Fecal Microbiota Transplantation (FMT) for Treatment of Ulcerative Colitis in Children

NCT01947101 | Completed Phase 1 DMC

• 2 other identifiers: H-30591FMT- Ulcerative Colitis
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

Patients diagnosed with ulcerative colitis (UC), a specific type of inflammatory bowel disease, will be invited to take part in this study. The investigators do not know what causes UC. However, the microbes (such as bacteria and viruses), which normally live in our intestines are thought to play an important role in the development of UC. There are many treatment options for UC such as steroids,and other medications that decrease inflammation. However, none of these can cure the disease. This study aims to treat pediatric UC in an alternative fashion by changing the microbes in the gut by giving the participant's stool specimens from healthy adult individuals. There is some evidence from a few cases of adult UC that this therapy may cure the disease or at least provide long lasting suppression (remission) of the symptoms. The investigators would like to test this therapeutic intervention in children with UC. The purpose of this study is to (1) examine the microbiome of stool and colon (2) and to determine whether healthy-donated stool enemas can treat pediatric ulcerative colitis. (3) The investigators will also study the effects of the treatment on colonic gene expression.

Conditions

Ulcerative Colitis

Keywords

Stool transplant; Ulcerative Colitis; Fecal transplant; Inflammatory Bowel Disease

Outcome Measures

Primary Outcomes (1)

• Assess safety of FMT treatment by recording the frequency of adverse events.

  To determine the safety and associated toxicities of serial FMTs in pediatric UC following withdrawal from standard therapy.

  12 months

Secondary Outcomes (1)

• Assess efficacy of FMT treatment with the aid of the Pediatric Ulcerative Colitis Activity (PUCAI) Index, a validated measure of clinical disease severity.

  1 year

Study Arms (1)

Ulcerative Colitis

EXPERIMENTAL

Fecal Microbiota Transplant

Biological: Fecal Microbiota Transplant

Interventions

Fecal Microbiota Transplant BIOLOGICAL

Fecal Microbiota Transplant will be endoscopically administered directly into the colon. Consecutive treatments will be given by rectal enema route.

Also known as: stool transplant

Ulcerative Colitis

Eligibility Criteria

• Age: 12 Years - 20 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients must have a diagnosis of ulcerative colitis based on clinical symptoms, as well as laboratory and colonoscopic findings, including histopathologic results of the intestinal mucosa.
• Pediatric Ulcerative Colitis Activity Index (PUCAI) \< 35 \[mild activity\] as assessed within 4 weeks prior to enrollment and clinical symptoms that are at least stable.
• Patients must be "immunomodulator dependent," i.e., have a history of steroid dependency (lack of clinical remission for over 8 weeks without steroid therapy), and/or being immunomodulator (azathioprine, 6-mercaptopurine, methotrexate, etc.) treated, and/or biologic agent (infliximab, adalimumab, etc.) treated
• Patients ≥ 12 and \< 21 years of age are eligible for this trial.
• Patients must be willing to be off current UC therapies.
• Willingness to undergo a surgical consultation prior to FMT treatment.
• Patients who are not enema trained will be required to undergo enema training prior to the start of FMT treatment.
• All patients and/or their parents or legally authorized representatives must sign a written informed consent document. Assent, when appropriate, will be obtained according to institutional guidelines.

You may not qualify if:

• Patients who are known to have the following will be excluded:
• Decompensated liver cirrhosis (bleeding varices, ascites, encephalopathy or icterus)
• Human immunodeficiency virus (HIV)/acquired immune deficiency syndrome (AIDS)
• Bone marrow transplantation within the past 150 days
• Other severe immunodeficiency
• Patients with severe prior allergic reaction to food will be excluded from the protocol.
• Patients who are pregnant or lactating will be excluded from the protocol.
• ≥ 18 years and \< 45 years
• Willing to provide stool and blood samples for laboratory testing.
• Body mass index \< 30
• Willing to fill out a detailed health screening questionnaire

Sponsors & Collaborators

• Baylor College of Medicine: lead

Study Sites (1)

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Colitis, Ulcerative; Inflammatory Bowel Diseases

Interventions

Fecal Microbiota Transplantation

Condition Hierarchy (Ancestors)

Colitis; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Colonic Diseases; Intestinal Diseases

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Officials

• Richard Kellermayer, MD

  Baylor College of Medicine - Texas Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: September 13, 2013
• First Posted: September 20, 2013
• Study Start: February 1, 2014
• Primary Completion: December 1, 2014
• Study Completion: May 1, 2016
• Last Updated: June 14, 2016

Record last verified: 2016-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)