Ganglioside and IBD

Assessing Whether Buttermilk Powder Improves Disease Activity in Pediatric IBD

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.

Study Overview

• Status: Recruiting
• Conditions: Inflammatory Bowel Diseases
• Intervention / Treatment: Other: Buttermilk Powder; Other: Anhydrous Milk Fat

• Study Type: Interventional
• Enrollment (Anticipated): 48
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Megan A Bailey; Phone Number: 714-509-8869; Email: mebailey@choc.org

Study Locations

• United States
  • California
    • Orange, California, United States, 92868
      • Recruiting
      • Children's Hospital of Orange County

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 9 years to 21 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: PARALLEL
• Masking: DOUBLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
ACTIVE_COMPARATOR: Treatment; 5 grams of buttermilk powder daily for 10 weeks, oral.	Other: Buttermilk Powder; Treatment with 5.0 g of buttermilk powder daily for 10 weeks.
PLACEBO_COMPARATOR: Placebo; 5 grams of milk powder (10% buttermilk powder, 90% anhydrous milk fat) daily for 10 weeks, oral.	Other: Anhydrous Milk Fat; Treatment with 5.0 g of milk powder (90% anhydrous milk fat, 10% buttermilk powder) daily for 10 weeks.

What is the study measuring?

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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hemoglobin, study start	g/L, from metabolic panel	Day 0
Hemoglobin, study end	g/L, from metabolic panel	Day 70
Platelets, study start	#, from metabolic panel	Day 0
Platelets, study end	#, from metabolic panel	Day 70
White blood cells, study start	#, from metabolic panel	Day 0
White blood cells, study end	#, from metabolic panel	Day 70
Albumin, study start	g/L, from metabolic panel	Day 0
Albumin, study end	g/L, from metabolic panel	Day 70
Hematocrit, study start	proportion, from metabolic panel	Day 0
Hematocrit, study end	proportion, from metabolic panel	Day 70

Collaborators and Investigators

• Sponsor: Children's Hospital of Orange County

Study record dates

Study Major Dates

• Study Start (ACTUAL): June 28, 2021
• Primary Completion (ANTICIPATED): June 15, 2023
• Study Completion (ANTICIPATED): September 15, 2023

Study Registration Dates

• First Submitted: February 23, 2021
• First Submitted That Met QC Criteria: March 1, 2021
• First Posted (ACTUAL): March 4, 2021

Study Record Updates

• Last Update Posted (ACTUAL): July 27, 2022
• Last Update Submitted That Met QC Criteria: July 25, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Intestinal Diseases; Inflammatory Bowel Diseases
• Other Study ID Numbers: 200109

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No