A Pilot Study of Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis
Status:
Recruiting
Trial end date:
2025-03-01
Target enrollment:
Participant gender:
Summary
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:
1. To evaluate the change in the stool microbiome pre-FMT vs post-FMT.
2. To evaluate changes in clinical symptoms pre-FMT and post-FMT.
Tertiary/Exploratory Objectives:
1. Preliminary evaluation of the safety of FMT in CGD-AC.
2. To evaluate other markers of intestinal and systemic inflammation pre-FMT and post-FMT.
3. To evaluate a washout period for beneficial effects of FMT on fecal calprotectin and the
microbiome.
4. 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:
1. 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.
2. Difference in Patient Reported Outcome-2 (PRO-2) pre-FMT and within 1 month post-FMT.
Tertiary/Exploratory Endpoints:
1. Unexpected AEs possibly, probably, or definitely related to FMT; bacteremia; initiation
of antibiotics above baseline antibiotics.
2. 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.
3. Changes in fecal calprotectin and microbiome indices over 6 months post-FMT.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)