly from 3 years to 5 years (optional))
- Need for associated treatments(yearly from 3 years to 15 years)
- Representation of TCR families(yearly from 3 years to 5 years)