Fecal microbiota transplantation for colitis in chronic granulomatous disease

A Pilot Study of Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis

This study is testing whether a treatment called fecal microbiota transplantation can help people aged 10-60 with chronic granulomatous disease who have colitis that hasn't improved with other treatments.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 10 Years to 60 Years
• Sex: All
• Sponsor: National Institutes of Health Clinical Center (CC) (nih)
• Locations: 1 site (Bethesda, Maryland)
• Trial ID: NCT05333471 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the use of fecal microbiota transplantation (FMT) to treat colitis associated with chronic granulomatous disease (CGD). Participants aged 10-60 with CGD and ineffective or side-effect-prone treatments will undergo a series of assessments, including stool sample collection and colonoscopy. The primary goal is to measure changes in intestinal inflammation, microbiome composition, and clinical symptoms before and after FMT. The study aims to determine the safety and efficacy of this innovative approach to managing CGD-related colitis.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 10-60 with confirmed chronic granulomatous disease and colitis who have not responded to previous treatments.

Not a fit: Patients with stable CGD-IBD treatment or those not meeting the inclusion criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce inflammation and improve quality of life for patients with CGD-associated colitis.

How similar studies have performed: While fecal microbiota transplantation has shown promise in other gastrointestinal conditions, this specific application for CGD-associated colitis is novel and untested.

Eligibility criteria

* 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 preceding FMT.
7. No planned escalation in CGD-IBD treatment for 1 month preceding FMT.
8. If taking monoclonal antibodies for CGD-IBD, the dose must be stable for 12 weeks with no planned escalation.
9. 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:

1. Evidence of acute GI infection, including active GI abscesses.
2. Presence of C difficile toxin gene in stool, as identified by PCR, in screening period.
3. History of intestinal obstruction definitively related to CGD-IBD.
4. History of fistulizing CGD-IBD or CGD-IBD intra-abdominal abscesses.
5. History of CGD-IBD related non-transversable intestinal strictures.
6. History of AEs attributable to previous FMT.
7. History of significant liver disease (eg, biopsy-proven nodular regenerative hyperplasia), including portal hypertension or cirrhosis.
8. Pregnant or breastfeeding.
9. History of severe food allergy.
10. Any contraindication to having colonoscopy under anesthesia.
11. Any condition that, in the opinion of the investigator, contraindicates participation in this study.

Where this trial is running

Bethesda, Maryland

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (RECRUITING)

Study contacts

• Principal investigator: Suchitra K Hourigan, M.D. — National Institute of Allergy and Infectious Diseases (NIAID)
• Study coordinator: Suchitra K Hourigan, M.D.

Conditions: Chronic Granulomatous Disease-associated Colitis, Microbiome, Inflammation, Fecal Calprotectin, Immunodeficiency