FMT in Pediatric Crohn's Disease
Condition: Crohn Disease; Pediatric Crohns Disease
MeSH terms: Crohn Disease
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The goal of this study is to evaluate the safety of Fecal Microbiota Transplantation (FMT) in
      children with Crohn's disease who are in remission. Safety will be the primary endpoint and
      Pediatric Crohn's Disease. Pediatric Crohn's Disease Activity Index (PCDAI) with other
      secondary endpoints including changes in gut microbial diversity will also be studied. All
      children will receive the equivalent of 50g of stools from a healthy donor into the jejunum
      through upper endoscopy. Also, 1-2 additional mucosal biopsies will be collected during
      patient's routine (standard of care) endoscopy. Subjects will have a total of 5 study visits
      within 24 weeks including phone call follow up on Day 7 after FMT.
    

Detailed description: 
      The Investigators hypothesize that children with Crohn's disease who are in remission can
      receive a single endoscopic dose of FMT with no significant safety concerns.

      All children will receive the equivalent of 50 g of stools from a healthy donor into the
      jejunum through upper endoscopy. Children will be seen at baseline, then 4 and 24 weeks after
      study drug administration begins. Stools will be collected and stored for gut microbial
      profiles during the study visit windows. In addition, a follow up telephone call will be
      performed 7 days after study drug is administered. If children undergo endoscopy as part of
      the standard of care, study staff will obtain 1-2 additional biopsies for evaluation of
      mucosal inflammation. This study will also capture any laboratory results if any of the
      subjects undergo laboratory testing as part of the standard of care. This study will define
      the effects of transplant on gut microbial profile using advanced molecular taxonomic
      approaches. Safety will be closely monitored by solicited (during defined telephone calls and
      study visits) and unsolicited adverse events (at all times). Safety will be the primary
      endpoint of this study. Secondary endpoints include Pediatric Crohn's Disease Activity Index
      (PCDAI), changes in gut microbial diversity - determined by gut microbial genomics and
      proteomics (16S ribosomal RNA, 16s rRNA), and outcome measures for mucosal inflammation and
      repair as reflected through laboratory testing such as the level for C-reactive protein
      (CRP), erythrocyte sedimentation rate (ESR), inflammatory cytokines of the colonic mucosa as
      well as the stool calprotectin level.
    

Intervention name(s): FMT
Intervention type(s): Biological
Intervention description(s): Fecal Microbiota Transplantation, single dose, 50g of stool, delivered via standard of care upper endoscopy into jejunum.
Other intervention name(s): Fecal Microbiota Transplantation
Intervention model: Single Group Assignment
Intervention model description: Primary outcome is safety in children with Crohn's disease in remission.
Masking: None (Open Label)
Primary purpose: Other
Keywords: Crohn's Disease; PCDAI; 16s rRNA; Fecal Microbiota Transplantation; Gut Microbiome; FMT