Chronic Granulomatous DiseasePhase 2NCT03983837
Elemental Diet for Treatment of Inflammatory Bowel Disease in Patients With Chronic Granulomatous Disease
A Phase 2 study testing Neocate Splash in people with Chronic Granulomatous Disease and Inflammatory Bowel Disease, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial summary
Background: Bacteria that live inside the stomach and intestines are important for health. Chronic granulomatous disease (CGD) and inflammatory bowel disease (IBD) can make people have unhealthy bacteria. This can lead to gastrointestinal (GI) problems. Read more about this study
- Who is it for?
- Ages 8 to 65 years
- Conditions the study is for
- Chronic Granulomatous Disease, Inflammatory Bowel Disease
- What is being tested?
- Neocate Splash
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Jun 2021to 18 Nov 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 8 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Individuals must meet all of the following criteria to be eligible for study participation:
- Aged 8-65 years.
- Have confirmed diagnoses of CGD, CGD-associated colitis, and IBD.
- Have a Simple Endoscopic Score for Crohn Disease (SES-CD) > 3 and/or HBI >= 5.
- Able to provide informed consent.
- Willing to allow storage of biological samples for future research.
- Willing to allow genetic testing on biological samples.
- Willing to be admitted as inpatient for up to 2 weeks to initiate the elemental diet.
Who cannot join
- Pregnancy.
- Active or ongoing infection that requires antibiotics other than the participant s known prophylaxis medications.
- Any condition that, in the opinion of the investigator, contraindicates participation in this study will be excluded from study participation.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Background:
Bacteria that live inside the stomach and intestines are important for health. Chronic granulomatous disease (CGD) and inflammatory bowel disease (IBD) can make people have unhealthy bacteria. This can lead to gastrointestinal (GI) problems. Researchers want to see if people with CGD and IBD feel better when they change the bacteria in the stomach by following a special liquid diet.
Read more
Objective:
To see if an elemental diet can change the bacteria in the stomach and intestines of people with CGD and IBD. Also, to see if this helps GI symptoms.
Eligibility:
People ages 8-65 years with CGD, CGD-associated colitis, and IBD.
Design:
Participants will first be screened with:
Upper GI endoscopy and/or colonoscopy. A long, thin tube with a tiny camera at the end will be passed into the participant s body through the mouth or anus. Tissue will be collected. Participants will be sedated for the procedure. They will be sedated using a special mask or small plastic tube placed in an arm vein using a needle.
Participants will be put on the special diet for up to 4 weeks. They will stay in the hospital for up to 2 weeks. They will have check-ups. They will have blood, urine, and stool samples collected. They will keep a symptom diary to record how they feel and any GI symptoms.
Participants will have 2 follow-up visits. The first will be right after they finish the diet. The second will be 4 weeks later. They will have blood, urine, and stool samples collected. They will learn about re-introducing other foods into their diet.
Read the detailed description
Chronic granulomatous disease (CGD) is a rare genetic immune disorder that results in recurrent and severe infections, and inflammatory dysregulation. For almost 50% of patients with CGD, this inflammation causes severe inflammatory bowel disease (IBD). However, treatment is long term and challenging as CGD-IBD is prone to relapse, and use of immune modulators must be balanced with increased risk of infectious complications. Conventional IBD (Crohn disease and ulcerative colitis) is likely caused by an inappropriate inflammatory response to intestinal microbes, which is influenced by the environment in a genetically susceptible host. While it is not yet clear if intestinal dysbiosis is a result or cause of intestinal inflammation, studies suggest that correcting intestinal dysbiosis may improve intestinal inflammation. Studies in patients with conventional IBD suggest that strict dietary restrictions can resolve symptoms of IBD and induce remission as effectively as glucocorticoids but without the side effects of those drugs. Because these diets alter the gut microbiome, they may serve as effective treatment for CGD-IBD.
In this study, we propose to treat 5 patients >= 8 years old who have CGD and IBD with 4 weeks of an elemental diet. Participants will stay as inpatients for up to 2 weeks and will return for follow-up at the end of the diet to begin tapering off and be reintroduced to normal food. The primary objective of this study is the evaluation of changes in the microbiome before and after the elemental diet. Secondary objectives are evaluations of tolerability and safety of the diet, along with changes in local and systemic markers of inflammation, and improvement of IBD symptoms as determined by reduction in Harvey-Bradshaw Index score.
- Being tested
- Neocate Splash
- Keywords
- Inflammatory Bowel Disease, Crohn disease, Ulcerative Colitis, Glucocorticoids, Microbiome
- Medical subject headings
- Granulomatous Disease, Chronic, Inflammatory Bowel Diseases, Crohn Disease, Colitis, Ulcerative
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Neocate SplashOther
Neocate Splash is a hypoallergenic, amino acid based elemental liquid diet that is taken orally.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Participants will be on this diet for 4 weeks. The amount per serving will be determined on the basis of the participant s weight and caloric needs, as determined by a staff dietician. Receives: Other: Neocate Splash |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in the relative abundance of proteobacteria in stool samples obtained after elemental diet.
Change in the relative abundance of proteobacteria in stool samples obtained after elemental diet.
Measured: Day 56
Secondary outcomes (7)
Unexpected AEs possibly, probably, or definitely related to the elemental diet.
Measured: Day 0 to 56
Ability to tolerate 4 weeks of elemental diet as measured by > 75% of caloric intake via elemental diet over the 4-week period.
Measured: Day 0 to 27
Weight loss.
Measured: Day 0 to 56
Initiation of antibiotics.
Measured: Day 0 to 56
Changes in fecal calprotectin, ESR, and CRP.
Measured: Day 56
Changes in the HBI at completion of the elemental diet and at 4 weeks after completion.
Measured: Day 28 and 56
Change in alpha and beta diversity after diet
Measured: Day 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2019
- Study start23 Jun 2021
- Primary completion18 Nov 2025
- Study completion18 Nov 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Chronic Granulomatous DiseaseStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Christa S Zerbe, M.D., Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03983837
- Sponsor's protocol ID
- 190108
- Other IDs
- 19-I-0108
- Status last verified
- 9 Dec 2025
- First submitted
- 7 Jun 2019
- Last updated
- 11 Dec 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Human data generated in this study will be shared for future research as follows:@@@-De-identified data in an NIH-funded or approved public repository.@@@-De-identified data in another public repository. @@@-Identified data in the Biomedical Translational Research Information System (automatic for activities in the CC).@@@-De-identified or identified data with approved outside collaborators under appropriate agreements.
Publications
Jones LB, McGrogan P, Flood TJ, Gennery AR, Morton L, Thrasher A, Goldblatt D, Parker L, Cant AJ. Special article: chronic granulomatous disease in the United Kingdom and Ireland: a comprehensive national patient-based registry. Clin Exp Immunol. 2008 May;152(2):211-8. doi: 10.1111/j.1365-2249.2008.03644.x.
PubMed 18410635Magnani A, Brosselin P, Beaute J, de Vergnes N, Mouy R, Debre M, Suarez F, Hermine O, Lortholary O, Blanche S, Fischer A, Mahlaoui N. Inflammatory manifestations in a single-center cohort of patients with chronic granulomatous disease. J Allergy Clin Immunol. 2014 Sep;134(3):655-662.e8. doi: 10.1016/j.jaci.2014.04.014. Epub 2014 Jun 27.
PubMed 24985400van den Berg JM, van Koppen E, Ahlin A, Belohradsky BH, Bernatowska E, Corbeel L, Espanol T, Fischer A, Kurenko-Deptuch M, Mouy R, Petropoulou T, Roesler J, Seger R, Stasia MJ, Valerius NH, Weening RS, Wolach B, Roos D, Kuijpers TW. Chronic granulomatous disease: the European experience. PLoS One. 2009;4(4):e5234. doi: 10.1371/journal.pone.0005234. Epub 2009 Apr 21.
PubMed 19381301
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Elemental Diet for Treatment of Inflammatory Bowel Disease in Patients With Chronic Granulomatous Disease
- ClinicalTrials.gov ID
- NCT03983837
- Acronym / sponsor ID
- 190108
- Keywords
- Inflammatory Bowel Disease, Crohn disease, Ulcerative Colitis, Glucocorticoids, Microbiome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- 23 Jun 2021 (actual)
- Primary completion
- 18 Nov 2025 (actual)
- Study completion
- 18 Nov 2025 (actual)
- First posted
- 12 Jun 2019 (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:
Bacteria that live inside the stomach and intestines are important for health. Chronic granulomatous disease (CGD) and inflammatory bowel disease (IBD) can make people have unhealthy bacteria. This can lead to gastrointestinal (GI) problems. Researchers want to see if people with CGD and IBD feel better when they change the bacteria in the stomach by following a special liquid diet.
Objective:
To see if an elemental diet can change the bacteria in the stomach and intestines of people with CGD and IBD. Also, to see if this helps GI symptoms.
Eligibility:
People ages 8-65 years with CGD, CGD-associated colitis, and IBD.
Design:
Participants will first be screened with:
Upper GI endoscopy and/or colonoscopy. A long, thin tube with a tiny camera at the end will be passed into the participant s body through the mouth or anus. Tissue will be collected. Participants will be sedated for the procedure. They will be sedated using a special mask or small plastic tube placed in an arm vein using a needle.
Participants will be put on the special diet for up to 4 weeks. They will stay in the hospital for up to 2 weeks. They will have check-ups. They will have blood, urine, and stool samples collected. They will keep a symptom diary to record how they feel and any GI symptoms.
Participants will have 2 follow-up visits. The first will be right after they finish the diet. The second will be 4 weeks later. They will have blood, urine, and stool samples collected. They will learn about re-introducing other foods into their diet.
Detailed description
Chronic granulomatous disease (CGD) is a rare genetic immune disorder that results in recurrent and severe infections, and inflammatory dysregulation. For almost 50% of patients with CGD, this inflammation causes severe inflammatory bowel disease (IBD). However, treatment is long term and challenging as CGD-IBD is prone to relapse, and use of immune modulators must be balanced with increased risk of infectious complications. Conventional IBD (Crohn disease and ulcerative colitis) is likely caused by an inappropriate inflammatory response to intestinal microbes, which is influenced by the environment in a genetically susceptible host. While it is not yet clear if intestinal dysbiosis is a result or cause of intestinal inflammation, studies suggest that correcting intestinal dysbiosis may improve intestinal inflammation. Studies in patients with conventional IBD suggest that strict dietary restrictions can resolve symptoms of IBD and induce remission as effectively as glucocorticoids but without the side effects of those drugs. Because these diets alter the gut microbiome, they may serve as effective treatment for CGD-IBD.
In this study, we propose to treat 5 patients >= 8 years old who have CGD and IBD with 4 weeks of an elemental diet. Participants will stay as inpatients for up to 2 weeks and will return for follow-up at the end of the diet to begin tapering off and be reintroduced to normal food. The primary objective of this study is the evaluation of changes in the microbiome before and after the elemental diet. Secondary objectives are evaluations of tolerability and safety of the diet, along with changes in local and systemic markers of inflammation, and improvement of IBD symptoms as determined by reduction in Harvey-Bradshaw Index score.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Other: Neocate SplashParticipants will be on this diet for 4 weeks. The amount per serving will be determined on the basis of the participant s weight and caloric needs, as determined by a staff dietician. |
Who may qualify
- Ages
- 8 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Individuals must meet all of the following criteria to be eligible for study participation:
- Aged 8-65 years.
- Have confirmed diagnoses of CGD, CGD-associated colitis, and IBD.
- Have a Simple Endoscopic Score for Crohn Disease (SES-CD) > 3 and/or HBI >= 5.
- Able to provide informed consent.
Key exclusion criteria (3 listed)
- Pregnancy.
- Active or ongoing infection that requires antibiotics other than the participant s known prophylaxis medications.
- Any condition that, in the opinion of the investigator, contraindicates participation in this study will be excluded from study participation.
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 |
|---|---|---|
| Neocate SplashNeocate Splash is a hypoallergenic, amino acid based elemental liquid diet that is taken orally. | Other | 1 |
Primary outcome
Change in the relative abundance of proteobacteria in stool samples obtained after elemental diet.
Time frame: Day 56
Change in the relative abundance of proteobacteria in stool samples obtained after elemental diet.
7 secondary outcomes
Unexpected AEs possibly, probably, or definitely related to the elemental diet.
Time frame: Day 0 to 56
Ability to tolerate 4 weeks of elemental diet as measured by > 75% of caloric intake via elemental diet over the 4-week period.
Time frame: Day 0 to 27
Weight loss.
Time frame: Day 0 to 56
Initiation of antibiotics.
Time frame: Day 0 to 56
Changes in fecal calprotectin, ESR, and CRP.
Time frame: Day 56
Changes in the HBI at completion of the elemental diet and at 4 weeks after completion.
Time frame: Day 28 and 56
Change in alpha and beta diversity after diet
Time frame: Day 56
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2025.
- Background: PubMed 18410635Jones LB, McGrogan P, Flood TJ, Gennery AR, Morton L, Thrasher A, Goldblatt D, Parker L, Cant AJ. Special article: chronic granulomatous disease in the United Kingdom and Ireland: a comprehensive national patient-based registry. Clin Exp Immunol. 2008 May;152(2):211-8. doi: 10.1111/j.1365-2249.2008.03644.x.
- Background: PubMed 24985400Magnani A, Brosselin P, Beaute J, de Vergnes N, Mouy R, Debre M, Suarez F, Hermine O, Lortholary O, Blanche S, Fischer A, Mahlaoui N. Inflammatory manifestations in a single-center cohort of patients with chronic granulomatous disease. J Allergy Clin Immunol. 2014 Sep;134(3):655-662.e8. doi: 10.1016/j.jaci.2014.04.014. Epub 2014 Jun 27.
- Background: PubMed 19381301van den Berg JM, van Koppen E, Ahlin A, Belohradsky BH, Bernatowska E, Corbeel L, Espanol T, Fischer A, Kurenko-Deptuch M, Mouy R, Petropoulou T, Roesler J, Seger R, Stasia MJ, Valerius NH, Weening RS, Wolach B, Roos D, Kuijpers TW. Chronic granulomatous disease: the European experience. PLoS One. 2009;4(4):e5234. doi: 10.1371/journal.pone.0005234. Epub 2009 Apr 21.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Human data generated in this study will be shared for future research as follows:@@@-De-identified data in an NIH-funded or approved public repository.@@@-De-identified data in another public repository. @@@-Identified data in the Biomedical Translational Research Information System (automatic for activities in the CC).@@@-De-identified or identified data with approved outside collaborators under appropriate agreements.
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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. This study is not recruiting at the moment.
Christa S Zerbe, 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 2 study testing Neocate Splash in people with Chronic Granulomatous Disease and Inflammatory Bowel Disease, 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 8 inclusion and 3 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: finished, as of the sponsor's last update on 11 Dec 2025. 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 phase mean?
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in the relative abundance of proteobacteria in stool samples obtained after elemental diet. (time frame: Day 56). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03983837, which also lists 3 linked publications.
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 11 Dec 2025.