Fecal Microbiota Transplant (FMT) in Pediatric Active Crohn's Colitis

• Register: ClinicalTrials.gov
• Last refreshed on: 21 January 2019
• Main ID: NCT02330211
• Date of registration: 29/12/2014
• Prospective Registration: Yes
• Primary sponsor: Stacy A. Kahn
• Public title: Fecal Microbiota Transplant (FMT) in Pediatric Active Crohn's Colitis
• Scientific title: A Phase I/II, Double Blinded, Placebo Controlled, Single-center Study of Fecal Microbiota Transplant (FMT) for the Treatment of Active Pediatric Crohn's Colitis
• Date of first enrolment: July 17, 2017
• Target sample size: 4
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02330211
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Stacy A Kahn, MD
• Affiliation: Boston Childrens Hospital - GI & Nutrition

Key inclusion & exclusion criteria

Inclusion Criteria:

Male and female children and young adults, aged 5 years to 30 years, who meet the following
inclusion criteria, will be enrolled in the study.

An initial subset of 10 subjects will be limited to patients with mild to moderate Crohn's
disease (i.e., PCDAI less than or equal to 30) and to individuals greater than or equal to
12 years of age. If FMT appears to be safe in this subset of patients after 8 weeks in the
study (to be assessed by a Data Safety Monitoring Board), expanded enrollment as is
described above will occur.

All patients must satisfy below criteria:

1. Have Crohn's colitis (PCDAI >10) and have failed, are intolerant to, or have refused
traditional first-line maintenance therapy.

2. Have had visual or histologic evidence of inflammation confirmed through colonoscopy
no more than 105 days prior to randomization.

3. Have negative test results for Hepatitis B (HBV), Hepatitis C (HCV), and Human
Immunodeficiency Virus (HIV).

4. Have a negative urine hCG test if female of childbearing potential.

5. Able to swallow antibiotic, FMT or placebo capsules.

6. Able to give informed consent and/or assent as appropriate (patients 12-17 will be
asked to provide written assent, patients 5-11 will be observed for assent or dissent
behaviorally, or with verbal/written communication)

7. Willing and able to participate in the study requirements, including serial stool
collection, survey completion and clinic visits.

8. Willing to undergo telephone follow-up to assess for safety and adverse events.

9. Must be free of any known food allergy.

10. Agrees and willing to have an enema for purposes of induction therapy.

Patients who have disease that has required other medications (including steroids,
immunosuppressives, and biologics) will be included.

Exclusion Criteria:

Subjects who fall into any of the following exclusion criteria at the time of screening are
not eligible for enrollment into the study.

1. Patients with extensive and/or severe CD (i.e. fistulizing disease, abscess, small
bowel obstruction, fevers).

2. Patients in a clinical remission (PCDAI <10).

3. Patients with recent (within 4 weeks) dose change of biologics, 5-ASA, steroids or
immunomodulators

4. Patients considered to have toxic megacolon.

5. Patients with a known drug allergy to vancomycin, metronidazole or polymixin.

6. Patients with a history of aspiration, gastroparesis, surgery involving the upper
gastrointestinal tract (that might affect upper gastrointestinal motility) or unable
to swallow pills.

7. Patients with esophageal dysmotility or swallowing dysfunction.

8. Patients with known food allergies.

9. Patients with positive test results for HBV, HCV, or HIV.

10. Female patients with a positive test result on a urine hCG test.

11. Patients unwilling or unable to give consent or participate in all study requirements.

12. Patients unable or unwilling to receive a retention enema for purposes of induction
therapy

13. Patients with recent (within 6 weeks) systemic antibiotic use

14. Patients who have testing consistent with active clostridium difficile.

15. Patients with known prior experience with donor FMT

Research personnel and care providers will be educated about inclusion/exclusion criteria
so that only appropriate patients are approached for informed consent. This will reduce the
burden of worry for families and patients as to whether they are suitable candidates for
the study. Subjects who are found to have no evidence of active disease by colonoscopy will
be excluded from the study.



Age minimum: 5 Years
Age maximum: 30 Years
Gender: All

Health Condition(s) or Problem(s) studied

Crohn Disease

Inflammatory Bowel Diseases

Intervention(s)

Biological: Fecal Microbiota Transplant

Biological: Placebo

Primary Outcome(s)

Safety and tolerability of universal donor FMT compared to placebo: FMT-related adverse events of grade 2 or above [Time Frame: At 8 weeks after start of FMT]

Improvement Pediatric Crohn's Disease Activity Index (PCDAI) by 12.5 points or more [Time Frame: At 8 weeks after start of FMT]

Secondary Outcome(s)

Safety and tolerability of universal donor FMT compared to placebo: FMT-related adverse events of grade 2 or above [Time Frame: At 1 year after start of FMT]

Remission of disease [Time Frame: At 8 weeks and 1 year after start of FMT]

Identify biomarkers that may confer a clinical response [Time Frame: At 8 weeks and 1 year after start of FMT]

Improvement Pediatric Crohn's Disease Activity Index (PCDAI) by 12.5 points or more [Time Frame: At 1 year after start of FMT]

Secondary ID(s)

P00014880

Source(s) of Monetary Support

Please refer to primary and secondary sponsors