A Pilot Study of Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis
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- STATUS
- Recruiting
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- End date
- Mar 1, 2025
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- participants needed
- 20
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- sponsor
- 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 improvement in clinical symptoms.
Primary Objective:
To evaluate the change in intestinal inflammation pre-FMT vs post-FMT.
Secondary Objectives:
- To evaluate the change in the stool microbiome pre-FMT vs post-FMT.
- To evaluate changes in clinical symptoms pre-FMT and post-FMT.
- Preliminary evaluation of the safety of FMT in CGD-AC.
Tertiary/Exploratory Objectives:
- To evaluate other markers of intestinal and systemic inflammation pre-FMT and post-FMT.
- To evaluate a washout period for beneficial effects of FMT on fecal calprotectin and the microbiome.
- To evaluate the effect of FMT on antibiotic resistance in CGD-AC.
Primary Endpoint:
Difference in fecal calprotectin pre-FMT within 1 month post-FMT.
Secondary Endpoints:
- Differences in alpha diversity, beta diversity, and relative abundance of taxa pre-FMT and within 1 month post-FMT. Assessment of engraftment of donor microbiome.
- Difference in Patient Reported Outcome-2 (PRO-2) pre-FMT and within 1 month post-FMT.
- Treatment-emergent adverse events (TEAEs).
Tertiary/Exploratory Endpoints:
- Changes in C-reactive protein (CRP) and erythrocyte sedimentation rate (ESR) pre-FMT and post-FMT; changes in Simple Endoscopic Score for Crohn s Disease (SES-CD) pre-FMT and post-FMT in those who undergo a second colonoscopy; changes in magnetic resonance (MR) enterography findings in those who undergo a second MR enterography.
- Changes in fecal calprotectin and microbiome indices over 6 months post-FMT.
- Changes in antibiotic resistance genes in stool microbiome pre-FMT and post-FMT.
Details
| Condition | Chronic Granulomatous Disease-associated Colitis |
|---|---|
| Treatment | OpenBiome FMT product FMP250 |
| Clinical Study Identifier | NCT05333471 |
| Sponsor | National Institute of Allergy and Infectious Diseases (NIAID) |
| Last Modified on | 28 October 2022 |
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