Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis
United States20 participantsStarted 2022-08-08
Plain-language summary
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.
Who can participate
Age range
10 Years – 60 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Difference in fecal calprotectin pre FMT and within 1 month post FMT.
Timeframe: Within 1 month
Trial details
NCT IDNCT05333471
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)