Examining the Efficacy of Fecal Microbiota Transplantation (FMT) and Dietary Fiber in Patients With Ulcerative Colitis

NCT ID: NCT03998488

Last Updated: 2025-01-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 27 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-01-31
• Study Completion Date: 2024-04-12

Brief Summary

A double-blind, randomized, placebo-controlled clinical trial examining the efficacy and safety of Fecal Microbiota Transplantation (FMT) and high fiber supplementation in patients with active mild to moderate Ulcerative Colitis (UC). All enrolled subjects will provide serological, stool and mucosal specimen at each clinic visit to help further define the alterations in microbial profiles and immune cell function in response to psyllium fiber after FMT treatment.

Detailed Description

This is a randomized, double-blind, placebo-controlled clinical trial with the following treatment assignments:

1. Investigational FMT (one-time)

1. Subject will blindly receive investigational FMT once at day 0 colonoscopy

2. Subjects in this group will blindly receive placebo FMP250 at week 8 by flexible sigmoidoscopy.

2. Investigational FMT (one-time) + Psyllium (2x/day for 8 weeks)

1. Subject will blindly receive investigational FMT once at day 0 colonoscopy

2. Subjects in this group will blindly receive placebo FMP250 at week 8 by flexible sigmoidoscopy.

3. Placebo FMT (one-time) +/- Psyllium (2x/day for 8 weeks)

1. Subject will blindly receive placebo FMT once at day 0 colonoscopy

2. Subjects in this group will blindly receive investigational FMP250 at week 8 by flexible sigmoidoscopy.

Subjects will blindly receive the investigational or placebo FMP250 treatment if they meet all inclusion and exclusion criteria during the day 0 screening colonoscopy. Subjects will receive follow-up phone calls at day 1, week 2, week 6, and week 10 post-FMT and will return for clinic visits at week 4, week 8 and week 12 post-FMT. Stool and blood samples will be collected for research purposes from subjects at every clinic visit (Day 0 colonoscopy, Week 4, Week 8, and Week 12). Mucosal biopsies will also be taken during the initial colonoscopy, at day 0 and during the follow-up flexible sigmoidoscopy at week 8. At week 8 post-FMT, all subjects will be evaluated by flexible sigmoidoscopy. Subjects originally randomized into the placebo cohort at week 0 will receive investigational FMP250 by flexible sigmoidoscopy at week 8, and subjects originally randomized into the investigational cohort at week 0 will receive placebo FMP250 by flexible sigmoidoscopy at week 8. Lastly, all subjects will be contacted for follow-up phone calls every subsequent 6 months for the next year.

Conditions

Ulcerative Colitis; Inflammatory Bowel Diseases

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Investigational FMT

Participants will be blindly randomized to receive a single dose of investigational FMT during the week 0 colonoscopy.

Additionally, participants will blindly receive a single dose of placebo FMT during the week 8 by flexible sigmoidoscopy.

Group Type: EXPERIMENTAL

Fecal Microbiota Transplantation

Intervention Type: DRUG

The proposed intervention will deliver 250 milliliters of FMT by colonoscopy to the investigational FMT treatment groups at week 0. The placebo treatment group will instead receive the placebo FMT by colonoscopy at week 0 and then the investigational FMT by flexible sigmoidoscopy at week 8.

Investigational FMT is biologically active human fecal material that is pre-screened, tested, quarantined, stored, packaged, and labeled by OpenBiome. Placebo FMP250 is a control unit made of glycerol, saline, and food dye that is stored, packaged, and labeled identically to the investigational FMT, to ensure blinding during delivery.

Investigational FMT + psyllium fiber

Participants will be blindly randomized to receive a single dose of investigational FMT during the week 0 colonoscopy. They will also receive fiber supplementation of 1 teaspoon 2x/day for 8 weeks.

Additionally, participants will blindly receive a single dose of placebo FMT during the week 8 by flexible sigmoidoscopy.

Group Type: ACTIVE_COMPARATOR

Fecal Microbiota Transplantation

Intervention Type: DRUG

The proposed intervention will deliver 250 milliliters of FMT by colonoscopy to the investigational FMT treatment groups at week 0. The placebo treatment group will instead receive the placebo FMT by colonoscopy at week 0 and then the investigational FMT by flexible sigmoidoscopy at week 8.

Investigational FMT is biologically active human fecal material that is pre-screened, tested, quarantined, stored, packaged, and labeled by OpenBiome. Placebo FMP250 is a control unit made of glycerol, saline, and food dye that is stored, packaged, and labeled identically to the investigational FMT, to ensure blinding during delivery.

Psyllium Husk Powder

Intervention Type: DIETARY_SUPPLEMENT

All subjects assigned to the fiber treatment arms will be required to take 1 teaspoon (approximately 5 grams) of psyllium husk powder twice a day (morning and night) for 8 weeks, beginning 3 days prior to Week 0 screening colonoscopy.

Psyllium husk powder is the dried and powdered form of a psyllium seed coat.

Placebo FMT +/- psyllium fiber

Participants will be blindly randomized to receive a single dose of placebo FMT during the week 0 colonoscopy. They may or may not also receive fiber supplementation of 1 teaspoon 2x/day for 8 weeks.

Additionally, participants will blindly receive a single dose of investigational FMT during the week 8 by flexible sigmoidoscopy.

Group Type: PLACEBO_COMPARATOR

Psyllium Husk Powder

Intervention Type: DIETARY_SUPPLEMENT

All subjects assigned to the fiber treatment arms will be required to take 1 teaspoon (approximately 5 grams) of psyllium husk powder twice a day (morning and night) for 8 weeks, beginning 3 days prior to Week 0 screening colonoscopy.

Psyllium husk powder is the dried and powdered form of a psyllium seed coat.

Interventions

Fecal Microbiota Transplantation

The proposed intervention will deliver 250 milliliters of FMT by colonoscopy to the investigational FMT treatment groups at week 0. The placebo treatment group will instead receive the placebo FMT by colonoscopy at week 0 and then the investigational FMT by flexible sigmoidoscopy at week 8.

Investigational FMT is biologically active human fecal material that is pre-screened, tested, quarantined, stored, packaged, and labeled by OpenBiome. Placebo FMP250 is a control unit made of glycerol, saline, and food dye that is stored, packaged, and labeled identically to the investigational FMT, to ensure blinding during delivery.

Intervention Type: DRUG

Psyllium Husk Powder

All subjects assigned to the fiber treatment arms will be required to take 1 teaspoon (approximately 5 grams) of psyllium husk powder twice a day (morning and night) for 8 weeks, beginning 3 days prior to Week 0 screening colonoscopy.

Psyllium husk powder is the dried and powdered form of a psyllium seed coat.

Intervention Type: DIETARY_SUPPLEMENT

Other Intervention Names

FMP250; FMT

Eligibility Criteria

Inclusion Criteria

• Male or Female ≥ 18 years of age.
• Documentation of prior history of mild to moderate UC.
• Endoscopy confirmed active UC ≥ 15 centimeters at week 0 screening colonoscopy.

a. As defined by a total Mayo scoring of 4-10 with an endoscopic sub-score ≥ 1.

• Patients must have a descending intact colon.
• Patients taking steroid or biologic therapy must be on a stable dose for 4 weeks prior to screening and maintained throughout the trial.
• Eligible patients willing to undergo screening testing prior to FMT to document baseline status:

1. Urine Testing

2. Blood Testing

3. Stool Testing

• Patients must discontinue anti-rCDI antibiotics (e.g. vancomycin, fidaxomicin) 48 hours prior to FMT delivery procedure.

Exclusion Criteria

• Biopsy proven Crohn's disease
• UC patients with severe disease (defined as a total mayo score >10)
• Clinical complications requiring emergent management (e.g. stricture, bowel obstruction, perforation and/or abscess)
• Concurrent C. difficile or other infections
• Primary sclerosing cholangitis
• Prior history of FMT
• Treatment for malignancy within past 5 years
• Active or latent tuberculosis
• Clinically meaningful laboratory abnormalities

1. Hb: < 8

2. ALT: greater than 3x the ULN (upper limit of normal)

• History of anaphylactic reactions to food allergens or allergy to psyllium husk
• Subject having any other condition that, in the opinion of the investigator, would jeopardize the safety or rights of the subject participating in the study, would make it unlikely for the subject to complete the study, or would confound the study.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 89 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Crohn's and Colitis Foundation

OTHER

Sponsor Role: collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIH

Sponsor Role: collaborator

Weill Medical College of Cornell University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Randy Longman, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Weill Medical College of Cornell University

Locations

Weill Cornell Medicine

New York, New York, United States

Countries

United States

References

Jacob V, Crawford C, Cohen-Mekelburg S, Viladomiu M, Putzel GG, Schneider Y, Chabouni F, O'Neil S, Bosworth B, Woo V, Ajami NJ, Petrosino JF, Gerardin Y, Kassam Z, Smith M, Iliev ID, Sonnenberg GF, Artis D, Scherl E, Longman RS. Single Delivery of High-Diversity Fecal Microbiota Preparation by Colonoscopy Is Safe and Effective in Increasing Microbial Diversity in Active Ulcerative Colitis. Inflamm Bowel Dis. 2017 Jun;23(6):903-911. doi: 10.1097/MIB.0000000000001132.

Reference Type: BACKGROUND

PMID: 28445246 (View on PubMed)

Gogokhia L, Tran N, Grier A, Nagayama M, Xiang G, Funez-dePagnier G, Lavergne A, Ericsson C, Ben Maamar S, Zhang M, Battat R, Scherl E, Lukin DJ, Longman RS. Donor composition and fiber promote strain engraftment in a randomized controlled trial of fecal microbiota transplant for ulcerative colitis. Med. 2025 Sep 12;6(9):100707. doi: 10.1016/j.medj.2025.100707. Epub 2025 Jun 2.

Reference Type: DERIVED

PMID: 40460824 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

5R01DK128257

Identifier Type: NIH

Identifier Source: secondary_id

View Link

19-04020045

Identifier Type: -

Identifier Source: org_study_id