Ganglioside and IBD

Not Applicable

Completed

• Conditions: Inflammatory Bowel Diseases
• Interventions: Other: Anhydrous Milk Fat; Other: Buttermilk Powder

• Registration Number: NCT04782544
• Lead Sponsor: Children's Hospital of Orange County

Brief Summary

Context. Inflammatory bowel disease (IBD) is a chronic and debilitating disorder. Novel treatment strategies aimed to resolve intestinal inflammation and induce disease remission are necessary. Dietary gangliosides are safe for consumption, bioavailable, and have shown clinical benefit in patients with inflammatory intestinal disease.

Objectives. The primary objective is this study is to determine the efficacy of dietary ganglioside in improving disease activity indices in pediatric patients with IBD. Secondary objectives include demonstrating the effectiveness of dietary ganglioside for improving quality of life, improving intestinal integrity, and reducing inflammation.

Study Design. Intervention: controlled trial (pilot). Participants. Inclusion criteria: aged 9-21 years, diagnosis of ulcerative colitis (UC) or ileal or colonic Crohn Disease (CD), active disease. Exclusion criteria: pregnancy, inadequate liver or renal function, active infectious disease, previous bowel resection, disease remission, drug/alcohol abuse, other serious medical conditions, indeterminate colitis.

Study Intervention. Patients with IBD (n=48) will be allocated to consume ganglioside treatment or placebo daily for 10 weeks. Treatment group will consume five grams of buttermilk powder daily or anhydrous milk fat.

Outcome Measures. The primary outcome is the disease activity index: pediatric Crohn's Disease Activity Index (pCDAI) or pediatric Ulcerative Colitis Activity Index (pUCAI). Secondary outcomes include: quality of life (IMPACT-III questionnaire), intestinal permeability (lactulose/mannitol urinary assay), C-reactive protein (CRP; blood exam), calprotectin (fecal exam).

Expected Outcomes. Relative to the placebo group, the treatment group will have improved disease activity indices, quality of life, and intestinal integrity over the 10-week study period. The treatment group will also show reduction in inflammation and calprotectin relative to the placebo group.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-02-23
• Study First Submitted QC: 2021-03-01
• Study First Posted: 2021-03-04
• Study Start: 2021-06-28
• Status Verified: 2022-07
• Primary Completion: 2023-09-15
• Study Completion: 2024-03-24
• Last Update Submitted: 2025-03-11
• Last Update Posted: 2025-03-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

Inclusion Criteria

1. Participants age 9-21 years
2. Mild-moderate IBD defined by ImproveCareNow (ICN) Physician Global Assessment (PGA); OR severe IBD defined by ICN PGA if stable for at least 120 days
3. Ileal, ileocolonic, colonic location of disease

Exclusion Criteria

1. Pregnancy
2. Previous bowel resection
3. Non-ileocolonic location of disease
4. Inadequate liver or renal function
5. On prescription medication for active infectious disease
6. Drug/alcohol abuse
7. Other serious medical conditions

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Anhydrous Milk Fat	5 grams of milk powder (10% buttermilk powder, 90% anhydrous milk fat) daily for 10 weeks, oral.
Treatment	Buttermilk Powder	5 grams of buttermilk powder daily for 10 weeks, oral.

Primary Outcome Measures

Name	Time	Method
Disease activity index	Day 70	Pediatric 1) Crohn Disease or 2) Ulcerative Colitis disease activity index

Secondary Outcome Measures

Name	Time	Method
C-reactive protein	Day 70	Blood test
ImproveCareNow Physician Global Assessment	Day 70	A disease activity index
Intestinal integrity	Day 70	Intestinal permeability (lactulose/mannitol) challenge
IMPACT-III questionnaire	Day 70	quality of life scales (0 = lowest, 100 = highest): well-being, emotional functioning, social functioning, body image
Calprotectin	Day 70	Stool test

Trial Locations

Locations (1)

Children's Hospital of Orange County; 🇺🇸 Orange, California, United States