REFOCUS: Refined Fecal Microbiota Transplantation (FMT) Delivered by Oral Capsules for Induction of Remission in Mild to Moderate Ulcerative Colitis - a Phase I Study
试验速览
- 阶段
- 早期 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Proportion of participants with an adverse
研究概览
简要总结
The researchers intend to prospectively study the safety, clinical efficacy and microbial outcomes in patients with recently diagnosed UC with FMT capsule therapy derived from pre-defined donors. Donors will be specifically screened for Fusobacterium and Sutterella species as well as for global diversity. It is unknown if treatment with antibiotics before FMT improves the engraftment and/or efficacy of FMT in UC, therefore the researchers plan to randomize subjects to receive pre-treatment with antibiotics or not before FMT therapy. The research team enroll patients from The Susan and Leonard Feinstein IBD Center and our established early diagnosis clinic at Mount Sinai Hospital (MSH).
详细描述
This is an open-label two arm pilot study to measure the safety, microbiological and clinical impacts of FMT in patients with ulcerative colitis. The researchers will prospectively enroll 16 UC patients (up to 20 subjects accounting for subjects dropping out) with moderate-severe disease from one tertiary care referral center. The overall objective of the study is to collect robust clinical data and create a tissue repository including blood, stool and biopsies to understand the safety, efficacy and microbial changes FMT has on UC patients. The central hypothesis is that pre-defined oral capsule administered FMT is safe and effective for the treatment of UC.
Objectives: To determine the tolerability, feasibility, and safety of using fecal microbiota transplantation orally as an induction agent for patients with ulcerative colitis. To determine whether fecal microbiota transplantation (FMT) delivered via oral capsules can induce clinical remission in patients with mild to moderate ulcerative colitis. Assess whether pretreatment with antibiotics improves engraftment and efficacy of FMT in UC. To characterize the impact of orally administered FMT on the microbiota of patients with ulcerative colitis, particularly those changes associated with response or lack of response.
Study Outcomes: Clinical remission at Week 8, defined as: Steroid-free clinical remission (Total Mayo less 2) and Endoscopic remission (Mayo endoscopic subscore 0 or 1)
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
盲法说明
Antibiotics and placebo will be disguised
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
FMT with Antibiotics
Participants will receive antibiotics before receiving Fecal Microbiota Transplantation
干预措施: Metronidazole (Drug)
FMT with Antibiotics
Participants will receive antibiotics before receiving Fecal Microbiota Transplantation
干预措施: Vancomycin (Drug)
FMT with Antibiotics
Participants will receive antibiotics before receiving Fecal Microbiota Transplantation
干预措施: Fecal Microbiota Transplantation (Biological)
FMT with placebo
Participants will receive placebo before receiving Fecal Microbiota Transplantation
干预措施: Placebo (Drug)
FMT with placebo
Participants will receive placebo before receiving Fecal Microbiota Transplantation
干预措施: Fecal Microbiota Transplantation (Biological)
结局指标
主要结局
Proportion of participants with an adverse
时间窗: 8 Weeks
Safety as measured by proportion of participants with an adverse event through week 8
Proportion of participants with a severe adverse
时间窗: 8 Weeks
Safety as measured by proportion of participants with a severe adverse event through week 8
次要结局
- ESR level(baseline and 8 weeks)
- Number of patients requiring escalation of medical therapies(8 weeks)
- Albumin levels(baseline and 8 weeks)
- Proportion of patients that achieve Mayo score 0 or 1(8 weeks)
- Number of patients with endoscopic remission(8 weeks)
- Change in Nancy score(8 weeks)
- Fecal calprotectin level(baseline and 8 weeks)
- C-reactive protein levels(baseline and 8 weeks)
- Hemoglobin levels(baseline and 8 weeks)
研究者
Ari M Grinspan
Associate Professor of Medicine
Icahn School of Medicine at Mount Sinai