Recruiting
Phase 1
Phase 2

FMT

Sponsor:

National Institute of Allergy and Infectious Diseases (NIAID)

Code:

NCT05333471

Conditions

Chronic Granulomatous Disease-associated Colitis

Eligibility Criteria

Sex: All

Age: 10 - 60

Healthy Volunteers: Not accepted

Interventions

MTP 101-LF

Study Details

Brief 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.

Conditions

Chronic Granulomatous Disease-associated Colitis

Study ID

NCT05333471

Start date

Aug 8, 2022

Status verified date

Feb 13, 2026

Completion date

Mar 1, 2027

Anticipated

Primary completion date

Mar 1, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 10 - 60

Healthy Volunteers: Not accepted

  • 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.

Study Design

Enrollment

20 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Interventional

As this is a single arm study, this arm includes all participants. Participants will receive fecal microbiota transplantation (FMT) delivered by colonoscopy as a treatment for chronic granulomatous disease (CGD)-associated colitis (AC).

Interventions

MTP 101-LF

Each unit of MTP-101-LF contains approximately 35 mL of fecal transplant product. Participants will receive approximately 32 mL via colonoscopy.

Primary outcome measure

  • Difference in fecal calprotectin pre FMT and within 1 month post FMT. [ Time Frame: Within 1 month ]

Central Contacts and Locations

Central contacts

Suchitra K Hourigan, M.D.

(240) 627-3995suchitra.hourigan@nih.gov

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

800-411-1222ccopr@nih.gov

More Information

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Last update posted

Mar 31, 2026

Last verified

Feb 13, 2026

Keywords

  • Microbiome
  • Inflammation
  • Fecal Calprotectin
  • Immunodeficiency

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by National Institute of Allergy and Infectious Diseases (NIAID) on 2026-03-31.