PediCRaFT: Pediatric Crohn's Disease Fecal Transplant Trial

NCT03378167 | Completed Phase 1 DMC

• 1 other identifier: 0000
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 2

Brief Summary

The objective of this study is to assess the feasibility of a novel colonic and oral fecal microbiota transplantation protocol for the treatment of active pediatric Crohn's disease (CD). Specifically, we will test the hypothesis that a protocol of combination fecal microbiota colonoscopic infusion and oral microbiota capsules (OMC), using live fecal material from anonymous unrelated donors, can improve the disease activity of pediatric CD patients.

Conditions

Crohn Disease; Pediatric Crohns Disease; Inflammatory Bowel Diseases; Colitis

Keywords

Paediatrics; Pediatrics; Crohn's; Crohn; Microbiome; Microbiota; FMT; Fecal Transplant; Fecal Microbiota Transplant; IBD-U; Inflammatory Bowel Disease Unclassified; Inflammatory Bowel Disease Indeterminate; IBD; Colitis

Outcome Measures

Primary Outcomes (4)

• Monthly Recruitment Rate

  Assessment of recruitment rate (based on patients meeting all eligibility criteria who were approached for trial entry)

  30 weeks

• Dropout Rate Post Enrolment

  Rate of patients leaving the trial (patient, or protocol directed exclusion) after enrolment

  30 weeks

• Rate of Patient Protocol Adherence

  Rate of patients providing all required blood, stool and urine samples per protocol

  30 weeks

• Rate of Adverse Events

  Rate of patients requiring hospitalization, or experiencing PCDAI increase ≥20 x 2 successive measures

  30 weeks

Secondary Outcomes (6)

• Clinical: Improvement in Disease Symptoms

  Baseline, Week 6, Week 30

• Clinical: Remission in Disease Symptoms

  Week 6, Week 30

• Clinical: Improvement in Serum Inflammatory Markers

  Baseline, Week 6, Week 30

• Clinical: Improvement in Mucosal Inflammatory Markers

  Baseline, Week 6, Week 30

• Clinical: Change in Urine Metabolomics

  Baseline, Week 6, Week 30

Study Arms (2)

MICROBIOTA

EXPERIMENTAL

Patients randomized to the INTERVENTION arm will receive a baseline fecal microbiota transplant (FMT) colonoscopic infusion at Week 0, followed by twice-weekly oral microbiota capsule (OMC) therapy for 6 weeks (including Week 0). (n = 30)

Biological: MICROBIOTA

PLACEBO

PLACEBO COMPARATOR

Patients randomized to the CONTROL arm will receive a baseline normal saline (NS) colonoscopic infusion at Week 0, followed by twice-weekly dextrose-containing oral placebo capsule (OPC) therapy for 6 weeks (including Week 0). (n = 15)

Biological: PLACEBO

Interventions

MICROBIOTA BIOLOGICAL

Fecal microbiota enema (RBX2660) infused via colonoscope x 1 + oral microbiota capsules (RBX7455) x 6 weeks. The fecal microbiota enema (RBX2660) prepared by Rebiotix has received Health Canada Clinical Trials Application (CTA), and U.S. Food and Drug Administration Investigational New Drug Application (IND) approval for clinical trials in patients with recurrent Clostridium difficile infection, and pediatric inflammatory bowel disease. The human-derived fecal oral microbiota capsule (RBX7455) has received U.S. Food and Drug Administration Investigational New Drug Application (IND) approval for clinical trials in patients with recurrent Clostridium difficile infection.

Also known as: RBX2660, RBX7455, Fecal microbiota transplant

MICROBIOTA

PLACEBO BIOLOGICAL

Placebo enema (Normal Saline) infused via colonoscope x 1 + oral placebo capsules (dextrose-containing capsules) x 6 weeks. NOTE: Patients randomized to the control group will be given the option of receiving open-label treatment, with the intervention therapy, either: upon completion of the trial, or if they are removed from the trial due to disease exacerbation or other adverse event, at the discretion of their primary gastroenterologist.

PLACEBO

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Pediatric patients
• ≥3yo
• Crohn's disease, or IBD-Unclassified favoring Crohn's disease (as deemed by the patient's primary pediatric gastroenterologist)
• Active symptoms

You may not qualify if:

• Currently enrolled in another clinical trial
• Unable to give informed consent or assent
• Severe comorbid medical illness (at discretion of patient's primary pediatric gastroenterologist)
• Concomitant Clostridium difficile infection
• Severe Crohn's disease flare requiring hospitalization
• Commenced new, or temporary medical therapies (ie. corticosteroids, antibiotics, prebiotics) within 4 weeks prior to commencing the trial; NB: Weaning doses of corticosteroid will be permitted (≤ 0.25mg/kg/day)

Sponsors & Collaborators

• McMaster Children's Hospital: lead
• St. Justine's Hospital: collaborator

Study Sites (2)

McMaster Children's Hospital

Hamilton, Ontario, M8V1A4, Canada

Location

Centre Hospitalier Universitaire Sainte-Justine, University of Montreal

Montreal, Quebec, H3T 1C5, Canada

Location

Related Publications (24)

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  PMID: 31784432 DERIVED

MeSH Terms

Conditions

Crohn Disease; Inflammatory Bowel Diseases; Colitis

Interventions

Microbiota; Fecal Microbiota Transplantation

Condition Hierarchy (Ancestors)

Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases; Colonic Diseases

Intervention Hierarchy (Ancestors)

Microbiological Phenomena; Biota; Biodiversity; Ecosystem; Environment; Ecological and Environmental Phenomena; Biological Phenomena; Environment and Public Health; Biological Therapy; Therapeutics

Study Officials

• Nikhil Pai, BSc, CNSC, MD, FRCPC

  McMaster Children's Hospital, Division of Pediatric Gastroenterology & Nutrition

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
• Masking Details: COLONOSCOPY: 1. Microbial enemas are drawn up in a 500cc syringe that is concealed with an opaque bag, used to infuse contents through the colonoscope. This will insure contents are blinded to non-study personnel in the room 2. Patients randomized to the control study arm will have identical concealment of the 500cc syringe used to infuse contents through the colonoscope. For the NS infusion, study personnel will add an additional 0.75 ml of commercially available (Club House® brand), food-grade food coloring (2 drops red, 3 drops green, 7 drops yellow) to confer a brown color to the clear normal saline solution. This step will maintain blinding for the patient and non-study personnel in the room, as the contents of the liquid will be visible endoscopically, but will still retain a similarly-colored brown appearance akin to human stool. ORAL CAPSULAR THERAPY: a), b) The opaque color of both the OMC and OPC will insure that blinding is preserved to the study patient.
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor, Division of Pediatric Gastroenterology & Nutrition

Model Details: Patients will be randomized 2:1 to one of two groups upon consent: intervention or control. 30 patients will be randomized to receive an FMT via colonoscopy + oral microbiota capsular (OMC) therapy (INTERVENTION), and 15 patients will be randomized to receive normal saline (NS) via colonoscopy + dextrose-containing oral capsules (oral placebo capsule, OPC) (CONTROL). Randomization will occur through a computer-generated block-randomization pattern (block size = 6 participants). Patients randomized to the control group will be given the option of receiving open-label treatment, with the intervention therapy, either: upon completion of the trial, or if they are removed from the trial due to disease exacerbation or other adverse event, at the discretion of their primary gastroenterologist.

Study Record Dates

• First Submitted: December 12, 2017
• First Posted: December 19, 2017
• Study Start: December 1, 2018
• Primary Completion: September 30, 2023
• Study Completion: September 30, 2023
• Last Updated: October 19, 2023

Record last verified: 2023-10

Locations

CA (2)