jRCTs031180397已完成不适用
A pilot study for hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin from HLA-mismatched related donor for non-malignant diseases
未提供0 个研究点目标入组 9 人开始时间: 2017年4月16日最近更新:
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 9
- 主要终点
- -
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Single Arm Study
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- 0age over 至 25age old not（—）
- 性别
- All
入选标准
- •Diagnosed as bone marrow failure, immunodeficiency, or inborn errors of metabolism
- •Fanconi anemia is excluded for bone marrow failure.
- •No HLA 5/6 (A, B, DR) or more matched related donor
- •No HLA 6/6 (A, B, DR) matched unrelated donor
- •Transplantation from 2- or more-antigen mismatched related donor
- •Age at transplantation <25 years
- •ECOG Performance status: 0-2
- •Sufficient organ function, as follows;
- •a. T-Bil <= 1.5 mg/dl
- •b. Serum creatinine <= 0.8 mg/dl (age <5y), <= 1.2 mg/dl (age 5-9y), <= 1.5 mg/dl (10y or older)
- •c. Ejection fraction 45% or better, and QTfc <0.45 sec
- •Obtained informed consent from a guardian of the patient
排除标准
- •Effusion with grade 2 or worse at CTCAE ver 4.0
- •Uncontrolled infection
- •Severe psychological disorders
- •Pregnant or suspected pregnancy
- •Not eligible for this study at the discretion of the investigator.
结局指标
主要结局
-
Incidence of events (acute GVHD grade III or more, graft failure, death) by day 30
次要结局
- engraftment at day 30(day 30)
- GVHD-free survival at day 100(day 100)
- mortality at day 100(day 100)
- time from transplantation to onset of a/cGVHD
- adverse event
- incidence of infectious disease at week 8(week 8)
- time from transplantation to death
研究者
相似试验
已完成
不适用
A pilot study for hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin from HLA-mismatched related donor for non-malignant diseases (Halo-NM)on-malignant diseases (bone marrow failure, immunodeficiency, inborn errors of metabolism)JPRN-UMIN000026159ational Center for Child Health and Development9
已完成
1 期
A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich SyndromeWiskott-Aldrich syndromeJPRN-UMIN000030806ational Center for Child Heath and Development3
招募中
2 期
Hematopoietic stem cell gene therapy study2024-515253-25-00Orchard Therapeutics (Europe) Limited20
招募中
3 期
The pilot study of autologous hematopoeitic stem cell transplantation in neuromyelitis optica patients at Siriraj HospitalEffectiveness of autologous hematopoietic stem cell transplantation in aquaporin-4 antibody seropositive neuromyelitis optica spectrum disorder patientsneuromyelitis optica spectrum disorderaquaporin-4 antibodyautologous hematopoiectic stem cell transplatationTCTR20210716002Faculty of Medicine Siriraj Hospital, Mahidol University10
招募中
2 期
A Phase II study:Haematopoietic Stem Cell Transplantation for highly activetreatment resistant multiple sclerosis .ACTRN12616000151437Austin Health20