Ulcerative ColitisPhase 1NCT03006809
Optimal Fecal Microbiota Transplant Dosing for Mild to Moderate Ulcerative Colitis
A Phase 1 study testing Fecal Microbiota Transplantation (FMT), OpenBiome and pretreatment antibiotics in people with Ulcerative Colitis, run by Najwa Elnachef at 1 site in United States.
Trial summary
This is a prospective unblinded, randomized trial for the use of Fecal Microbiota Transplantation (FMT) for the treatment of Ulcerative Colitis (UC), in combination with or without antibiotic pretreatment. Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Ulcerative Colitis
- What is being tested?
- Fecal Microbiota Transplantation (FMT), OpenBiome and pretreatment antibiotics
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Mar 2017to 4 Apr 2021
- Phase
- 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 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patients with history of mild to moderate Ulcerative Colitis confirmed by endoscopy and pathology.
- Total Mayo score 4-9, endoscopic subscore ≥1; patients who have not had endoscopic evaluation within one year of enrollment will have flexible sigmoidoscopy for evaluation.
- Age 18 - 64 and deemed otherwise healthy at the discretion of the investigator.
- Concurrent therapies with mesalamine (stable x 4 weeks), immunomodulators (stable x 3 months), and biologic agents (stable x 3 months) will be allowed to continue during study.
- If patient is on prednisone, the dose must be ≤ 10mg/day at the time of treatment and will be weaned by 2.5mg/week during the study period.
Who cannot join
- Severe or refractory UC defined as Mayo score ≥10, endoscopic disease activity score 3
- Untreated enteric infection (positive stool test for any of the following: Clostridium difficile, Salmonella, Shigella, Yersinia, Campylobacter, enteropathogenic E. coli or other enteric infection at the discretion of the investigator.
- History of colectomy
- Disease limited to distal proctitis
- Patients taking probiotics within 6 weeks of planned FMT therapy.
- Severe immunodeficiency, inherited or acquired (e.g. HIV, chemotherapy or radiation therapy)
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- Patients with the following laboratory abnormalities: absolute neutrophil count (ANC) < 1000 / µl, platelets <50 x 10^9 /L,, hemoglobin <6.5 g/dL..
- History of anaphylaxis (severe allergic reaction) to food allergens (e.g. tree nuts, shellfish)
- Dysphagia (oropharyngeal, esophageal, functional, neuromuscular)
- History of recurrent aspiration episodes
- Documented severe gastroparesis
- Active intestinal obstruction
- Patients with renal insufficiency (GFR < 50ml/min)
- Allergy to the following generally regarded as safe ingredients (GRAS): glycerol, acid resistant HPMC, gellan gum, cocoa butter, titanium dioxide
- Adverse event attributable to any previous FMT
- Allergy/intolerance to proton pump inhibitor therapy
- Allergy/intolerance to vancomycin, metronidazole, or neomycin.
- Non-steroidal inflammatory medications (NSAIDs) as long-term treatment, defined as use for at least 4 days a week each month.
- Cholestyramine use
- Any condition in which the investigator thinks the FMT treatment may pose a health risk (e.g. severely immunocompromised)
- Simultaneous participation in another interventional clinical trial
- Patients who are pregnant, breast feeding or planning pregnancy during study trial period.
- During the trial period until one week after the trial end: Non-use of appropriate contraceptives in females of childbearing potential (e.g. condoms, intrauterine device (IUD), hormonal contraception, or other means considered adequate by the responsible investigator) or in males with a child-fathering potential (condoms, or other means considered adequate by the responsible investigator during treatment) or well-founded doubt about the patient's cooperation
- Patients with any other significant medical condition that could confound or interfere with evaluation of safety, tolerability or prevent compliance with the study protocol at the discretion of the investigator
- Life expectancy <6 months
Trial locations and contacts
1 site in 1 country.
United States1 site
UCSF Division of Gastroenterology at Mount Zion
San Francisco, California, 94143
About this study
This is a prospective unblinded, randomized trial for the use of Fecal Microbiota Transplantation (FMT) for the treatment of Ulcerative Colitis (UC), in combination with or without antibiotic pretreatment.
Read the detailed description
This is a prospective open-label, randomized trial for the use of Fecal Microbiota Transplantation (FMT) for the treatment of Ulcerative Colitis (UC), in combination with or without antibiotic pretreatment.
This trial involves 11 study visits at UCSF in San Francisco, CA.
The routes of administration will be via colonoscopy for all subjects with maintenance therapy administered orally (i.e. using encapsulated FMT) for half of the subjects and per rectum by enema in the other half of the subjects. Additionally, the utility of pretreatment antibiotics will be assessed.
- Conditions
- Ulcerative Colitis
- Being tested
- Fecal Microbiota Transplantation (FMT), OpenBiome, pretreatment antibiotics
- Keywords
- Ulcerative colitis, FMT, fecal transplant
- Medical subject headings
- Colitis, Ulcerative
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fecal Microbiota Transplantation (FMT), OpenBiomeBiological
Delivered by colonoscopy, enema or orally (as capsules)
pretreatment antibioticsOther
Also called vancomycin, metronidazole, neomycin
5 day course (vancomycin PO 500mg bid, metronidazole PO 500mg bid, and neomycin PO 500mg bid) starting 6 days prior FMT
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | pretreatment antibiotics + FMT delivered by colonoscopy + FMT capsules per week x 6 weeks Receives: Biological: Fecal Microbiota Transplantation (FMT), OpenBiome; Other: pretreatment antibiotics |
| 2 | Experimental | no antibiotics, FMT delivered by colonoscopy + FMT capsules per week x 6 weeks Receives: Biological: Fecal Microbiota Transplantation (FMT), OpenBiome |
| 3 | Experimental | pretreatment antibiotics + FMT delivered by colonoscopy + FMT delivered by enema once per week x 6 weeks Receives: Biological: Fecal Microbiota Transplantation (FMT), OpenBiome; Other: pretreatment antibiotics |
| 4 | Experimental | no antibiotics, FMT delivered by colonoscopy + FMT delivered by enema once per week x 6 weeks Receives: Biological: Fecal Microbiota Transplantation (FMT), OpenBiome |
What is being measured
The primary outcome is the main question the study is designed to answer.
The occurrence of a Serious Adverse event (SAE), solicited and unsolicited AE, new gastrointestinal medical condition and diagnoses from FMT treatment or new infection from FMT treatment
safety endpoint
Measured: up to 1 year
Steroid-free Clinical Remission at week 9 + endoscopic remission or response defined as total Mayo score ≤ 2 with all four sub-scores ≤ 1 and a ≥ 1 point reduction in endoscopy sub-score
Measured: 8 weeks post initial treatment
Secondary outcomes (9)
Changes in microbiome with FMT therapy. Changes in the microbiome: assessed by frequent stool sampling for 16S rRNA analysis prior to each FMT therapy and after the last capsule/enema dose
Measured: up to 1 year
Clinical Response: decrease in Mayo score by ≥ 3 points, decrease in bleeding subscore by ≥ 1, or absolute subscore of 0-1
Measured: 8 weeks post initial treatment
Progression of disease defined by initiation of anti-TNF agents or Corticosteroids: Initiation of anti-TNF agents (such as infliximab, adalimumab, certolizumab, vedolizumab and steroids). Includes time gap until additional agents are started.
Measured: 2, 4 and 8 weeks post initial FMT
Progression of disease defined by increase in dosages of current UC medications
Measured: 2, 4 and 8 weeks post initial FMT
Progression of disease defined by time to colectomy
Measured: up to one year post initial FMT
Time to death secondary to UC
Measured: up to one year post initial FMT
Progression of disease defined by clinical flare (Time to next flare)
Measured: 2, 4 and 8 weeks post initial FMT
Increase in Quality of Life (based on RAND SF-36 survey and score)
Measured: 8 weeks post initial FMT
Changes in Mood/Depression Score (based on PHQ-9 survey and score)
Measured: 8 weeks post initial FMT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2016 (estimated)
- Study start2 Mar 2017
- Primary completion4 May 2020
- Study completion4 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Najwa Elnachef
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Najwa Elnachef, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03006809
- Sponsor's protocol ID
- 16-20066
- Status last verified
- Apr 2021
- First submitted
- 21 Dec 2016
- Last updated
- 28 Apr 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Smith BJ, Piceno Y, Zydek M, Zhang B, Syriani LA, Terdiman JP, Kassam Z, Ma A, Lynch SV, Pollard KS, El-Nachef N. Strain-resolved analysis in a randomized trial of antibiotic pretreatment and maintenance dose delivery mode with fecal microbiota transplant for ulcerative colitis. Sci Rep. 2022 Apr 1;12(1):5517. doi: 10.1038/s41598-022-09307-5.
PubMed 35365713
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Optimal Fecal Microbiota Transplant Dosing for Mild to Moderate Ulcerative Colitis
- ClinicalTrials.gov ID
- NCT03006809
- Acronym / sponsor ID
- 16-20066
- Conditions
- Ulcerative Colitis
- Keywords
- Ulcerative colitis, FMT, fecal transplant
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Najwa ElnachefUniversity, hospital or other
- Study start
- 2 Mar 2017 (actual)
- Primary completion
- 4 May 2020 (actual)
- Study completion
- 4 Apr 2021 (actual)
- First posted
- 30 Dec 2016 (estimated)
- 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.
This is a prospective unblinded, randomized trial for the use of Fecal Microbiota Transplantation (FMT) for the treatment of Ulcerative Colitis (UC), in combination with or without antibiotic pretreatment.
Detailed description
This is a prospective open-label, randomized trial for the use of Fecal Microbiota Transplantation (FMT) for the treatment of Ulcerative Colitis (UC), in combination with or without antibiotic pretreatment.
This trial involves 11 study visits at UCSF in San Francisco, CA.
The routes of administration will be via colonoscopy for all subjects with maintenance therapy administered orally (i.e. using encapsulated FMT) for half of the subjects and per rectum by enema in the other half of the subjects. Additionally, the utility of pretreatment antibiotics will be assessed.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Biological: Fecal Microbiota Transplantation (FMT), OpenBiome, Other: pretreatment antibioticspretreatment antibiotics + FMT delivered by colonoscopy + FMT capsules per week x 6 weeks |
| 2 | Experimental | Biological: Fecal Microbiota Transplantation (FMT), OpenBiomeno antibiotics, FMT delivered by colonoscopy + FMT capsules per week x 6 weeks |
| 3 | Experimental | Biological: Fecal Microbiota Transplantation (FMT), OpenBiome, Other: pretreatment antibioticspretreatment antibiotics + FMT delivered by colonoscopy + FMT delivered by enema once per week x 6 weeks |
| 4 | Experimental | Biological: Fecal Microbiota Transplantation (FMT), OpenBiomeno antibiotics, FMT delivered by colonoscopy + FMT delivered by enema once per week x 6 weeks |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Patients with history of mild to moderate Ulcerative Colitis confirmed by endoscopy and pathology.
- Total Mayo score 4-9, endoscopic subscore ≥1; patients who have not had endoscopic evaluation within one year of enrollment will have flexible sigmoidoscopy for evaluation.
- Age 18 - 64 and deemed otherwise healthy at the discretion of the investigator.
- Concurrent therapies with mesalamine (stable x 4 weeks), immunomodulators (stable x 3 months), and biologic agents (stable x 3 months) will be allowed to continue during study.
- If patient is on prednisone, the dose must be ≤ 10mg/day at the time of treatment and will be weaned by 2.5mg/week during the study period.
Key exclusion criteria (25 listed)
- Severe or refractory UC defined as Mayo score ≥10, endoscopic disease activity score 3
- Untreated enteric infection (positive stool test for any of the following: Clostridium difficile, Salmonella, Shigella, Yersinia, Campylobacter, enteropathogenic E. coli or other enteric infection at the discretion of the investigator.
- History of colectomy
- Disease limited to distal proctitis
- Patients taking probiotics within 6 weeks of planned FMT therapy.
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 |
|---|---|---|
| Fecal Microbiota Transplantation (FMT), OpenBiomeDelivered by colonoscopy, enema or orally (as capsules) | Biological | 1, 2, 3, 4 |
| pretreatment antibioticsAlso: vancomycin, metronidazole, neomycin5 day course (vancomycin PO 500mg bid, metronidazole PO 500mg bid, and neomycin PO 500mg bid) starting 6 days prior FMT | Other | 1, 3 |
Primary outcome
The occurrence of a Serious Adverse event (SAE), solicited and unsolicited AE, new gastrointestinal medical condition and diagnoses from FMT treatment or new infection from FMT treatment
Time frame: up to 1 year
safety endpoint
Steroid-free Clinical Remission at week 9 + endoscopic remission or response defined as total Mayo score ≤ 2 with all four sub-scores ≤ 1 and a ≥ 1 point reduction in endoscopy sub-score
Time frame: 8 weeks post initial treatment
9 secondary outcomes
Changes in microbiome with FMT therapy. Changes in the microbiome: assessed by frequent stool sampling for 16S rRNA analysis prior to each FMT therapy and after the last capsule/enema dose
Time frame: up to 1 year
Clinical Response: decrease in Mayo score by ≥ 3 points, decrease in bleeding subscore by ≥ 1, or absolute subscore of 0-1
Time frame: 8 weeks post initial treatment
Progression of disease defined by initiation of anti-TNF agents or Corticosteroids: Initiation of anti-TNF agents (such as infliximab, adalimumab, certolizumab, vedolizumab and steroids). Includes time gap until additional agents are started.
Time frame: 2, 4 and 8 weeks post initial FMT
Progression of disease defined by increase in dosages of current UC medications
Time frame: 2, 4 and 8 weeks post initial FMT
Progression of disease defined by time to colectomy
Time frame: up to one year post initial FMT
Time to death secondary to UC
Time frame: up to one year post initial FMT
Progression of disease defined by clinical flare (Time to next flare)
Time frame: 2, 4 and 8 weeks post initial FMT
Increase in Quality of Life (based on RAND SF-36 survey and score)
Time frame: 8 weeks post initial FMT
Changes in Mood/Depression Score (based on PHQ-9 survey and score)
Time frame: 8 weeks post initial FMT
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2021.
- Derived from this study: PubMed 35365713Smith BJ, Piceno Y, Zydek M, Zhang B, Syriani LA, Terdiman JP, Kassam Z, Ma A, Lynch SV, Pollard KS, El-Nachef N. Strain-resolved analysis in a randomized trial of antibiotic pretreatment and maintenance dose delivery mode with fecal microbiota transplant for ulcerative colitis. Sci Rep. 2022 Apr 1;12(1):5517. doi: 10.1038/s41598-022-09307-5.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Fecal Microbiota Transplantation (FMT), OpenBiome and pretreatment antibiotics in people with Ulcerative Colitis, run by Najwa Elnachef 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?
The sponsor lists 5 inclusion and 25 exclusion criteria. 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: finished, as of the sponsor's last update on 28 Apr 2021. 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. 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?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The occurrence of a Serious Adverse event (SAE), solicited and unsolicited AE, new gastrointestinal medical condition and diagnoses from FMT treatment or new infection from FMT treatment (time frame: up to 1 year). 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?
On the ClinicalTrials.gov record for NCT03006809, which also lists 1 linked publication.
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 28 Apr 2021.