Elemental Diet for Treatment of Inflammatory Bowel Disease in Patients With Chronic Granulomatous Disease

NCT03983837 | Completed Phase 2

• 2 other identifiers: 19010819-I-0108
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Chronic Granulomatous Disease; Inflammatory Bowel Disease

Keywords

Inflammatory Bowel Disease; Crohn disease; Ulcerative Colitis; Glucocorticoids; Microbiome

Outcome Measures

Primary Outcomes (1)

• 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.

  Day 56

Secondary Outcomes (7)

• Unexpected AEs possibly, probably, or definitely related to the elemental diet.

  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.

  Day 0 to 27

• Weight loss.

  Day 0 to 56

• Initiation of antibiotics.

  Day 0 to 56

• Changes in fecal calprotectin, ESR, and CRP.

  Day 56

Study Arms (1)

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.

Other: Neocate Splash

Interventions

Neocate Splash OTHER

Neocate Splash is a hypoallergenic, amino acid based elemental liquid diet that is taken orally.

1

Eligibility Criteria

• Age: 8 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• National Institute of Allergy and Infectious Diseases (NIAID): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• 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.

  PMID: 18410635 BACKGROUND

• Magnani 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.

  PMID: 24985400 BACKGROUND

• van 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.

  PMID: 19381301 BACKGROUND

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Granulomatous Disease, Chronic; Inflammatory Bowel Diseases; Crohn Disease; Colitis, Ulcerative

Condition Hierarchy (Ancestors)

Phagocyte Bactericidal Dysfunction; Leukocyte Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases; Colitis; Colonic Diseases

Study Officials

• Christa S Zerbe, M.D.

  National Institute of Allergy and Infectious Diseases (NIAID)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 7, 2019
• First Posted: June 12, 2019
• Study Start: June 23, 2021
• Primary Completion: November 18, 2025
• Study Completion: November 18, 2025
• Last Updated: December 11, 2025

Record last verified: 2025-12-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: CSR
• Time Frame: Data will be shared at the time of or shortly after publication.
• Access Criteria: Requests for data will be reviewed and approved/disapproved by the PI.

Locations

US (1)