Clinical trial NCT04782544: Ganglioside and IBD
Official title: Assessing Whether Buttermilk Powder Improves Disease Activity in Pediatric IBD
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Inflammatory Bowel Diseases
Lead sponsor: Children's Hospital of Orange County
Sponsor class: OTHER
First posted: 2021-03-04
Enrollment: 11
Sex: ALL
Minimum age (years): 9
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Disease activity index
timeFrame: Day 0
measure: Disease activity index
timeFrame: Day 70
Secondary outcomes:
measure: ImproveCareNow Physician Global Assessment
timeFrame: Day 0
measure: ImproveCareNow Physician Global Assessment
timeFrame: Day 70
measure: IMPACT-III questionnaire
timeFrame: Day 0
measure: IMPACT-III questionnaire
timeFrame: Day 70
measure: Intestinal integrity
timeFrame: Day 0
measure: Intestinal integrity
timeFrame: Day 70
measure: Calprotectin
timeFrame: Day 0
measure: Calprotectin
timeFrame: Day 70
measure: C-reactive protein
timeFrame: Day 0
measure: C-reactive protein
timeFrame: Day 70
Other outcomes:
measure: Hemoglobin, study start
timeFrame: Day 0
measure: Hemoglobin, study end
timeFrame: Day 70
measure: Platelets, study start
timeFrame: Day 0
measure: Platelets, study end
timeFrame: Day 70
measure: White blood cells, study start
timeFrame: Day 0
measure: White blood cells, study end
timeFrame: Day 70
measure: Albumin, study start
timeFrame: Day 0
measure: Albumin, study end
timeFrame: Day 70
measure: Hematocrit, study start
timeFrame: Day 0
measure: Hematocrit, study end
timeFrame: Day 70
Locations:
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
Study officials:
References: