A Pilot Study of Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Difference in fecal calprotectin pre FMT and within 1 month post FMT.
研究概览
简要总结
Background:
Chronic granulomatous disease (CGD) weakens the body's defense against germs. CGD can also damage the colon. It can cause inflammation (colitis) that disrupts the good bacteria. Placing good bacteria from donor stool into the intestine of a person with CGD (called fecal microbiota transplantation, or FMT) may help.
Objective:
To see if FMT can reduce inflammation in the colon.
Eligibility:
People aged 10-60 who have CGD and colitis, and the treatments they have tried are not helping or have side effects.
Design:
Participants will have a telehealth screening visit. They will have a medical record review and medical history. They will collect stool samples at home and mail them to NIH.
Participants will stay at the NIH hospital for 3-5 days. Each day, they will have the following:
Physical exam
Medical history and medicine review
Surveys about CGD and how it affects their life
Blood, stool, and urine tests
Participants will have a colonoscopy. They will be sedated. A long, flexible tube will be inserted into their rectum. The tube will deliver the FMT material to their colon. Small samples of intestinal tissue will be collected.
Participants may have an optional MRI of the digestive tract.
Participants will have 9 follow-up telehealth visits over 6 months. They will be asked about their symptoms and side effects. They will fill out short surveys. They will collect stool and urine samples at home. Up to 2 visits can be done in person. At these visits, they may have the option to have an MRI and another colonoscopy to get more tissue samples.
Participation will last for 6-7 months.
详细描述
Study Description:
This is a prospective, single-site, single-arm, open-label pilot trial to evaluate the use of fecal microbiota transplantation (FMT) delivered by colonoscopy as a treatment for chronic granulomatous disease (CGD)-associated colitis (AC). Participants will be evaluated for changes in intestinal inflammation, the microbiome, and symptoms associated with CGD-AC. It is hypothesized that FMT will reduce intestinal inflammation as measured by fecal calprotectin within 1 month compared to baseline (pre-FMT); there will be associated changes in the underlying stool microbiome and improvement in clinical symptoms.
Primary Objective:
To evaluate the change in intestinal inflammation pre-FMT vs post-FMT.
Secondary Objectives:
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
- 10 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •INCLUSION CRITERIA:
- •In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- •Aged >=10 to <=60 years.
- •Able to provide informed consent (for ages >=18 years) or has a parent or guardian who can provide informed consent on their behalf (for ages <18 years).
- •Have confirmed prior diagnoses of CGD and CGD-AC (or CGD-IBD with evidence of colitis on colonoscopy).
- •Fecal calprotectin level >=200 microgram/g.
- •HBI score >=5 (to be evaluated on Day 1).
- •No planned change in systemic antibiotic regimen for CGD for 1 month preceding FMT.
- •No planned escalation in CGD-IBD treatment for 1 month preceding FMT.
- •If taking monoclonal antibodies for CGD-IBD, the dose must be stable for 12 weeks with no planned escalation.
- •Participants who can become pregnant must agree to use at least one highly effective method of contraception when engaging in sexual activities that can result in pregnancy, starting at screening until the end of study participation. Highly effective methods include a barrier (eg, condom, diaphragm, cervical cap), intrauterine device, or hormonal contraception.
排除标准
- •An individual who meets any of the following criteria will be excluded from participation in this study:
- •Evidence of acute GI infection, including active GI abscesses.
- •Presence of C difficile toxin gene in stool, as identified by PCR, in screening period.
- •History of intestinal obstruction definitively related to CGD-IBD.
- •History of fistulizing CGD-IBD or CGD-IBD intra-abdominal abscesses.
- •History of CGD-IBD related non-transversable intestinal strictures.
- •History of AEs attributable to previous FMT.
- •History of significant liver disease (eg, biopsy-proven nodular regenerative hyperplasia), including portal hypertension or cirrhosis.
- •Pregnant or breastfeeding.
- •History of severe food allergy.
- •Any contraindication to having colonoscopy under anesthesia.
- •Any condition that, in the opinion of the investigator, contraindicates participation in this study.
研究组 & 干预措施
Interventional
As this is a single arm study, this arm includes all participants. Participants will receive fecal microbiota transplantation (FMT) delivered by colonoscopy as a treatment for chronic granulomatous disease (CGD)-associated colitis (AC).
干预措施: MTP 101-LF (Drug)
结局指标
主要结局
Difference in fecal calprotectin pre FMT and within 1 month post FMT.
时间窗: Within 1 month
To evaluate the change in intestinal inflammation pre-FMT vs post FMT
次要结局
- Difference in PRO-2 pre-FMT and within 1 month post-FMT.(Within 1 month)
- Treatment-Emergent Adverse Events(Through end of study)
- Differences in alpha diversity, beta diversity, and relative abundance of taxa pre-FMT within 1 month post-FMT and assessment of engraftment of donor microbiome and Assessment of engraftment of donor microbiome.(Within 1 month)