A Phase I/II Study Evaluating the Safety and the Efficacy of Human T Lymphoid Progenitor (HTLP) Injection to Accelerate Immune Reconstitution After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- reconstitution of the CD3+ TCRαβ+ cell compartment
研究概览
简要总结
The purpose of this study is to evaluate the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients.
详细描述
Not provided
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pediatric patients affected by any type of SCID confirmed by clinical, immunological and/or molecular diagnosis and eligible for an allogeneic HSCT
- •Absence of a matched sibling donor or a matched unrelated donor (MUD) 10/10
- •Clinical conditions incompatible with the search of a MUD
- •Written, informed consent of parents/ legal representative (child)
- •Age ≤ 2 years at the time of screening
- •No prior therapy with allogeneic stem cell transplantation
- •No treatment with another investigational drug within one month before inclusion
- •Patient affiliated to social security
排除标准
- •Presence of an HLA genoidentical donor
- •Absence of written parental consent
- •Treatment with another investigational drug within one month before inclusion
- •Positive for HIV infection by genome PCR
- •Contra-indication to allogeneic transplantation or conditioning therapy (except SCID patients with DNA repair deficiency)
研究组 & 干预措施
Human T Lymphoid Progenitor (HTLP) injection
Human T Lymphoid Progenitor cells (HTLPs) obtained after a brief period of ex vivo culture in the presence of the fusion protein DL-4, Retronectin® and a combination of cytokines
干预措施: Human T Lymphoid Progenitor (HTLP) injection (Drug)
结局指标
主要结局
reconstitution of the CD3+ TCRαβ+ cell compartment
时间窗: Month 3
determined by the presence of ≥ 300/µL total, circulating CD3+ TCRαβ+ T cells to evaluate the efficacy
Dose limiting toxicity (DLT)
时间窗: 3 months post-transplant
to evaluate the procedure safety
次要结局
- NK cell numbers(Month 6, month 12)
- Cumulative incidence of acute and chronic episodes of graft versus host disease (GVHD)(24 months post-transplant)
- Immunoglobulin (Ig) levels(Month 6, month 12)
- Cumulative incidence of infections(12 months post-transplant)
- presence of recent thymic emigrants(Month 3, month 6, month 12)
- TCR rearrangements(Month 3, month 6, month 12)
- B-cell reconstitution(Month 6, month 12)
- Time course of reconstitution of the different T cell subpopulations(Month 3, month 6, month 12)
- T-cell receptor excision circles (TREC ) number in peripheral blood(Month 3, month 6, month 12)
- Overall survival(2 years post-transplant)