Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector Encoding the ITGB2 Gene
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
- 3
- 主要终点
- Hematopoietic stem cell transplant (HSCT) free survival
研究概览
简要总结
This Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) is a continuation of a Phase 1/2 clinical study to evaluate the safety and efficacy of the infusion of autologous hematopoietic stem cells transduced with a lentiviral vector encoding the ITGB2 gene
详细描述
Following the end of participation in Study RP-L201-0318, patients will be offered enrollment into this LTFU protocol. Patients will be followed for up to 15 years following the RP-L201 infusion in the parent study, until the patient dies, withdraws consent, or is lost to follow-up (whichever occurs first).
For all follow-up visits, remote evaluation facilitated by local health care providers (with blood sample shipment to relevant laboratory facilities) is permitted; however, annual visits to the study center are required during initial 3 years post- RP-L201 infusion. Visits where a bone marrow sample is being collected are required to be performed at the study center for the duration of the study. Peripheral Blood samples and bone marrow samples will be archived and tested when clinically or scientifically indicated, as in the event of development of a second malignancy.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 3 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Enrolled in the Phase I/II Study RP-L201-
- •Received an autologous infusion of CD34+ hematopoietic stem cells modified with a lentiviral vector containing the ITGB2 gene, encoding for the human CD18 receptor in the parent Study RP-L201-
- •Able to adhere to the study visit schedule and other protocol requirements.
- •Provided written informed consent and, as applicable, assent to participate in the current study.
排除标准
- •There are no criteria for exclusion in this study.
结局指标
主要结局
Hematopoietic stem cell transplant (HSCT) free survival
时间窗: 15 years
Survival without allogeneic-HSCT.
次要结局
- Event free survival(15 years)
- Overall Survival(15 years)
- Long-term genetic correction in PB CD15+ granulocytes(15 years)
- Incidence of significant infections(15 years)
- Resolution of LAD-I-related skin rash(15 years)
- Long-term CD18 neutrophil expression by flow cytometry(15 Years)
- Long-term CD11 neutrophil expression by flow cytometry(15 Years)
- Long-term genetic correction in peripheral blood mononuclear cells (PBMCs)(15 years)
- Incidence of hematologic malignancy(15 Years)
- Incidence of hospitalizations(15 years)
- Resolution of LAD-I-related periodontal abnormalities(15 years)
- Improvement or resolution of LAD-I related neutrophilia(15 Years)
- Improvement or resolution of LAD-I-related leukocytosis.(15 Years)
- Incidence of Investigational Product (IP) related serious adverse events (SAEs)(15 Years)