Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Patients With Fanconi Anaemia Subtype A: FANCOLEN-I
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 9
- 试验地点
- 2
- 主要终点
- Insertion site analysis in blood
研究概览
简要总结
This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial. After completion of the FANCOLEN-I study, eligible subjects will be followed for a total of 15 years post gene therapy treatment. No investigational drug product will be administered during this study.
详细描述
This long-term follow-up protocol will evaluate the long term safety and efficacy of the infusion of autologous CD34+ cells transduced with lentiviral vector (LV) carrying the FANCA gene.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Enrolled in the FANCOLEN-I study
- •Treated with gene therapy in the FANCOLEN-I study
- •Able to adhere to the study visit schedule and protocol requirements
- •Provided written informed consent and, as applicable, assent to participate
排除标准
- •There are no exclusion criteria for this study
研究组 & 干预措施
Subjects with Fanconi Anaemia Subtype A (FA-A)
Subjects treated with ex vivo lentiviral gene therapy product in FANCOLEN-I trial and agree to participate in this long-term follow-up (LTFU) study
干预措施: Safety and efficacy assessments (Other)
结局指标
主要结局
Insertion site analysis in blood
时间窗: 15 years post-drug product infusion
Determine long term clonality
Phenotypic correction
时间窗: 15 years post-drug product infusion
Determine phenotypic correction of bone marrow and peripheral blood cells by resistance to DNA-damaging agents
Hematologic stabilization
时间窗: 15 years post-drug product infusion
Monitor for long term stability and normalization of blood counts
Monitor long term safety of patients through blood laboratory evaluations and general health status
时间窗: 15 years post-drug product infusion
Evaluate long term safety following infusion of hematopoietic cells transduced with therapeutic lentiviral vector (LV)
Long term genetic correction assessed in bone marrow and blood
时间窗: 15 years post-drug product infusion
Determine long term persistence of therapeutic LV in hematopoietic cells in bone marrow and blood
Replication competent lentivirus (RCL)
时间窗: 15 years post-drug product infusion
Evaluate RCL in peripheral blood
Assessment for Malignancies
时间窗: 15 years post-drug product infusion
Monitor for incidence of hematologic malignancies and solid organ tumors
次要结局
未报告次要终点