Fecal Microbiota Transplantation (FMT) in the Management of Ulcerative Colitis (UC)
Primary Purpose
Ulcerative Colitis
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Fecal Microbiota Transplantation
Sponsored by
About this trial
This is an interventional treatment trial for Ulcerative Colitis focused on measuring Fecal Microbiota Transplantation, Ulcerative Colitis
Eligibility Criteria
Inclusion Criteria:
- Patients with biopsy proven ulcerative colitis (UC), including those with inadequately controlled UC (flare) as defined by failure of standard medical therapy, steroid-dependence, and/or need for escalation of medical care as determined by severity index (Mayo Score), endoscopic or histologic study, and/or medical provider
- Have active disease, defined with a Mayo Score > 3 and Mayo endoscopic subscore >1
- Subjects whom the investigator believes can and will comply with the requirements of the protocol
- Able to provide informed written consent.
Exclusion Criteria:
- Biopsy-proven Crohn's disease or indeterminate colitis
- Acute abdomen or other clinical emergencies requiring emergent management (for example: stricture, bowel obstruction, perforation and/or abscess)
- Primary sclerosing cholangitis (PSC)
- Pregnancy
- Concurrent Clostridium difficile infection or other known infection
- Prior history of fecal microbiota transplantation
- Other causes of diarrhea, including but not limited to tube feeds and medications (for example, kayaxelate, metformin, lactulose, laxatives, magnesium)
- Major congenital defects
- Subjects with recent malignancy in the last 5 years, excluding non-melanoma skin malignancies
- Anaphylactic reactions to any foods
- Any antibiotic use within the last 3 months
- Subject having any other condition that, in the opinion of the investigator, would jeopardize the safety or rights of the participant participating in the study, would make it unlikely for the participant to complete the study, or would confound the study
Sites / Locations
- Weill Cornell Medical College
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Fecal Microbiota Transplantation
Arm Description
Individuals with Ulcerative Colitis will undergo a fecal microbiota transplantation.
Outcomes
Primary Outcome Measures
Safety post-FMT as determined by interview for adverse events
Patient information regarding adverse events and safety of FMT for UC will be collected throughout the study period, including day 0, weeks 1, 2, 4, 6, 12 24, and then every 6 months until 36 months post-FMT. Throughout the study period, patients will be assessed for safety with questions regarding general well-being (such as "how have you been feeling?"), as well as specific questions to evaluate for occurrence of adverse events. Patients will also be questioned regarding stool form and frequency, presence of abdominal pain, fevers and subjective well-being.
Secondary Outcome Measures
Clinical remission
Defined by Mayo score ≤ 2 without any subscore >1, and Mayo endoscopic subscore 0-1
Clinical Response
Defined by decrease in Mayo score by 3 points, decrease in bleeding subscore by 1, or absolute subscore of 0-1
Progression of disease defined by initiation of anti-TNF agents
Initiation of anti-TNF agents (such as infliximab, adalimumab, certolizumab), vedolizumab, steroids. Includes time gap until additional agents are started
Progression of disease defined by increase in dosages of current UC medications
Increase in dosages of current ulcerative colitis specific medications
Progression of disease defined by time to colectomy
Time to colectomy rates and increase in time to colectomy
Death secondary to UC
Time to death secondary to ulcerative colitis
Progression of disease defined by clinical flare
Time to next flare
Microbial changes
- Alterations in microbial profiles as defined by sequence of genetic material from fecal material.
Immunological changes
- Alterations in immune cell function as defined by RNA sequencing and flow cytometry
Full Information
NCT ID
NCT02516384
First Posted
July 29, 2015
Last Updated
February 20, 2018
Sponsor
Weill Medical College of Cornell University
1. Study Identification
Unique Protocol Identification Number
NCT02516384
Brief Title
Fecal Microbiota Transplantation (FMT) in the Management of Ulcerative Colitis (UC)
Official Title
Fecal Microbiota Transplantation (FMT) in the Management of Ulcerative Colitis (UC)
Study Type
Interventional
2. Study Status
Record Verification Date
February 2018
Overall Recruitment Status
Completed
Study Start Date
October 1, 2016 (Actual)
Primary Completion Date
December 31, 2016 (Actual)
Study Completion Date
January 31, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Weill Medical College of Cornell University
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Inflammatory bowel disease is a condition caused by gastrointestinal immune system dysregulation and affected by both genetic and environmental factors. Differences in intestinal bacteria exist between IBD patients and healthy controls, but the role of intestinal bacteria in the development and treatment of IBD remains largely unknown. Fecal microbiota transplantation (FMT) is the transfer of gastrointestinal bacteria from a healthy donor to a patient with altered microbial diversity with the intent of restoring a normal bacterial balance. Most studies focus on its use in treating Clostridium difficile (CDI), an infection characterized by dysbiosis. Given the role of dysbiosis in IBD, the investigators hypothesize that FMT may be beneficial in IBD. The purpose of this study is to prospectively examine the safety of FMT in the management of ulcerative colitis (UC).
Detailed Description
Ulcerative colitis (UC) is a chronic inflammatory bowel disease (IBD) with significant morbidity and mortality. Current therapies remain limited by side effects and loss of response over time, and there is an ongoing need for new therapies. Fecal microbiota transplantation (FMT), which has proven to be safe and effective in the management of Clostridium difficile infection (CDI) has been proposed as a therapy for UC. There have been studies examining the role of FMT in UC, but they have shown mixed results, and have not examined the underlying immunologic and microbial changes to explain how and why FMT works from specific donors and in certain recipients. Furthermore, no studies have examined the long-term safety of FMT in patients with UC. This proposal aims to examine: (a) the short- and long-term safety of FMT in patients with UC, (b) the efficacy of FMT as a therapy for mild-moderate UC, and (c) the microbial and immunologic changes that occur after FMT, to help understand how and why it works in this group of patients.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Ulcerative Colitis
Keywords
Fecal Microbiota Transplantation, Ulcerative Colitis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Fecal Microbiota Transplantation
Arm Type
Experimental
Arm Description
Individuals with Ulcerative Colitis will undergo a fecal microbiota transplantation.
Intervention Type
Biological
Intervention Name(s)
Fecal Microbiota Transplantation
Intervention Description
We will use fecal microbiota transplantation (FMT), with fecal material obtained from OpenBiome or donor directed, to assess safety (as primary outcome) and efficacy (as secondary outcome) in adult (>18 year old) patients with active ulcerative colitis (UC).
Primary Outcome Measure Information:
Title
Safety post-FMT as determined by interview for adverse events
Description
Patient information regarding adverse events and safety of FMT for UC will be collected throughout the study period, including day 0, weeks 1, 2, 4, 6, 12 24, and then every 6 months until 36 months post-FMT. Throughout the study period, patients will be assessed for safety with questions regarding general well-being (such as "how have you been feeling?"), as well as specific questions to evaluate for occurrence of adverse events. Patients will also be questioned regarding stool form and frequency, presence of abdominal pain, fevers and subjective well-being.
Time Frame
36 months post-FMT
Secondary Outcome Measure Information:
Title
Clinical remission
Description
Defined by Mayo score ≤ 2 without any subscore >1, and Mayo endoscopic subscore 0-1
Time Frame
2, 4 and 12 weeks post fecal microbiota transplantation
Title
Clinical Response
Description
Defined by decrease in Mayo score by 3 points, decrease in bleeding subscore by 1, or absolute subscore of 0-1
Time Frame
2, 4 and 12 weeks post fecal microbiota transplantation
Title
Progression of disease defined by initiation of anti-TNF agents
Description
Initiation of anti-TNF agents (such as infliximab, adalimumab, certolizumab), vedolizumab, steroids. Includes time gap until additional agents are started
Time Frame
2, 4 and 12 weeks post fecal microbiota transplantation
Title
Progression of disease defined by increase in dosages of current UC medications
Description
Increase in dosages of current ulcerative colitis specific medications
Time Frame
2, 4 and 12 weeks post fecal microbiota transplantation
Title
Progression of disease defined by time to colectomy
Description
Time to colectomy rates and increase in time to colectomy
Time Frame
up to three year follow-up period post fecal microbiota transplantation
Title
Death secondary to UC
Description
Time to death secondary to ulcerative colitis
Time Frame
Anytime during the three year follow-up period post fecal microbiota transplantation
Title
Progression of disease defined by clinical flare
Description
Time to next flare
Time Frame
2, 4 and 12 weeks post fecal microbiota transplantation
Title
Microbial changes
Description
- Alterations in microbial profiles as defined by sequence of genetic material from fecal material.
Time Frame
0, 2 and 4 weeks post fecal microbiota transplantation
Title
Immunological changes
Description
- Alterations in immune cell function as defined by RNA sequencing and flow cytometry
Time Frame
0, 2 and 4 weeks post fecal microbiota transplantation
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients with biopsy proven ulcerative colitis (UC), including those with inadequately controlled UC (flare) as defined by failure of standard medical therapy, steroid-dependence, and/or need for escalation of medical care as determined by severity index (Mayo Score), endoscopic or histologic study, and/or medical provider
Have active disease, defined with a Mayo Score > 3 and Mayo endoscopic subscore >1
Subjects whom the investigator believes can and will comply with the requirements of the protocol
Able to provide informed written consent.
Exclusion Criteria:
Biopsy-proven Crohn's disease or indeterminate colitis
Acute abdomen or other clinical emergencies requiring emergent management (for example: stricture, bowel obstruction, perforation and/or abscess)
Primary sclerosing cholangitis (PSC)
Pregnancy
Concurrent Clostridium difficile infection or other known infection
Prior history of fecal microbiota transplantation
Other causes of diarrhea, including but not limited to tube feeds and medications (for example, kayaxelate, metformin, lactulose, laxatives, magnesium)
Major congenital defects
Subjects with recent malignancy in the last 5 years, excluding non-melanoma skin malignancies
Anaphylactic reactions to any foods
Any antibiotic use within the last 3 months
Subject having any other condition that, in the opinion of the investigator, would jeopardize the safety or rights of the participant participating in the study, would make it unlikely for the participant to complete the study, or would confound the study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Carl V Crawford, MD
Organizational Affiliation
Weill Medical College of Cornell University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Weill Cornell Medical College
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
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Fecal Microbiota Transplantation (FMT) in the Management of Ulcerative Colitis (UC)
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