Evaluation of the Efficacy of Superdonor Fecal Microbiota Transplantation in Patients With Mild-to-moderate Ulcerative Colitis: a Double-blind Randomized Controlled Trial
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 42
- 试验地点
- 1
- 主要终点
- Number of patients who will obtain remission of disease 8 weeks after treatments
研究概览
简要总结
In the last decades fecal microbiota transplantation (FMT) has been established as a highly effective option in the treatment of recurrent Clostridioides difficile infection (rCDI), with a success rate of nearly 90%. For this reason, it is recommended by international guidelines as a treatment option for this indication in clinical practice.
Recently, a considerable body of evidences, suggest FMT as an effective and safe treatment in patients affected by Ulcerative Colitis (UC). In a recent meta-analysis of 324 subjects with UC, 30.4% of patients achieved both clinical and endoscopic remission after FMT compared to placebo (9.8%, P<0.00001). However, among the various published trials there is a fair variability in terms of methods and results, which are not comparable to those obtained in the rCDI.
Nowadays, one of the most critical factors involved in the effectiveness of FMT in UC patients, is the choice of the donor.
In addition, several studies have shown that some donors are associated with a higher clinical response rate than others. This hypothesis has been demonstrated in patients affected by irritable bowel syndrome, in which the use of a super-donor (a healthy person who has the predictive clinical and lifestyle characteristics of a healthy microbiota, and with a microbial profile associated with favorable clinical conditions) resulted in significantly higher clinical efficacy rates than placebo, similar to those obtained in rCDI (89%).
Currently, studies that explored the efficacy of the super-donor FMT in UC patients are not yet available. Aim of this study is to investigate the efficacy of super - donor FMT, compared with placebo FMT, in the treatment of UC. The investigators will randomize adult patients with a recent diagnosis of UC to FMT from super - donors or placebo, by colonoscopy (first infusion) and capsules administration. Then, patients will be followed up 2 months after FMT.
详细描述
In the last decades fecal microbiota transplantation (FMT) has been established as a highly effective option recurrent Clostridioides difficile infection (rCDI) treatment, with a success rate of nearly 90%. For this reason, it is recommended by international guidelines as a treatment option for this indication in clinical practice.
Recently, a considerable body of evidence supports FMT as an effective and safe treatment in patients affected by Ulcerative Colitis (UC). In a recent meta-analysis of 324 subjects with UC, 30.4% of patients achieved both clinical and endoscopic remission after FMT compared to placebo (9.8%, P<0.00001). However, among the various published trials there is a fair variability in terms of methods and results, which are not comparable to those obtained in the rCDI.
Nowadays, one of the most critical factors involved in the effectiveness of FMT in UC patients is the choice of the donor. In addition, several studies have shown that some donors have been associated with a higher clinical response rate than others. This hypothesis has been demonstrated in patients affected by irritable bowel syndrome, in which the use of a super-donor (a healthy person who has the predictive clinical and lifestyle characteristics of a healthy microbiota, and with a microbial profile associated with favorable clinical conditions) resulted in significantly higher clinical efficacy rates than placebo, similar to those obtained in rCDI (89%).
Currently, studies that explored the efficacy of the superdonor FMT in UC patients are not yet available.
The extended aims of this study are:
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
To mask treatments to physicisans and recipients, both FMT flasks and syringes will be covered with dark-coloured paper before the infusion, and the patients will be unable to see the endoscopic display during the procedure. Super - donor capsules or placebo ones will look the same. Moreover, the physicians who will evsaluate patients at follow-up will not aware of the treatment being administered.
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
- •Age between 18 and 75 years old
- •Mild to Moderate Ulcerative Colitis (total Mayo score 3 -10 + endoscopic subscore ≥ 1) in stable maintenance therapies (> 4 weeks with Aminosalicylates, > 6 weeks with immunosuppressant or biologics agents);
- •Recent diagnosis (< 12 months) of Ulcerative Colitis;
- •Ability to provide written informed consent
- •Ability to be compliant with the scheduled procedures
排除标准
- •Age < 18 years old
- •Known active gastrointestinal disorders (e.g. infectious gastroenteritis, coeliac disease, Crohn disease, irritable bowel syndrome, chronic pancreatitis, biliary salt diarrhoea)
- •Previous colorectal surgery or cutaneous stoma
- •Current or recent (< 2 weeks) therapy with drugs that could possibly alter gut microbiota (e.g. antimicrobials, probiotics, proton pump inhibitors or metformin)
- •Decompensated heart failure or heart disease with ejection fraction lower than 30%
- •Severe respiratory insufficiency
- •Psychiatric disorders
- •Pregnancy or breastfeeding
- •Unable to give informed consent
研究组 & 干预措施
Donor FMT (D-FMT)
Patients enrolled in this arm will receive donor FMT
干预措施: FMT (Biological)
Placebo FMT (P - FMT)
Patients enrolled in this arm will receive placebo FMT
干预措施: Placebo FMT (Other)
结局指标
主要结局
Number of patients who will obtain remission of disease 8 weeks after treatments
时间窗: 2 months
The investigators will evaluate evaluate the number of participants who will obtain disease remission (assessed by total mayo score \</= 2, or endoscopic Mayo \</= 1) 8 weeks after treatments.
次要结局
- Number of patients who will obtain remission of disease 1 and 4 weeks after treatments.(1 months)
- Number of patients who will obtain clinical response(2 months)
- Evaluation of changes in recipients' microbiome after treatments, at each time point.(2 months)
- Number of patients who will obtain clinical disease remission 1,4 and 8 weeks after treatments.(2 months)
- Evaluation of changes in serum cytokines after treatments, at each time point.(2 months)
- Number of patients who will obtain endoscopic remission 1,4 and 8 weeks after treatments(2 months)
研究者
IANIRO GIANLUCA
Dr.
Fondazione Policlinico Universitario Agostino Gemelli IRCCS