Feasibility, Safety, and Potential Efficacy of Fecal Microbiota Transplantation (FMT) for Gastrointestinal Dysfunction in Children Following Hematopoietic Cell Transplant (HCT).
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Proportion of participants expressing interest who meet eligibility
研究概览
简要总结
The study participant is being asked to take part in this clinical trial, a type of research study, because the participant has Gastrointestinal (GI) symptoms following a Hematopoietic Cell Transplant (HCT).
Primary Objective
- To determine the safety and feasibility of FMT for treating a GvHD of the gut following HCT.
- To determine the safety and feasibility of FMT for treating HCT induced gut dysfunction.
Secondary Objectives
- To assess the potential efficacy of FMT for treating a GvHD of the gut following HCT.
- To assess the potential efficacy of FMT for treating HCT induced gut dysfunction.
详细描述
Participants will be eligible to receive an FMT on or after Day +30 post-HCT. FMT will be performed using FMP material obtained from OpenBiome. 60 mL of FMP will be administered via NJ tube and 250 mL via colonoscopy. A second FMT may be performed at least 14 days after the initial FMT in GI clinical symptoms have partially improved or have not changed. The second FMT will be administered using the same procedure as in the initial FMT
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 22 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age < 22 years old.
- •Received an allogeneic HCT greater than or equal to 30 days prior to enrollment
- •Diagnosed with one of the following conditions:
- •Steroid-resistant gut a GvHD (defined as GI symptoms that do not improve within 5 days after initial steroid therapy, >/= 1mg/kg of prednisolone) OR
- •Steroid-dependent gut a GvHD (defined as the presence of a response to methylprednisolone 2 mg/kg/day but relapsing when an attempt was made to taper steroid treatment).
- •Current or prolonged GI dysfunction following HCT, defined as having diarrhea or loose stools >/= 4 weeks with at least one of the following:
- •Requiring NG or G-tube feeds
- •Requiring TPN or IVF for more than 4 weeks
- •Diagnosis of gastroparesis by GI specialist documented in the medical record
- •Willing and able to provide informed assent/consent
排除标准
- •Participant is at risk for aspiration pneumonia
- •History of anaphylactic allergy to foods that are not excluded from the stool donor diet
- •Female participant who is pregnant or nursing
- •History of previous FMT
- •Intra-abdominal surgery within 4 weeks of enrollment
- •At increased risk for peritonitis: presence of intra-abdominal devices (G-or GJ-tubes are acceptable), receiving peritoneal dialysis, or ascites
- •Concurrent abdominal radiation therapy
- •Any acute or chronic illness/condition as well as medication that in the opinion of the investigator puts the subject at greater risk from FMT or may confound the study results.
研究组 & 干预措施
Stratum A
Diagnosed with GvHD
干预措施: Fecal microbiota transplant (FMT) (Drug)
Stratum B
GI Dysfunction
干预措施: Fecal microbiota transplant (FMT) (Drug)
结局指标
主要结局
Proportion of participants expressing interest who meet eligibility
时间窗: 2 years
Participant eligibility will be a primary outcome measure for the following groups: * FMT for treating a GvHD of the gut following HCT * FMT for treating HCT induced gut dysfunction
Proportion of participants recruited in the eligible population
时间窗: 2 years
Participant recruitment will be a primary outcome measure for the following groups: * FMT for treating a GvHD of the gut following HCT * FMT for treating HCT induced gut dysfunction
Proportion of participants with a serious adverse event occurring within 30 days following FMT
时间窗: 30 Days
Serious adverse events will be a primary outcome measure for the following groups: * FMT for treating a GvHD of the gut following HCT * FMT for treating HCT induced gut dysfunction
Proportion of participants providing all protocol required stool samples
时间窗: 3 years
Stool specimens will be a primary outcome measure for the following groups: * FMT for treating a GvHD of the gut following HCT * FMT for treating HCT induced gut dysfunction
Proportion of participants with a non-serious adverse event occurring within 30 days following FMT
时间窗: 30 Days
Non-serious adverse events will be a primary outcome measure for the following groups: * FMT for treating a GvHD of the gut following HCT * FMT for treating HCT induced gut dysfunction
Proportion of participants that drop up post-enrollment
时间窗: 3 years
Participant retention will be a primary outcome measure for the following groups: * FMT for treating a GvHD of the gut following HCT * FMT for treating HCT induced gut dysfunction
次要结局
- Percentage of participants who reduce or discontinue steroids at the end of the study(1 year)
- Proportion of participants with a complete response or a partial response(180 days)