Pediatric FEcal Microbiota Transplant for Ulcerative Colitis

NCT02487238 | Completed Phase 1 DMC

• 3 other identifiers: 15-364NIF-15375HAH-17-002
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 3

Brief Summary

The PediFETCh study is a pilot trial designed to assess the feasibility of fecal microbiota transplants for the therapy of pediatric ulcerative colitis (UC) and pediatric inflammatory bowel disease-unclassified (IBD-U). Investigators will test the hypothesis that a protocol of twice-weekly retention enemas delivered over six weeks, using fecal transplant material from a healthy donor, will improve clinical and biological disease markers in patients with pediatric UC or IBD-U.

Conditions

Inflammatory Bowel Disease; Ulcerative Colitis

Keywords

Microbiome; Microbiota; Fecal Microbial Transplant; Fecal Microbiota Transplant; FMT; Gastroenterology; IBD; Inflammatory Bowel Disease; Ulcerative Colitis; UC; Inflammatory Bowel Disease Unclassified; IBD-U; Rebiotix; RBX2660; Pediatrics; McMaster Children's Hospital; Centre Hospitalier Universitaire Sainte-Justine; Children's Hospital at London Health Sciences Centre

Outcome Measures

Primary Outcomes (1)

• Feasibility (Composite Measure)

  Evaluation of: participant recruitment/retention/eligibility/acceptance/adverse events

  30 weeks

Secondary Outcomes (6)

• Microbiome Change (Composite Measure)

  Weeks: 0/3/6/12/18/24/30

• Clinical Remission

  Weeks: 0/1/2/3/4/5/6/12/18/24/30

• Clinical Improvement

  Weeks: 0/1/2/3/4/5/6/12/18/24/30

• Biological Improvement

  Weeks: 0/3/6/18/30

• Mucosal Healing

  Weeks: 0/3/6/12/18/24/30

Study Arms (2)

Fecal Microbiota Enema

EXPERIMENTAL

Live, healthy, human donor stool prepared as fecal enemas. Fecal enemas are prepared and collected by Rebiotix(®) (RBX2660), using extensively screened donor stool. Enemas will be administered on site at one of the participating trial sites by trained study investigators. Enemas are given: 2x per week for 6 weeks (total = 12 enemas over 6 weeks). Patients will be masked to enema contents.

Biological: Fecal Microbiota Enema

Normal Saline Enema

PLACEBO COMPARATOR

Normal saline enemas will be administered on site at one of the participating trial sites by trained study investigators. Enemas are given: 2x per week for 6 weeks (total = 12 enemas over 6 weeks). Patients will be masked to enema contents.

Biological: Normal Saline Enema

Interventions

Fecal Microbiota Enema BIOLOGICAL

Active intervention.

Also known as: FMT, RBX2660, Rebiotix, Fecal microbial enema

Fecal Microbiota Enema

Normal Saline Enema BIOLOGICAL

Placebo comparator.

Normal Saline Enema

Eligibility Criteria

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

You may qualify if:

• yo to 17yo at start of trial
• Followed at a participating clinical trial site: a) McMaster Children's Hospital, b) Children's Hospital at London Health Sciences Centre, c) Centre Hospitalier Universitaire Sainte-Justine
• Ulcerative colitis (UC) or Inflammatory bowel disease unclassified (IBD-U)
• Evidence of active clinical, biological, or mucosal disease
• Ongoing treatment is acceptable provided the patient has had no significant changes to medications, or medication dose for at least 4 weeks prior to starting the trial

You may not qualify if:

• Active participation in another therapeutic trial
• Unable to give informed consent, or assent
• Active Clostridium difficile infection
• Significant change in medication type, or medication dose within the preceding 4 weeks prior to starting the trial
• Starting new treatments, or having a significant change in medication dosing during the trial
• Significant, consecutive rise in PUCAI score during the trial
• Hospitalization during trial

Sponsors & Collaborators

• McMaster Children's Hospital: lead
• London Health Sciences Centre: collaborator
• St. Justine's Hospital: collaborator

Study Sites (3)

McMaster Children's Hospital

Hamilton, Ontario, L8N 3Z5, Canada

Location

Children's Hospital London Health Science Centre

London, Ontario, N6A 5W9, Canada

Location

CHU Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Related Publications (16)

• Rossen NG, Fuentes S, van der Spek MJ, Tijssen JG, Hartman JH, Duflou A, Lowenberg M, van den Brink GR, Mathus-Vliegen EM, de Vos WM, Zoetendal EG, D'Haens GR, Ponsioen CY. Findings From a Randomized Controlled Trial of Fecal Transplantation for Patients With Ulcerative Colitis. Gastroenterology. 2015 Jul;149(1):110-118.e4. doi: 10.1053/j.gastro.2015.03.045. Epub 2015 Mar 30.

  PMID: 25836986 BACKGROUND

• Moayyedi P, Surette MG, Kim PT, Libertucci J, Wolfe M, Onischi C, Armstrong D, Marshall JK, Kassam Z, Reinisch W, Lee CH. Fecal Microbiota Transplantation Induces Remission in Patients With Active Ulcerative Colitis in a Randomized Controlled Trial. Gastroenterology. 2015 Jul;149(1):102-109.e6. doi: 10.1053/j.gastro.2015.04.001. Epub 2015 Apr 7.

  PMID: 25857665 BACKGROUND

• Michail S, Durbin M, Turner D, Griffiths AM, Mack DR, Hyams J, Leleiko N, Kenche H, Stolfi A, Wine E. Alterations in the gut microbiome of children with severe ulcerative colitis. Inflamm Bowel Dis. 2012 Oct;18(10):1799-808. doi: 10.1002/ibd.22860. Epub 2011 Dec 14.

  PMID: 22170749 BACKGROUND

• Kunde S, Pham A, Bonczyk S, Crumb T, Duba M, Conrad H Jr, Cloney D, Kugathasan S. Safety, tolerability, and clinical response after fecal transplantation in children and young adults with ulcerative colitis. J Pediatr Gastroenterol Nutr. 2013 Jun;56(6):597-601. doi: 10.1097/MPG.0b013e318292fa0d.

  PMID: 23542823 BACKGROUND

• Kellermayer R, Nagy-Szakal D, Harris RA, Luna RA, Pitashny M, Schady D, Mir SA, Lopez ME, Gilger MA, Belmont J, Hollister EB, Versalovic J. Serial fecal microbiota transplantation alters mucosal gene expression in pediatric ulcerative colitis. Am J Gastroenterol. 2015 Apr;110(4):604-6. doi: 10.1038/ajg.2015.19. No abstract available.

  PMID: 25853207 BACKGROUND

• Suskind DL, Brittnacher MJ, Wahbeh G, Shaffer ML, Hayden HS, Qin X, Singh N, Damman CJ, Hager KR, Nielson H, Miller SI. Fecal microbial transplant effect on clinical outcomes and fecal microbiome in active Crohn's disease. Inflamm Bowel Dis. 2015 Mar;21(3):556-63. doi: 10.1097/MIB.0000000000000307.

  PMID: 25647155 BACKGROUND

• Vandenplas Y, Veereman G, van der Werff Ten Bosch J, Goossens A, Pierard D, Samsom JN, Escher JC. Fecal Microbial Transplantation in Early-Onset Colitis: Caution Advised. J Pediatr Gastroenterol Nutr. 2015 Sep;61(3):e12-4. doi: 10.1097/MPG.0000000000000281. No abstract available.

  PMID: 24399213 BACKGROUND

• Shimizu H, Arai K, Abe J, Nakabayashi K, Yoshioka T, Hosoi K, Kuroda M. Repeated fecal microbiota transplantation in a child with ulcerative colitis. Pediatr Int. 2016 Aug;58(8):781-5. doi: 10.1111/ped.12967. Epub 2016 Jun 21.

  PMID: 27324973 BACKGROUND

• Kumagai H, Yokoyama K, Imagawa T, Inoue S, Tulyeu J, Tanaka M, Yamagata T. Failure of Fecal Microbiota Transplantation in a Three-Year-Old Child with Severe Refractory Ulcerative Colitis. Pediatr Gastroenterol Hepatol Nutr. 2016 Sep;19(3):214-220. doi: 10.5223/pghn.2016.19.3.214. Epub 2016 Sep 29.

  PMID: 27738605 BACKGROUND

• Narula N, Kassam Z, Yuan Y, Colombel JF, Ponsioen C, Reinisch W, Moayyedi P. Systematic Review and Meta-analysis: Fecal Microbiota Transplantation for Treatment of Active Ulcerative Colitis. Inflamm Bowel Dis. 2017 Oct;23(10):1702-1709. doi: 10.1097/MIB.0000000000001228.

  PMID: 28906291 BACKGROUND

• Suskind DL, Singh N, Nielson H, Wahbeh G. Fecal microbial transplant via nasogastric tube for active pediatric ulcerative colitis. J Pediatr Gastroenterol Nutr. 2015 Jan;60(1):27-9. doi: 10.1097/MPG.0000000000000544.

  PMID: 25162366 BACKGROUND

• Paramsothy S, Kamm MA, Kaakoush NO, Walsh AJ, van den Bogaerde J, Samuel D, Leong RWL, Connor S, Ng W, Paramsothy R, Xuan W, Lin E, Mitchell HM, Borody TJ. Multidonor intensive faecal microbiota transplantation for active ulcerative colitis: a randomised placebo-controlled trial. Lancet. 2017 Mar 25;389(10075):1218-1228. doi: 10.1016/S0140-6736(17)30182-4. Epub 2017 Feb 15.

  PMID: 28214091 BACKGROUND

• Wang AY, Popov J, Pai N. Fecal microbial transplant for the treatment of pediatric inflammatory bowel disease. World J Gastroenterol. 2016 Dec 21;22(47):10304-10315. doi: 10.3748/wjg.v22.i47.10304.

  PMID: 28058011 BACKGROUND

• Cammarota G, Ianiro G, Tilg H, Rajilic-Stojanovic M, Kump P, Satokari R, Sokol H, Arkkila P, Pintus C, Hart A, Segal J, Aloi M, Masucci L, Molinaro A, Scaldaferri F, Gasbarrini G, Lopez-Sanroman A, Link A, de Groot P, de Vos WM, Hogenauer C, Malfertheiner P, Mattila E, Milosavljevic T, Nieuwdorp M, Sanguinetti M, Simren M, Gasbarrini A; European FMT Working Group. European consensus conference on faecal microbiota transplantation in clinical practice. Gut. 2017 Apr;66(4):569-580. doi: 10.1136/gutjnl-2016-313017. Epub 2017 Jan 13.

  PMID: 28087657 BACKGROUND

• Costello SP, Waters O, Bryant RV, et al. Short Duration, Low Intensity, Pooled Fecal Microbiota Transplantation Induces Remission in Patients with Mild-Moderately Active Ulcerative Colitis: A Randomised Controlled Trial. Gastroenterology 152(5): S198-S199, 2017.

  BACKGROUND

• Pai N, Popov J. Protocol for a randomised, placebo-controlled pilot study for assessing feasibility and efficacy of faecal microbiota transplantation in a paediatric ulcerative colitis population: PediFETCh trial. BMJ Open. 2017 Aug 21;7(8):e016698. doi: 10.1136/bmjopen-2017-016698.

  PMID: 28827258 RESULT

Related Links

• Protocol for a randomised, placebo-controlled pilot study for assessing feasibility and efficacy of faecal microbiota transplantation in a paediatric ulcerative colitis population: PediFETCh trial

MeSH Terms

Conditions

Inflammatory Bowel Diseases; Colitis, Ulcerative

Condition Hierarchy (Ancestors)

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

Study Officials

• Nikhil Pai, MD FRCPC

  McMaster Children's Hospital

  PRINCIPAL INVESTIGATOR

• Jelena Popov, BSc

  McMaster Children's Hospital Division of Pediatric Gastroenterology & Nutrition

  STUDY DIRECTOR

• Tim Ramsay, PhD

  The Ottawa Hospital Research Institute

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Masking Details: Patients will not be aware of which type of enema they have been randomized to receive (fecal microbial enema, or normal saline enema). Patients who were randomized to the normal saline enema will still be eligible to receive the fecal microbial enema when they finish the trial; these patients will know what they are receiving and no longer need masking.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Model Details: Patients will be randomized to receive either the fecal microbial enema containing healthy donor stool OR the normal saline enema placebo. Patients who receive the normal saline enema placebo may be eligible to still receive the fecal microbial enema when they finish the trial.

Study Record Dates

• First Submitted: June 25, 2015
• First Posted: July 1, 2015
• Study Start: November 1, 2015
• Primary Completion: December 1, 2018
• Study Completion: December 1, 2018
• Last Updated: January 30, 2019

Record last verified: 2019-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Open-access.
• Access Criteria: Pai N, Popov J. Protocol for a randomised, placebo-controlled pilot study for assessing feasibility and efficacy of faecal microbiota transplantation in a paediatric ulcerative colitis population: PediFETCh trial. BMJ Open. 2017;7(8):e016698.

Locations

CA (3)