Fecal Microbiota Transplant (FMT) in Pediatric Active Ulcerative Colitis and Pediatric Active Crohn's Colitis
Condition: Inflammatory Bowel Diseases; Ulcerative Colitis; Crohn Disease
MeSH terms: Crohn Disease; Colitis; Colitis, Ulcerative; Intestinal Diseases; Inflammatory Bowel Diseases; Ulcer
Study type: Interventional
Phase: Phase 1/Phase 2

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.
    

Detailed description: 
      This is a single-center pilot, phase I/II, randomized, prospective, double-blinded,
      placebo-controlled study of FMT in the treatment of active pediatric UC and active pediatric
      CD. The primary aim is to assess safety and feasibility of a weekly FMT maintenance therapy.
      A total of 10 patients with active UC (as defined by PUCAI score of >9) and 10 patients with
      active CD (as defined by PDCAI score of >10) will be enrolled and randomized to receive FMT
      or placebo-FMT (study treatment) by retention enema for 1 week and oral, frozen encapsulated
      inocula/placebo for 7 weeks. After the first 8 weeks, subjects on FMT who improve or subjects
      on placebo-FMT who do not improve will have the option to continue on study treatment or
      switch to open-label FMT until the end of 4 months from study initiation. Subjects will be
      followed by telephone to assess adverse events for a total of 6 months after their last FMT
      dose.

      An initial subset of no more than 20 subjects will be enrolled in the study (will be limited
      to only those patients 12 years of age or older and to those who have mild to moderate
      disease) and randomized to receive FMT or placebo. We'd expect short term adverse events to
      occur within 7 days of FMT administration. Individual subject safety data will be reviewed by
      the PI to assess whether FMT appears to be safe in the subject before continuing the subject
      towards open-label use of FMT.

      Patient metadata and stool samples will be collected at key time points. The patient-reported
      metadata collection technique will allow for numerous clinical correlations to be parsed out
      using the random forest machine learning capabilities of synthetic learning in microbial
      ecology (SLiME) to identify taxonomic features associated with important clinical parameters.
    

Intervention name(s): Fecal Microbiota Transplant (FMT); Placebo
Intervention type(s): Biological; Biological
Intervention description(s): The study intervention consists of frozen, bottled or encapsulated fecal microbiota preparations that have been screened and prepared to a uniform and rigorous standard by OpenBiome. FMT is performed by patients receiving a retention enema and swallowing capsules, introducing stool from a healthy donor into their intestinal tract.; Placebo administration will consist of both a placebo retention enema and placebo capsules.
Other intervention name(s): Screened, healthy human donor stool
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Colitis; Fecal Microbiota Transplant; Ulcerative colitis; inflammatory bowel disease; Crohn's Disease