Ulcerative ColitisEarly Phase 1REFOCUSNCT04968951
Refined Fecal Microbiota Transplantation (FMT) for Ulcerative Colitis (UC)
A Early Phase 1 study testing Metronidazole, Vancomycin and Fecal Microbiota Transplantation in people with Ulcerative Colitis and FMT, run by Ari M Grinspan at 1 site in United States.
Trial summary
The researchers intend to prospectively study the safety, clinical efficacy and microbial outcomes in patients with recently diagnosed UC with FMT capsule therapy derived from pre-defined donors. Donors will be specifically screened for Fusobacterium and Sutterella species as well as for global diversity. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Ulcerative Colitis, FMT, Fecal Microbiota Transplant
- What is being tested?
- Metronidazole, Vancomycin and Fecal Microbiota Transplantation
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Nov 2021to 22 Aug 2022
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
- Adults aged 18 to 75 with UC, with initial diagnosis of UC >3 months prior to time of study enrollment visit
- Partial Mayo score of 4-10 with endoscopic subscore ≥2 on flexible sigmoidoscopy
- Permissible UC medications include oral or rectal administered mesalamines
- Corticosteroids must be discontinued at least 4 weeks before enrollment
- Documented negative C. difficile and GI PCR for enteric pathogens (BioFire) testing before commencement of fecal microbiota transplantation
- Previous documentation of ulcerative colitis based on colonoscopy/flexible sigmoidoscopy with compatible histology
Trial locations and contacts
1 site in 1 country.
United States1 site
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
About this study
Official title: REFOCUS: Refined Fecal Microbiota Transplantation (FMT) Delivered by Oral Capsules for Induction of Remission in Mild to Moderate Ulcerative Colitis - a Phase I Study
The researchers intend to prospectively study the safety, clinical efficacy and microbial outcomes in patients with recently diagnosed UC with FMT capsule therapy derived from pre-defined donors. Donors will be specifically screened for Fusobacterium and Sutterella species as well as for global diversity. It is unknown if treatment with antibiotics before FMT improves the engraftment and/or efficacy of FMT in UC, therefore the researchers plan to randomize subjects to receive pre-treatment with antibiotics or not before FMT therapy. The research team enroll patients from The Susan and Leonard Feinstein IBD Center and our established early diagnosis clinic at Mount Sinai Hospital (MSH).
Read the detailed description
This is an open-label two arm pilot study to measure the safety, microbiological and clinical impacts of FMT in patients with ulcerative colitis. The researchers will prospectively enroll 16 UC patients (up to 20 subjects accounting for subjects dropping out) with moderate-severe disease from one tertiary care referral center. The overall objective of the study is to collect robust clinical data and create a tissue repository including blood, stool and biopsies to understand the safety, efficacy and microbial changes FMT has on UC patients. The central hypothesis is that pre-defined oral capsule administered FMT is safe and effective for the treatment of UC.
Objectives: To determine the tolerability, feasibility, and safety of using fecal microbiota transplantation orally as an induction agent for patients with ulcerative colitis. To determine whether fecal microbiota transplantation (FMT) delivered via oral capsules can induce clinical remission in patients with mild to moderate ulcerative colitis. Assess whether pretreatment with antibiotics improves engraftment and efficacy of FMT in UC. To characterize the impact of orally administered FMT on the microbiota of patients with ulcerative colitis, particularly those changes associated with response or lack of response.
Study Outcomes: Clinical remission at Week 8, defined as: Steroid-free clinical remission (Total Mayo less 2) and Endoscopic remission (Mayo endoscopic subscore 0 or 1)
- Conditions
- Ulcerative Colitis, FMT, Fecal Microbiota Transplant
- Being tested
- Metronidazole, Placebo, Vancomycin, Fecal Microbiota Transplantation
- Keywords
- FMT, Fecal Microbiota Transplantation, Fecal Microbiota Transplant, Fecal Matter Transplant, Ulcerative colitis, UC
- Medical subject headings
- Colitis, Ulcerative
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetronidazoleDrug
Also called Flagyl
Antibiotic treatment - 250 mg every 6 hrs for 5 days
PlaceboDrug
Placebo treatment
VancomycinDrug
Antibiotic treatment - 125 mg every 6 hrs for 5 days
Fecal Microbiota TransplantationBiological
Also called FMT
Encapsulated biologically active human fecal material (donor stool) is provided in capsule form. The fecal material is homogenized with sterile saline, then pelleted and re-suspended in sterile saline/40% glycerol. Participants will receive 15 FMT capsules per day for 3 consecutive days. Capsule are to be swallowed under direct supervision.
| Study group | Role | What they receive |
|---|---|---|
| FMT with Antibiotics | Active comparator | Participants will receive antibiotics before receiving Fecal Microbiota Transplantation Receives: Drug: Metronidazole; Drug: Vancomycin; Biological: Fecal Microbiota Transplantation |
| FMT with placebo | Placebo comparator | Participants will receive placebo before receiving Fecal Microbiota Transplantation Receives: Drug: Placebo; Biological: Fecal Microbiota Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants with an adverse
Safety as measured by proportion of participants with an adverse event through week 8
Measured: 8 Weeks
Proportion of participants with a severe adverse
Safety as measured by proportion of participants with a severe adverse event through week 8
Measured: 8 Weeks
Secondary outcomes (9)
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.
Measured: 8 weeks
Proportion of patients that achieve Mayo score 0 or 1
Proportion of patients with initial Mayo 2/3 disease at flexible sigmoidoscopy that achieve endoscopic improvement (Mayo score 0 or 1). The Mayo Score is a composite of subscores (each rated 0-3) from four categories, including stool frequency, rectal bleeding, findings of flexible proctosigmoidoscopy or colonoscopy, and physician's global assessment, with a total score ranging from 0-12, with higher score indicating worse health outcomes.
Measured: 8 weeks
Number of patients with endoscopic remission
Number of patients with endoscopic remission as defined by a Mayo score of 0
Measured: 8 weeks
Change in Nancy score
Change in histological score assessed by the Nancy score. The Nancy score includes 3 histologic items that define 5 grades of activity: absence of significant histologic disease, chronic inflammatory infiltrate with no acute inflammatory infiltrate, mildly active disease, moderately active disease, and severely active disease. The Nancy index is defined by a 5-level classification ranging from grade 0 (absence of significant histological disease activity) to grade 4 (severely active disease). higher score indicating worse health outcomes.
Measured: 8 weeks
Fecal calprotectin level
Mean change fecal calprotectin levels
Measured: baseline and 8 weeks
C-reactive protein levels
Mean change C-reactive protein levels
Measured: baseline and 8 weeks
Hemoglobin levels
Mean change hemoglobin levels
Measured: baseline and 8 weeks
Albumin levels
Mean change albumin levels
Measured: baseline and 8 weeks
ESR level
Mean change erythrocyte sedimentation rate (ESR)
Measured: baseline and 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2021
- Study start10 Nov 2021
- Primary completion22 Aug 2022
- Study completion22 Aug 2022
Why it stopped: insufficient funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ulcerative ColitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Participants are assigned to one of two groups. One group will be assigned to receive pre-treatment with antibiotics before Fecal Microbiota Transportation. The other group will be assigned to receive pre-treatment without antibiotics (with placebo) before Fecal Microbiota Transplantation.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ari M Grinspan
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Ari M Grinspan, Icahn School of Medicine at Mount Sinai
- Investigators
- Ari Grinspan, MD, Principal investigator, Icahn School of Medicine at Mount Sinai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04968951
- Sponsor's protocol ID
- GCO 18-1464
- Acronym
- REFOCUS
- Status last verified
- Feb 2024
- First submitted
- 9 Jul 2021
- Last updated
- 26 Feb 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: There is not a plan to make IPD available
Study at a glance
From the registry record, last updated by the sponsor on 26 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- REFOCUS: Refined Fecal Microbiota Transplantation (FMT) Delivered by Oral Capsules for Induction of Remission in Mild to Moderate Ulcerative Colitis - a Phase I Study
- ClinicalTrials.gov ID
- NCT04968951
- Acronym / sponsor ID
- REFOCUS / GCO 18-1464
- Conditions
- Ulcerative Colitis, FMT, Fecal Microbiota Transplant
- Keywords
- FMT, Fecal Microbiota Transplantation, Fecal Microbiota Transplant, Fecal Matter Transplant, Ulcerative colitis, UC
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: insufficient funding
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Ari M GrinspanUniversity, hospital or other
- Study start
- 10 Nov 2021 (actual)
- Primary completion
- 22 Aug 2022 (actual)
- Study completion
- 22 Aug 2022 (actual)
- First posted
- 20 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The researchers intend to prospectively study the safety, clinical efficacy and microbial outcomes in patients with recently diagnosed UC with FMT capsule therapy derived from pre-defined donors. Donors will be specifically screened for Fusobacterium and Sutterella species as well as for global diversity. It is unknown if treatment with antibiotics before FMT improves the engraftment and/or efficacy of FMT in UC, therefore the researchers plan to randomize subjects to receive pre-treatment with antibiotics or not before FMT therapy. The research team enroll patients from The Susan and Leonard Feinstein IBD Center and our established early diagnosis clinic at Mount Sinai Hospital (MSH).
Detailed description
This is an open-label two arm pilot study to measure the safety, microbiological and clinical impacts of FMT in patients with ulcerative colitis. The researchers will prospectively enroll 16 UC patients (up to 20 subjects accounting for subjects dropping out) with moderate-severe disease from one tertiary care referral center. The overall objective of the study is to collect robust clinical data and create a tissue repository including blood, stool and biopsies to understand the safety, efficacy and microbial changes FMT has on UC patients. The central hypothesis is that pre-defined oral capsule administered FMT is safe and effective for the treatment of UC.
Objectives: To determine the tolerability, feasibility, and safety of using fecal microbiota transplantation orally as an induction agent for patients with ulcerative colitis. To determine whether fecal microbiota transplantation (FMT) delivered via oral capsules can induce clinical remission in patients with mild to moderate ulcerative colitis. Assess whether pretreatment with antibiotics improves engraftment and efficacy of FMT in UC. To characterize the impact of orally administered FMT on the microbiota of patients with ulcerative colitis, particularly those changes associated with response or lack of response.
Study Outcomes: Clinical remission at Week 8, defined as: Steroid-free clinical remission (Total Mayo less 2) and Endoscopic remission (Mayo endoscopic subscore 0 or 1)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- Participants are assigned to one of two groups. One group will be assigned to receive pre-treatment with antibiotics before Fecal Microbiota Transportation. The other group will be assigned to receive pre-treatment without antibiotics (with placebo) before Fecal Microbiota Transplantation.
| Arm | Type | Receives |
|---|---|---|
| FMT with Antibiotics | Active comparator | Drug: Metronidazole, Drug: Vancomycin, Biological: Fecal Microbiota TransplantationParticipants will receive antibiotics before receiving Fecal Microbiota Transplantation |
| FMT with placebo | Placebo comparator | Drug: Placebo, Biological: Fecal Microbiota TransplantationParticipants will receive placebo before receiving Fecal Microbiota Transplantation |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MetronidazoleAlso: FlagylAntibiotic treatment - 250 mg every 6 hrs for 5 days | Drug | FMT with Antibiotics |
| PlaceboPlacebo treatment | Drug | FMT with placebo |
| VancomycinAntibiotic treatment - 125 mg every 6 hrs for 5 days | Drug | FMT with Antibiotics |
| Fecal Microbiota TransplantationAlso: FMTEncapsulated biologically active human fecal material (donor stool) is provided in capsule form. The fecal material is homogenized with sterile saline, then pelleted and re-suspended in sterile saline/40% glycerol. Participants will receive 15 FMT capsules per day for 3 consecutive days. Capsule are to be swallowed under direct supervision. | Biological | FMT with Antibiotics, FMT with placebo |
Primary outcome
Proportion of participants with an adverse
Time frame: 8 Weeks
Safety as measured by proportion of participants with an adverse event through week 8
Proportion of participants with a severe adverse
Time frame: 8 Weeks
Safety as measured by proportion of participants with a severe adverse event through week 8
9 secondary outcomes
Number of patients requiring escalation of medical therapies
Time frame: 8 weeks
Proportion of patients that achieve Mayo score 0 or 1
Time frame: 8 weeks
Number of patients with endoscopic remission
Time frame: 8 weeks
Change in Nancy score
Time frame: 8 weeks
Fecal calprotectin level
Time frame: baseline and 8 weeks
C-reactive protein levels
Time frame: baseline and 8 weeks
Hemoglobin levels
Time frame: baseline and 8 weeks
Albumin levels
Time frame: baseline and 8 weeks
ESR level
Time frame: baseline and 8 weeks
Results and safety
The study was stopped early (reason given: insufficient funding). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2024.
- Individual participant dataSharing planned: No. There is not a plan to make IPD available
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ari Grinspan, MD principal investigator
Icahn School of Medicine at Mount Sinai
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Metronidazole, Vancomycin and Fecal Microbiota Transplantation in people with Ulcerative Colitis and FMT, run by Ari M Grinspan at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: insufficient funding). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of participants with an adverse (time frame: 8 Weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Feb 2024.