A Phase I Study of Fecal Microbiota Transplantation (FMT) in Immunomodulator Dependent Pediatric Ulcerative Colitis (UC)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Assess safety of FMT treatment by recording the frequency of adverse events.
研究概览
简要总结
Patients diagnosed with ulcerative colitis (UC), a specific type of inflammatory bowel disease, will be invited to take part in this study. The investigators do not know what causes UC. However, the microbes (such as bacteria and viruses), which normally live in our intestines are thought to play an important role in the development of UC. There are many treatment options for UC such as steroids,and other medications that decrease inflammation. However, none of these can cure the disease.
This study aims to treat pediatric UC in an alternative fashion by changing the microbes in the gut by giving the participant's stool specimens from healthy adult individuals. There is some evidence from a few cases of adult UC that this therapy may cure the disease or at least provide long lasting suppression (remission) of the symptoms. The investigators would like to test this therapeutic intervention in children with UC.
The purpose of this study is to (1) examine the microbiome of stool and colon (2) and to determine whether healthy-donated stool enemas can treat pediatric ulcerative colitis. (3) The investigators will also study the effects of the treatment on colonic gene expression.
详细描述
FMT RECIPIENTS:
Participants will be recruited from the patient population treated by the Pediatric Gastroenterology, Hepatology, and Nutrition Section at Baylor College of Medicine/Texas Children's Hospital.
I. Activities prior to starting FMT therapy:
- Participants will be asked to discontinue or taper their current home medications for ulcerative colitis.
- Participants will be asked to complete enema training.
- The participant and family will be counseled in case emergency surgical and/or intensive care interventions are required.
- Survey Completion to assess clinical symptoms.
II. Initiation of FMT Therapy:
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 Years 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patients must have a diagnosis of ulcerative colitis based on clinical symptoms, as well as laboratory and colonoscopic findings, including histopathologic results of the intestinal mucosa.
- •Pediatric Ulcerative Colitis Activity Index (PUCAI) < 35 [mild activity] as assessed within 4 weeks prior to enrollment and clinical symptoms that are at least stable.
- •Patients must be "immunomodulator dependent," i.e., have a history of steroid dependency (lack of clinical remission for over 8 weeks without steroid therapy), and/or being immunomodulator (azathioprine, 6-mercaptopurine, methotrexate, etc.) treated, and/or biologic agent (infliximab, adalimumab, etc.) treated
- •Patients ≥ 12 and < 21 years of age are eligible for this trial.
- •Patients must be willing to be off current UC therapies.
- •Willingness to undergo a surgical consultation prior to FMT treatment.
- •Patients who are not enema trained will be required to undergo enema training prior to the start of FMT treatment.
- •All patients and/or their parents or legally authorized representatives must sign a written informed consent document. Assent, when appropriate, will be obtained according to institutional guidelines.
排除标准
- •Patients who are known to have the following will be excluded:
- •Decompensated liver cirrhosis (bleeding varices, ascites, encephalopathy or icterus)
- •Human immunodeficiency virus (HIV)/acquired immune deficiency syndrome (AIDS)
- •Bone marrow transplantation within the past 150 days
- •Other severe immunodeficiency
- •Patients with severe prior allergic reaction to food will be excluded from the protocol.
- •Patients who are pregnant or lactating will be excluded from the protocol.
- •Donor Inclusion Criteria:
- •≥ 18 years and < 45 years
- •Willing to provide stool and blood samples for laboratory testing.
- •Body mass index < 30
- •Willing to fill out a detailed health screening questionnaire
结局指标
主要结局
Assess safety of FMT treatment by recording the frequency of adverse events.
时间窗: 12 months
To determine the safety and associated toxicities of serial FMTs in pediatric UC following withdrawal from standard therapy.
次要结局
- Assess efficacy of FMT treatment with the aid of the Pediatric Ulcerative Colitis Activity (PUCAI) Index, a validated measure of clinical disease severity.(1 year)
研究者
Richard Kellermayer
Assistant Professor
Baylor College of Medicine