Phase 2 N=15 Randomized Quadruple-blind Treatment

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

Inflammatory Bowel Diseases · Ulcerative Colitis · Crohn Disease

Bottom Line

View on ClinicalTrials.gov: NCT02330653 ↗

Enrolled (actual)

Serious AEs

17.7%

Results posted

Oct 2023

Primary outcome: Primary: 1. Safety and Tolerability of Universal Donor FMT Compared to Placebo: FMT-related Adverse Events Grade 2 or Above — 4; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Fecal Microbiota Transplant (FMT) (Biological); Placebo (Biological)
Age: Pediatric, Adult · 5+ yrs
Sex: All
Sponsor: Stacy A. Kahn
Primary completion: Apr 2019

Outcome Measures

Outcome	Result	p-value
PRIMARY 1. Safety and Tolerability of Universal Donor FMT Compared to Placebo: FMT-related Adverse Events Grade 2 or Above	4; 0	—
SECONDARY Remission of Disease	1; 0; 2; 0	—
SECONDARY Improvement in Inflammatory Biomarkers	1; 3; 1; 1; 0; 2	—
SECONDARY Percentage of Donor Microbiome Present in Transplant Recipient	74.8; 41.8; 68.3; 47.9	—
SECONDARY Number of Participants With Improvement in Disease Activity	2; 0; 1; 0; 3; 5	—

Summary

The primary aims of this phase I/II, randomized, placebo controlled study are the assessment of safety and tolerability of universal donor FMT compared to placebo in pediatric and young adult subjects (ages 5 years through 30 years) with active ulcerative colitis (UC) or active Crohn's colitis (CD) who have failed, are intolerant to, or have refused traditional first-line maintenance therapy. Secondary objectives include the identification biomarkers in both donor and recipient that may confer a clinical response and to establish whether or not ongoing FMT maintenance therapy is required for maintenance of clinical benefit in pediatric UC or pediatric CD.

Eligibility 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.

Two initial subsets will be created: an initial subset of 20 subjects limited to patients greater than or equal to 12 years of age with mild to moderate ulcerative colitis (i.e., PUCAI 9) or CD (PDCAI >10) and have failed, are intolerant to, or have refused first-line maintenance therapy.

Have had visual or histologic evidence of inflammation confirmed through colonoscopy no more than 105 days prior to randomization.
Have negative test results for Hepatitis B (HBV), Hepatitis C (HCV), and Human Immunodeficiency Virus (HIV).
Have a negative urine hCG test if female of childbearing potential.
Able to swallow antibiotic, FMT or placebo capsules.
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)
Willing and able to participate in the study requirements, including serial stool collection, survey completion and clinic visits.
Willing to undergo telephone follow-up to assess for safety and adverse events.
Must be free of any known food allergy.
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.

Patients with extensive and/or severe CD (i.e. fistulizing disease, abscess, small bowel obstruction, fevers).
Patients in a clinical remission (PUCAI < 9) or (PCDAI <10).
Patients with recent (within 4 weeks) dose change of biologics, 5-ASA, steroids or immunomodulators
Patients considered to have toxic megacolon.
Patients with a known drug allergy to vancomycin, metronidazole or polymyxin.
Patients with a history of aspiration, gastroparesis, surgery involving the upper gastrointestinal tract (that might affect upper gastrointestinal motility) or unable to swallow pills.
Patients with esophageal dysmotility or swallowing dysfunction.
Patients with known food allergies.
Patients with positive test results for HBV, HCV, or HIV.
Female patients with a positive test result on a urine hCG test.
Patients unwilling or unable to give consent or participate in all study requirements.
Patients unable or unwilling to receive a retention enema for purposes of induction therapy.
Patients with recent (within 6 weeks) systemic antibiotic use.
Patients who have testing consistent with active clostridium difficile.
Patients with known prior experience with donor FMT.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02330653). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.