Optimal Fecal Microbiota Transplant Dosing for Mild to Moderate Ulcerative Colitis

• Register: ClinicalTrials.gov
• Last refreshed on: 10 May 2021
• Main ID: NCT03006809
• Date of registration: 21/12/2016
• Prospective Registration: Yes
• Primary sponsor: Najwa Elnachef
• Public title: Optimal Fecal Microbiota Transplant Dosing for Mild to Moderate Ulcerative Colitis
• Scientific title: Optimal Fecal Microbiota Transplant Dosing for Mild to Moderate Ulcerative Colitis
• Date of first enrolment: March 2, 2017
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03006809
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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.

Exclusion Criteria:

- 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)

- 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



Age minimum: 18 Years
Age maximum: 64 Years
Gender: All

Health Condition(s) or Problem(s) studied

Ulcerative Colitis

Intervention(s)

Other: pretreatment antibiotics

Biological: Fecal Microbiota Transplantation (FMT), OpenBiome

Primary Outcome(s)

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]

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]

Secondary Outcome(s)

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]

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]

Changes in Mood/Depression Score (based on PHQ-9 survey and score) [Time Frame: 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]

Progression of disease defined by clinical flare (Time to next flare) [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 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 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]

Secondary ID(s)

16-20066

Source(s) of Monetary Support

Please refer to primary and secondary sponsors