A Single-arm, Open-label, Study to Evaluate the Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) Undergoing Stem Cell Transplantation After Reduced Intensity Conditioning
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 主要终点
- Incidence of graft failure
研究概览
简要总结
This study is designed to assess the safety and exploratory efficacy of using HSC835 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
详细描述
This phase II study is designed to assess the safety of the Novartis product HSC835 and its ability to achieve donor blood stem cell engraftment in patients with certain Inherited Metabolic Disorders who undergo stem cell transplantation. A reduced intensity conditioning will be used prior to transplantation. Patients with Hurler syndrome, MLD, Krabbe or cALD could be eligible for this study.
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
- 12 Months 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with Hurler syndrome, Metachromatic leukodystrophy (MLD), Globoid cell leukodystrophy (Krabbe) or Cerebral adrenoleukodystropy (cALD) -Adequate organ function -Availability of eligible donor material
排除标准
- •Availability of a matched-related donor who is not a carrier of the same genetic defect -Active infection at screening -Prior myeloablative transplant -Pregnant or nursing women and women of child bearing potential unless using highly effective contraception methods. For the pediatric population, female patients of child bearing potential who do not agree to abstinence or agree to use highly effective contraception methods -Sexually active male patients unless using condoms as contraception -Human Immunodeficiency virus (HIV) infection
研究组 & 干预措施
HSC835
HSC835 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation
干预措施: Umbilical cord blood transplantation with HSC835 (Drug)
结局指标
主要结局
Incidence of graft failure
时间窗: 42 days
Incidence of infusional toxicities
时间窗: 48 hours
Incidence of neutrophil recovery
时间窗: 42 days
次要结局
- Time to neutrophil recovery(42 days)
- Time to platelet recovery(180 days)
- Number of patients with grade II-IV acute graft versus host disease (aGVHD)(100 days)
- Number of patients with chronic graft versus host disease (cGVHD)(1 and 2 years)
- Incidence of death(100 days, 1 year and 2 years)