Ganglioside and IBD

Study Overview
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Brief Summary
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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.

Official Title
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Assessing Whether Buttermilk Powder Improves Disease Activity in Pediatric IBD

Conditions
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Inflammatory Bowel Diseases

Intervention / Treatment
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* Other: Buttermilk Powder
* Other: Anhydrous Milk Fat


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participants age 9-21 years 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 Ileal, ileocolonic, colonic location of disease Exclusion Criteria: Pregnancy Previous bowel resection Non-ileocolonic location of disease Inadequate liver or renal function On prescription medication for active infectious disease Drug/alcohol abuse Other serious medical conditions

Ages Eligible for Study
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Minimum Age: 9 Years
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Treatment<br>5 grams of buttermilk powder daily for 10 weeks, oral. | Other: Buttermilk Powder<br>* Treatment with 5.0 g of buttermilk powder daily for 10 weeks.<br>|
| Placebo Comparator: Placebo<br>5 grams of milk powder (10% buttermilk powder, 90% anhydrous milk fat) daily for 10 weeks, oral. | Other: Anhydrous Milk Fat<br>* Treatment with 5.0 g of milk powder (90% anhydrous milk fat, 10% buttermilk powder) daily for 10 weeks.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Disease activity index | Pediatric 1) Crohn Disease or 2) Ulcerative Colitis disease activity index | Day 0 | 
| Disease activity index | Pediatric 1) Crohn Disease or 2) Ulcerative Colitis disease activity index | Day 70 | 

Secondary Outcome Measures

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