FMT for Remission of Active Ulcerative Colitis in Adults

Study Overview
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Brief Summary
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The goal of this study is to establish the safety and effectiveness of lyophilized (LYO) fecal microbiota transplant (FMT) for treating ulcerative colitis (UC) in adults. The protocol is being re-designed to address relevant, current research questions in the context of FMT treatment for UC. Once a final protocol is approved, this webpage will be updated.

Official Title
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A Randomized Double-blind, Placebo-controlled Trial of Lyophilized Fecal Microbiota Transplantation for the Induction of Remission in Adults With Active Ulcerative Colitis

Conditions
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Ulcerative Colitis, Inflammatory Bowel Diseases

Intervention / Treatment
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* Biological: FMT oral
* Biological: FMT enema
* Other: Placebo oral
* Other: Placebo enema


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Able to provide informed consent. Willing and able to comply with all the required trial procedures Active ulcerative colitis as defined by Mayo score > 3 AND Mayo endoscopic sub-score > 1 (within 30 days before enrollment, or at baseline) Exclusion Criteria: Planned or actively taking another investigational product Abdominal surgery within the past 60 days Patients with neutropenia with absolute neutrophil count <0.5 x 109/L at - Evidence of toxic megacolon or gastrointestinal perforation on imaging Peripheral white blood cell count > 35.0 x 109/L at enrollment AND temperature > 38.0oC Active infectious diarrhea at the time of enrolment Increase in medical therapy for UC within 3 months of enrollment. Continued treatment with stable dose of 5-ASA, azathioprine, 6-mercaptopurine, cyclosporine, prednisone and/or anti- TNF agents for at least 3 months of is allowed Severe UC requiring hospitalization at the time of enrolment Pregnant or lactating History of anaphylaxis to any food Requiring oral and/or intravenous systemic antibiotic therapy at the time of study enrolment Unwilling to discontinue probiotic (yogurt is allowed) Severe underlying disease such that the patient is not expected to survive for at least 30 days. Any condition that in the opinion of the investigator that would pose harm to the participant or the research staff for the potential participant to take part in the trial

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo oral & enema<br>twice weekly x 8 weeks: 10 placebo oral capsules + placebo enema | Other: Placebo oral<br>* placebo given orally (10 capsules) twice weekly for total of 8 weeks<br>Other: Placebo enema<br>* placebo given via enema (1) twice weekly for total of 8 weeks<br>|
| Active Comparator: LYO-FMT oral + placebo enema<br>twice weekly x 8 weeks: 10 LYO-FMT oral capsules + 1 placebo enema | Biological: FMT oral<br>* lyophilized FMT given orally (10 capsules) twice weekly for total of 8 weeks<br>Other: Placebo enema<br>* placebo given via enema (1) twice weekly for total of 8 weeks<br>|
| Active Comparator: LYO-FMT oral + LYO-FMT enema<br>twice weekly x 8 weeks: 10 LYO-FMT oral capsules + 1 LYO-FMT enema | Biological: FMT oral<br>* lyophilized FMT given orally (10 capsules) twice weekly for total of 8 weeks<br>Biological: FMT enema<br>* lyophilized FMT given via enema (1) twice weekly for total of 8 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Remission of UC | achievement of remission of UC as defined by Mayo score ≤ 2 AND Mayo endoscopic score of ≤ 1 | 9 weeks following receipt of LYO-FMT | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence/absence of adverse events upon treatment with LYO-FMT [safety and tolerability] | Safety of LYO-FMT in patients with active UC as determined by absence of adverse events | up to 5 years post-FMT | 
| UC disease progression | Determine progression of UC based on development of any of the following: Clinical flare of UC requiring hospitalization up to 3 months post-FMT Increase in dosages of current UC specific medications up to 3 months' post FMT Time to colectomy for UC flare up to 12 months' post FMT Time to death directly attributable to UC up to 5 years post FMT Improvement in clinical response defined by decrease in Partial Mayo score by ≥ 3 points from pre to post LYO-FMT. Improvement in patient-reported health related QoL using Valuation of Lost Productivity and (VOLP) and RAND VR12 measured at pre and at 5 weeks, 12 and 24 weeks following LYO-FMT and annually for 5 years Reduction in biologic inflammatory markers (serum c-reactive protein (CRP) and fecal calprotectin) from pre to post LYO-FMT | immediately after FMT (study) treatment period up to 5 years post-FMT | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ulcerative colitis, Fecal Microbiota transplant, FMT, Inflammatory bowel disease, IBD