PrOspective inteRventional Study Exploring the mIcrobiota recolONization in Steroid-Refractory Graft-versus-Host Disease Patients Receiving MaaT013
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 18
- 主要终点
- Change of gut microbiota composition
研究概览
简要总结
The objective of the ORION study is to explore the changes of gut microbiota composition following MaaT013 administration and its impact on the immune system in GVHD patients.
详细描述
The french regulatory authority ANSM approved in July 2019 the use of MaaT013, a pooled microbiome-based enema formulation, under a formalized named-patient use program in France called "Autorisation Temporaire d'Utilisation - ATU nominative protocolisée - ATUn" or Early Access.
The ATUn is indicated to provide a benefit in the treatment of patients with grade III-IV aGVHD:
- In case of initial resistance to CS alone or in association or in case of failure to other treaments
- In first-line therapy in association with CS in case of steroid-dependance (inability to taper CS dose <0.5 mg/kg/d)
- In case of digestive aGvHD with overlap syndrome The objective of the ORION study is to explore the changes of gut microbiota composition following MaaT013 administration and its impact on the immune system in GVHD patients.
Blood and stool samples will be collected at each visit which explained why the study is thus categorized as research involving the human person with low risks and constraints clinical trial (RIPH2).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient ≥18 years old with Grade III-IV aGVHD with gut involvement undergoing treatment with MaaT013 through named-patient use program ATUn / Early Access
- •Signature of informed and written consent by the subject or by the subject's legally acceptable representative for patients under guardianship or trusteeship.
- •Affiliated or recipient from a social security scheme
排除标准
- •Pregnancy and breastfeeding
- •Vulnerable patients such as: minors, persons deprived of liberty, persons in Intensive Care Unit unable to provide informed consent prior to the intervention.
结局指标
主要结局
Change of gut microbiota composition
时间窗: inclusion (day 0) to month 6
Sample comparisons will be performed using similarity metric such as Bray-Curtis, Jaccard distances, Sorensen, Pearson or Spearman indice.
Change of gut microbiota diversity
时间窗: inclusion (day 0) to month 6
Diversity of gut microbiota will be evaluated using alpha-diversity metric (Simpson or Shannon or Richness indice) throughout the study
Change of gut microbiota composition (phylogenetic profiles)
时间窗: inclusion (day 0) to month 6
Gut microbiota composition will be evaluated with phylogenetic profiling. Relative abundance of bacterial taxa will be described at each visit throughout the study.
次要结局
- Correlation between gut microbiota and immune parameters(inclusion (day 0) to month 6)
- Gut microbiota baseline characterization based on clinical response(day 0)
- Change in immune parameters following MaaT013 administration(inclusion (day 0) to month 6)