Chronic Granulomatous Disease-associated ColitisPhase 1/2NCT05333471
Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis
A Phase 1/2 study testing MTP 101-LF in people with Chronic Granulomatous Disease-associated Colitis, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 to 60 years
- Conditions the study is for
- Chronic Granulomatous Disease-associated Colitis
- What is being tested?
- MTP 101-LF
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Aug 2022to 1 Mar 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
- HBI score >=5 (to be evaluated on Day 1).
Show all 10Show fewer
- 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.
- If taking monoclonal antibodies for CGD-IBD, the dose must be stable for 12 weeks with no planned escalation.
- 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.
Who cannot join
- 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 definitively related to CGD-IBD.
- History of fistulizing CGD-IBD or CGD-IBD intra-abdominal abscesses.
- History of CGD-IBD related non-transversable intestinal strictures.
Show all 12Show fewer
- History of AEs attributable to previous FMT.
- History of significant liver disease (eg, biopsy-proven nodular regenerative hyperplasia), including portal hypertension or cirrhosis.
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR), 800-411-1222 ext. TTY dial 711, ccopr@nih.gov
About this study
Official title: A Pilot Study of Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis
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.
Read more
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.
Read the detailed 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.
- Being tested
- MTP 101-LF
- Keywords
- Microbiome, Inflammation, Fecal Calprotectin, Immunodeficiency
- Medical subject headings
- Inflammation, Immunologic Deficiency Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MTP 101-LFDrug
Each unit of MTP-101-LF contains approximately 35 mL of fecal transplant product. Participants will receive approximately 32 mL via colonoscopy.
| Study group | Role | What they receive |
|---|---|---|
| Interventional | Experimental | 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). Receives: Drug: MTP 101-LF |
What is being measured
The primary outcome is the main question the study is designed to answer.
Difference in fecal calprotectin pre FMT and within 1 month post FMT.
To evaluate the change in intestinal inflammation pre-FMT vs post FMT
Measured: Within 1 month
Secondary outcomes (3)
Difference in PRO-2 pre-FMT and within 1 month post-FMT.
To evaluate the change in the stool microbiome pre-FMT vs post-FMT.
Measured: Within 1 month
Differences in alpha diversity, beta diversity, and relative abundance of taxa pre-FMT within 1 month post-FMT and assessment of engraftment of donor microbiome and Assessment of engraftment of donor microbiome.
To evaluate the change in the stool microbiome pre-FMT vs post FMT.
Measured: Within 1 month
Treatment-Emergent Adverse Events
Preliminary evaluation of the safety of FMT in CGD-IBD.
Measured: Through end of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2022
- Study start8 Aug 2022
- Primary completion1 Mar 2027 (estimated)
- Study completion1 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Granulomatous Disease-associated ColitisStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Suchitra K Hourigan, M.D., Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05333471
- Sponsor's protocol ID
- 10000809
- Other IDs
- 000809-I
- Status last verified
- 13 Feb 2026
- First submitted
- 15 Apr 2022
- Last updated
- 31 Mar 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Raw, de-identified data may be shared as part of publication. This may include microbiome and metabolome data.
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Fecal Microbiota Transplantation for Chronic Granulomatous Disease-Associated Colitis
- ClinicalTrials.gov ID
- NCT05333471
- Acronym / sponsor ID
- 10000809
- Keywords
- Microbiome, Inflammation, Fecal Calprotectin, Immunodeficiency
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- 8 Aug 2022 (actual)
- Primary completion
- 1 Mar 2027 (estimated)
- Study completion
- 1 Mar 2027 (estimated)
- First posted
- 19 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Interventional | Experimental | Drug: MTP 101-LFAs 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). |
Who may qualify
- Ages
- 10 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (12 listed)
- 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 definitively related to CGD-IBD.
- History of fistulizing CGD-IBD or CGD-IBD intra-abdominal abscesses.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MTP 101-LFEach unit of MTP-101-LF contains approximately 35 mL of fecal transplant product. Participants will receive approximately 32 mL via colonoscopy. | Drug | Interventional |
Primary outcome
Difference in fecal calprotectin pre FMT and within 1 month post FMT.
Time frame: Within 1 month
To evaluate the change in intestinal inflammation pre-FMT vs post FMT
3 secondary outcomes
Difference in PRO-2 pre-FMT and within 1 month post-FMT.
Time frame: Within 1 month
Differences in alpha diversity, beta diversity, and relative abundance of taxa pre-FMT within 1 month post-FMT and assessment of engraftment of donor microbiome and Assessment of engraftment of donor microbiome.
Time frame: Within 1 month
Treatment-Emergent Adverse Events
Time frame: Through end of study
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Raw, de-identified data may be shared as part of publication. This may include microbiome and metabolome data.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Suchitra K Hourigan, M.D. principal investigator
National Institute of Allergy and Infectious Diseases (NIAID)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing MTP 101-LF in people with Chronic Granulomatous Disease-associated Colitis, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 31 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Suchitra K Hourigan, M.D. ((240) 627-3995, suchitra.hourigan@nih.gov), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Difference in fecal calprotectin pre FMT and within 1 month post FMT. (time frame: Within 1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Mar 2026; AzTrials last refreshed its copy on 8 Nov 2026.