Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis

  • STATUS
    Recruiting
  • End date
    Mar 1, 2025
  • participants needed
    20
  • sponsor
    National Institute of Allergy and Infectious Diseases (NIAID)
Updated on 19 June 2022

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:

  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.

Details
Condition Chronic Granulomatous Disease-associated Colitis
Treatment OpenBiome FMT product FMP250
Clinical Study IdentifierNCT05333471
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)
Last Modified on19 June 2022

Eligibility

Yes No Not Sure

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
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
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

Exclusion Criteria

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
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
Use of monoclonal antibodies within the last 3 months
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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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