A Pilot Study of Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis
National Institute of Allergy and Infectious Diseases (NIAID)
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.
Description
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 i…
Eligibility
- Age range
- 10–60 years
- Sex
- All
- Healthy volunteers
- No
* INCLUSION CRITERIA: In order to be eligible to participate in this study, an individual must meet all of the following criteria: 1. Aged \>=10 to \<=60 years. 2. 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). 3. Have confirmed prior diagnoses of CGD and CGD-AC (or CGD-IBD with evidence of colitis on colonoscopy). 4. Fecal calprotectin level \>=200 microgram/g. 5. HBI score \>=5 (to be evaluated on Day 1). 6. No planned change in systemic antibiotic regimen for CGD for 1 month preced…
Interventions
- DrugMTP 101-LF
Each unit of MTP-101-LF contains approximately 35 mL of fecal transplant product. Participants will receive approximately 32 mL via colonoscopy.
Location
- National Institutes of Health Clinical CenterBethesda, Maryland