Clinical trial NCT04671186: Role of Probiotics in Treatment of Pediatric NAFLD Patients by Assessing With Fibroscan
Official title: Role of Probiotics in Treatment of Pediatric Nonalcoholic Fatty Liver Disease (NAFLD) Patients by Assessing With Fibroscan
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Non-Alcoholic Fatty Liver Disease
Lead sponsor: Northwell Health
Sponsor class: OTHER
First posted: 2020-12-17
Enrollment: 47
Sex: ALL
Minimum age (years): 5
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Participants will be blindly randomized at Visit 1 into one of two groups (probiotic or placebo). Probiotic group will receive Lactobacillus rhamnosus strain GG one capsule oral daily (10 billion CFU/day) and the other group receive placebo oral capsule daily throughout the study. All participants in either group will undergo: Life style modification as per NASPGHAN NAFLD guidelines. Fibroscan assessing CAP and TE score once every 3 month until the end of study. Monitoring ALT every 3 months until end of study as per standard of care guidelines.Collection of stool sample to be sent for microbiome analysis at the time of diagnosis, 3 months and 6 months (end of the study). Secondary variables collected, Age, Gender, Race, BMI, Fibroscan probe.
Primary purpose: TREATMENT

Brief summary: The goal of this study is to evaluate the role of probiotics in the treatment of pediatric NAFLD by evaluating for steatosis and fibrosis with a Fibroscan machine. This will be done by performing a fibroscan during each visit and comparing patients' ALT at various intervals. This is an important study as current pediatric guidelines only recommend lifestyle modifications for the treatment of NAFLD and the use of ALT and sonogram to assess improvement as standard of care. Investigators hypothesized that treatment with a probiotic will demonstrate an improvement in NAFLD as assessed by a fibroscan of liver which is good for the monitoring of steatosis and fibrosis. Additionally, analysis of fecal microbiome results may offer insight into targeted therapy in the future.

Detailed description: The goal/objective of this study is to evaluate the role of probiotics in the treatment of NAFLD by assessing with fibroscan in both newly diagnosed and known pediatric patients with NAFLD. This will be done by performing a fibroscan during each visit and comparing patient ALT and fecal microbiome at various intervals.

The primary endpoint is to assess the effect of probiotic treatment in liver steatosis and fibrosis by assessing with fibroscan using CAP score and TE staging. The higher the CAP score (S1, S2, S3) so worse the steatosis. TE staging (F0-F4), higher the staging indicates worsening of fibrosis.

Secondary endpoints include:

* The rate of decline of ALT over 3 month time interval and through whole study starting at initial diagnosis.
* The rate of decrease in hepatic steatosis and BMI

This study will target pediatric patients with NAFLD. Participants will be new and previously diagnosed NAFLD patients between the ages of 5 years and 18 years.

This is single center study. Investigators will enroll patients from The Division of Pediatric Gastroenterology, Liver Disease and Nutrition.

Participants will be blindly randomized into one of two groups: probiotic or placebo.

Investigators expect the study to run for 1 year from 09/15/2020 to 09/14/2021. Investigators will enroll patients over a 6 month period. After randomization into two separate groups (probiotic vs placebo), Investigators plan to collect data for the subsequent 6 months following enrollment.

Eligibility criteria: Inclusion Criteria:

* This study will target pediatric patients with NAFLD. Participants will be new and previously diagnosed NAFLD patients between the ages of 5 years and 18 years. Diagnosis must be made by elevated serum ALT (two times above the sex specific upper limit of normal) and a sonogram consistent with steatosis

Exclusion Criteria:

* Exclusion criteria for both groups

  * Concomitant underlying liver disease such as but not limited to autoimmune hepatitis
  * Concomitant infectious hepatitis
  * Medication use of steroids, methotrexate, metformin, and therapeutic dose of Vit. E
  * Recent antibiotic use in last 4 weeks
  * Clinically significant weight loss (at least 5% reduction in weight from baseline level \[18\]) on follow up NAFLD patients who were on life style modification intervention
  * Cirrhosis (Fibroscan score ≥14.0)\>

Primary outcomes:
measure: The role of probiotics in the treatment of pediatric NAFLD by evaluating for steatosis with a Fibroscan machine.
timeFrame: One year
measure: The role of probiotics in the treatment of pediatric NAFLD by evaluating for fibrosis with a Fibroscan machine.
timeFrame: One year
Secondary outcomes:
measure: Rate of decline in ALT.
timeFrame: One year
measure: Change in fecal microbiome
timeFrame: One year
measure: Decrease in BMI
timeFrame: One year
Other outcomes:
Locations:
city: New Hyde Park
country: United States
facility: Cohen Children's Medical Center
state: New York
Study officials:
affiliation: Cohen Children's Medical Center
name: Shari Sheflin-Findling
role: PRINCIPAL_INVESTIGATOR
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