Fecal Microbiota Transplantation in Pediatric Ulcerative Colitis (UC) (FMT)

Repeated and Multiple Fecal Microbiota Transplantations in Active Pediatric Ulcerative Colitis (UC)

This study included two topics: one was to test the efficacy and safety of fecal microbiota transplants plus partial enteral nutrition (PEN) in refractory pediatric UC where conventional therapy has failed, and the other was to explore the efficacy and safety of FMT plus PEN as first-line therapy for pediatric active UC

Study Overview

• Status: Recruiting
• Conditions: Ulcerative Colitis
• Intervention / Treatment: Other: Fecal Microbiota Transplantation

Detailed Description

Recent studies have suggested that gut imbalance and deregulation of immunological responses plays a pivotal role in the disease development of UC, and that FMT could be a useful treatment. In the refractory ulcerative colitis group, our study is aims to explore FMT plus PEN in the treatment of refractory pediatric UC. In the induction stage of UC, standard therapy remained unchanged, FMT and PEN treatment are added, and the investigators hope the withdrawal of conventional drug therapy was gradually reduced. Refractory UC is defined as refractory to standard therapy (e.g., steroids, immunomodulators, cyclosporine, tacrolimus, or anti-TNF agents).

As a first-line treatment group for UC, our study is aims to explore FMT plus PEN as a first-line treatment for active UC in children. participants treated with FMT coupled with PEN are defined as the FMT group, and those treated with PEN coupled with mesalazine served as the PEN group.

FMT treatment is given for at least one course of FMT treatment. If repeated FMTs are received, it is usually at a 2 month interval.

All the participants received PEN (80% of total calories as a polymeric diet, Peptamen, Nestle, Vevey, and Switzerland) intervention to help induce and maintain clinical remission.

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Biao Zou; Phone Number: +8685726753; Email: 464021552@qq.com
• Study Contact Backup: Name: Sainan Shu, MD, PhD; Phone Number: shusainan@163.com; Email: shusainan@163.com

Study Locations

• China
  • Wuhan, China, 430030
    • Recruiting
    • Tongji Hospital
    • Contact: Sainan Shu, professor; Phone Number: 13886011908; Email: shusainan@163.com
    • Contact: Biao Zou; Phone Number: +8685726753; Email: 464021552@qq.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 16 years (Child)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

age of older than 2 years and younger than 16 years with no genetic diseases; as a first-line treatment group for UC, newly diagnosed with mild-to-moderate UC (defined by the PUCAI of >10 and≤64); In the refractory ulcerative colitis group, all refractory pediatric with mild-to-moderate UC (defined by the PUCAI of >10 and≤64) defined by children who failed conventional treatment (hormone, immunosuppressant, biologics); agree to received regularly colonoscopy

Exclusion Criteria:

Children who were treated by PEN (80%) less than 8 weeks; As a first-line treatment group for UC, patients who were treated with corticosteroids, methotrexate, thiopurines, and anti-TNF agents as their first-line treatment; Known contraindication to all FMT infusion method such as nasoduodenal tube insertion, oesophago-gastro-duodenoscopy (OGD), enteroscopy, colonoscopy, enema and Fecal capsule; Unwilling to give informed consent/ assent

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: FMT group; In the refractory ulcerative colitis group, our study is aims to explore repeated and multiple FMTs plus PEN in the treatment of refractory pediatric UC;

Other: Fecal Microbiota Transplantation; In the induction stage of UC, FMT group received FMT and PEN intervention, and FMT group are given for at least one course of FMT treatment. If repeated FMTs are received, it is usually at a 2 month interval. All the participants received PEN (80% of total calories as a polymeric diet, Peptamen, Nestle, Vevey, and Switzerland) intervention to help induce and maintain clinical remission.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
clinical response	reduction in the Pediatric Ulcerative Colitis Activity Index (PUCAI) ≥30% from baseline	8-12 weeks after FMT
clinical remission	Clinical remission defined as a PUCAI <10	8-12 weeks after FMT
safety of FMT	All possible adverse events: fever, abdominal pain, infectious diseases and others.	8-12 weeks after FMT

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients requiring escalation of medical therapies	Number of patients requiring escalation of medical therapies based on clinical relapse. Clinical relapse is defined by requiring additional medical therapy.	8-12 weeks after FMT
Number of patients with endoscopic remission	Number of patients with endoscopic remission as defined by a PUCAI score of 0	8-12 weeks after FMT
Fecal calprotectin level	Mean change of Fecal calprotectin levels	8-12 weeks after FMT
C-reactive protein levels	Mean change of C-reactive protein levels	8-12 weeks after FMT
erythrocyte sedimentation rate (ESR) level	Mean change of erythrocyte sedimentation rate (ESR)	8-12 weeks after FMT
The number of stools or bloody stools	Improvement in the number of stools or bloody stools	8-12 weeks after FMT
gut microbial	Fecal 16S RNA or macrogene sequencing was performed. Fecal samples were obtained from donor and recipient. The fecal samples and isolated microbiota samples were frozen immediately and underwent DNA extraction using standard methods.	before treatment and 4 weeks after treatment

Collaborators and Investigators

• Sponsor: Biao Zou
• Investigators: Study Director: Zhihua Huang, Tongji Hospital

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2022
• Primary Completion (Estimated): June 30, 2025
• Study Completion (Estimated): June 30, 2025

Study Registration Dates

• First Submitted: December 25, 2022
• First Submitted That Met QC Criteria: December 25, 2022
• First Posted (Actual): January 11, 2023

Study Record Updates

• Last Update Posted (Actual): October 13, 2023
• Last Update Submitted That Met QC Criteria: October 11, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Fecal microbiota transplantation, Ulcerative Colitis
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Gastrointestinal Diseases; Gastroenteritis; Colonic Diseases; Intestinal Diseases; Inflammatory Bowel Diseases; Ulcer; Colitis; Colitis, Ulcerative
• Other Study ID Numbers: 63639979

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