Study Summary
This trial will test whether a combination of colonic and oral fecal microbiota transplantation can improve the disease activity of pediatric Crohn's disease patients.
Eligible Conditions
- Crohn's Disease
- Inflammatory Bowel Disease
- Pediatric Crohn's Disease
- Inflammatory Bowel Disease (IBD)
Treatment Effectiveness
Effectiveness Progress
Study Objectives
4 Primary · 6 Secondary · Reporting Duration: Week 6, Week 30
30 weeks
Dropout Rate Post Enrolment
Monthly Recruitment Rate
Rate of Adverse Events
Rate of Patient Protocol Adherence
Week 30
Clinical: Change in Fecal Microbiome
Clinical: Change in Urine Metabolomics
Clinical: Improvement in Disease Symptoms
Clinical: Improvement in Mucosal Inflammatory Markers
Clinical: Improvement in Serum Inflammatory Markers
Week 6, Week 30
Clinical: Remission in Disease Symptoms
Trial Safety
Safety Progress
Trial Design
2 Treatment Groups
MICROBIOTA
1 of 2
PLACEBO
1 of 2
Experimental Treatment
Non-Treatment Group
45 Total Participants · 2 Treatment Groups
Primary Treatment: MICROBIOTA · Has Placebo Group · Phase 1
MICROBIOTA
Biological
Experimental Group · 1 Intervention: MICROBIOTA · Intervention Types: BiologicalPLACEBO
Biological
PlaceboComparator Group · 1 Intervention: PLACEBO · Intervention Types: BiologicalTrial Logistics
Trial Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: week 6, week 30
Who is running the clinical trial?
McMaster Children's HospitalLead Sponsor
34 Previous Clinical Trials
16,036 Total Patients Enrolled
McMaster Children's Hospital (McMaster University)UNKNOWN
Centre Hospitalier Universitaire Sainte-Justine (CHU-SJ) (University of Montreal)UNKNOWN
Nikhil Pai, BSc, MDPrincipal InvestigatorMcMaster Children's Hospital, Division of Pediatric Gastroenterology & Nutrition
Nikhil Pai, BSc, CNSC, MD, FRCPCPrincipal InvestigatorMcMaster Children's Hospital, Division of Pediatric Gastroenterology & Nutrition
Eligibility Criteria
Age 3 - 17 · All Participants · 3 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:You currently have noticeable symptoms of your medical condition.
The clinical trial is not open to children.
You have Crohn's disease, or a type of inflammatory bowel disease that is suspected to be Crohn's disease by your pediatric gastroenterologist.
References
- Agrawal, Manasi, Olga C. Aroniadis, Lawrence J. Brandt, Colleen Kelly, Sarah Freeman, Christina Surawicz, Elizabeth Broussard, et al.. 2016. “The Long-term Efficacy and Safety of Fecal Microbiota Transplant for Recurrent, Severe, and Complicated Clostridium Difficile Infection in 146 Elderly Individuals”. Journal of Clinical Gastroenterology. Ovid Technologies (Wolters Kluwer Health). doi:10.1097/mcg.0000000000000410.
- Conte, M P, S Schippa, I Zamboni, M Penta, F Chiarini, L Seganti, J Osborn, P Falconieri, O Borrelli, and S Cucchiara. 2006. “Gut-associated Bacterial Microbiota in Paediatric Patients with Inflammatory Bowel Disease”. Gut. BMJ. doi:10.1136/gut.2005.078824.
- Vandenplas, Y., G. Veereman, J. van der Werff ten Bosch, A. Goossens, D. Pierard, J.N. Samsom, and J.C. Escher. 2015. “Fecal Microbial Transplantation in Early-onset Colitis”. Journal of Pediatric Gastroenterology & Nutrition. Ovid Technologies (Wolters Kluwer Health). doi:10.1097/mpg.0000000000000281.
- Turner, Dan, Anthony R. Otley, David Mack, Jeffrey Hyams, J. de Bruijne, Krista Uusoue, Thomas D. Walters, et al.. 2007. “Development, Validation, and Evaluation of a Pediatric Ulcerative Colitis Activity Index: A Prospective Multicenter Study”. Gastroenterology. Elsevier BV. doi:10.1053/j.gastro.2007.05.029.
- Sekirov, Inna, Shannon L. Russell, L. Caetano M. Antunes, and B. Brett Finlay. 2010. “Gut Microbiota in Health and Disease”. Physiological Reviews. American Physiological Society. doi:10.1152/physrev.00045.2009.
- Pai, Nikhil, and Jelena Popov. 2017. “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. BMJ. doi:10.1136/bmjopen-2017-016698.
- Kelly, Colleen R, Chioma Ihunnah, Monika Fischer, Alexander Khoruts, Christina Surawicz, Anita Afzali, Olga Aroniadis, et al.. 2014. “Fecal Microbiota Transplant for Treatment of Clostridium Difficile Infection in Immunocompromised Patients”. American Journal of Gastroenterology. Ovid Technologies (Wolters Kluwer Health). doi:10.1038/ajg.2014.133.
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- Tedelind, Sofia, Fredrik Westberg, Martin Kjerrulf, and Alexander Vidal. 2007. “Anti-inflammatory Properties of the Short-chain Fatty Acids Acetate and Propionate: A Study with Relevance to Inflammatory Bowel Disease”. World Journal of Gastroenterology. Baishideng Publishing Group Inc.. doi:10.3748/wjg.v13.i20.2826.
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- Kunde, Sachin, Angela Pham, Sarah Bonczyk, Teri Crumb, Meg Duba, Harold Conrad, Deborah Cloney, and Subra Kugathasan. 2013. “Safety, Tolerability, and Clinical Response After Fecal Transplantation in Children and Young Adults with Ulcerative Colitis”. Journal of Pediatric Gastroenterology & Nutrition. Ovid Technologies (Wolters Kluwer Health). doi:10.1097/mpg.0b013e318292fa0d.
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- Maslowski, Kendle M., Angelica T. Vieira, Aylwin Ng, Jan Kranich, Frederic Sierro, Di Yu, Heidi C. Schilter, et al.. 2009. “Regulation of Inflammatory Responses by Gut Microbiota and Chemoattractant Receptor GPR43”. Nature. Springer Science and Business Media LLC. doi:10.1038/nature08530.
- Kellermayer, Richard, Dorottya Nagy-Szakal, Alan R Harris, Ruth Ann Luna, Milena Pitashny, Deborah Schady, Sabina A V Mir, et al.. 2015. “Serial Fecal Microbiota Transplantation Alters Mucosal Gene Expression in Pediatric Ulcerative Colitis”. American Journal of Gastroenterology. Ovid Technologies (Wolters Kluwer Health). doi:10.1038/ajg.2015.19.
- Gevers, Dirk, Subra Kugathasan, Lee A. Denson, Yoshiki Vázquez-Baeza, Will Van Treuren, Boyu Ren, Emma Schwager, et al.. 2014. “The Treatment-naive Microbiome in New-onset Crohn’s Disease”. Cell Host & Microbe. Elsevier BV. doi:10.1016/j.chom.2014.02.005.
- Suskind, David L., Namita Singh, Heather Nielson, and Ghassan Wahbeh. 2015. “Fecal Microbial Transplant via Nasogastric Tube for Active Pediatric Ulcerative Colitis”. Journal of Pediatric Gastroenterology & Nutrition. Ovid Technologies (Wolters Kluwer Health). doi:10.1097/mpg.0000000000000544.
- Orenstein, Robert, Erik Dubberke, Robert Hardi, Arnab Ray, Kathleen Mullane, Darrell S. Pardi, and Mayur S. Ramesh. 2015. “Safety and Durability of RBX2660 (microbiota Suspension) for Recurrentclostridium Difficileinfection: Results of the PUNCH CD Study”. Clinical Infectious Diseases. Oxford University Press (OUP). doi:10.1093/cid/civ938.
- “American Society of Clinical Oncology Policy Statement: Oversight of Clinical Research”. 2003. “American Society of Clinical Oncology Policy Statement: Oversight of Clinical Research”. Journal of Clinical Oncology. American Society of Clinical Oncology (ASCO). doi:10.1200/jco.2003.04.026.
- Putignani, Lorenza, Federica Del Chierico, Andrea Petrucca, Pamela Vernocchi, and Bruno Dallapiccola. 2014. “The Human Gut Microbiota: A Dynamic Interplay with the Host from Birth to Senescence Settled During Childhood”. Pediatric Research. Springer Science and Business Media LLC. doi:10.1038/pr.2014.49.
- Suskind, David L., Mitchell J. Brittnacher, Ghassan Wahbeh, Michele L. Shaffer, Hillary S. Hayden, Xuan Qin, Namita Singh, et al.. 2015. “Fecal Microbial Transplant Effect on Clinical Outcomes and Fecal Microbiome in Active Crohnʼs Disease”. Inflammatory Bowel Diseases. Oxford University Press (OUP). doi:10.1097/mib.0000000000000307.
- Shimizu, Hirotaka, Katsuhiro Arai, Jun Abe, Kazuhiko Nakabayashi, Takako Yoshioka, Kenji Hosoi, and Makoto Kuroda. 2016. “Repeated Fecal Microbiota Transplantation in a Child with Ulcerative Colitis”. Pediatrics International. Wiley. doi:10.1111/ped.12967.
- Cammarota, Giovanni, Gianluca Ianiro, Herbert Tilg, Mirjana Rajilić-Stojanović, Patrizia Kump, Reetta Satokari, Harry Sokol, et al.. 2017. “European Consensus Conference on Faecal Microbiota Transplantation in Clinical Practice”. Gut. BMJ. doi:10.1136/gutjnl-2016-313017.
- Suskind, David L., Mitchell J. Brittnacher, Ghassan Wahbeh, Michele L. Shaffer, Hillary S. Hayden, Xuan Qin, Namita Singh, et al.. 2015. “Fecal Microbial Transplant Effect on Clinical Outcomes and Fecal Microbiome in Active Crohnʼs Disease”. Inflammatory Bowel Diseases. Oxford University Press (OUP). doi:10.1097/mib.0000000000000307.
- Pai, Nikhil, and Jelena Popov. 2017. “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. BMJ. doi:10.1136/bmjopen-2017-016698.
- Orenstein, Robert, Erik Dubberke, Robert Hardi, Arnab Ray, Kathleen Mullane, Darrell S. Pardi, and Mayur S. Ramesh. 2015. “Safety and Durability of RBX2660 (microbiota Suspension) for Recurrentclostridium Difficileinfection: Results of the PUNCH CD Study”. Clinical Infectious Diseases. Oxford University Press (OUP). doi:10.1093/cid/civ938.
- Kunde, Sachin, Angela Pham, Sarah Bonczyk, Teri Crumb, Meg Duba, Harold Conrad, Deborah Cloney, and Subra Kugathasan. 2013. “Safety, Tolerability, and Clinical Response After Fecal Transplantation in Children and Young Adults with Ulcerative Colitis”. Journal of Pediatric Gastroenterology & Nutrition. Ovid Technologies (Wolters Kluwer Health). doi:10.1097/mpg.0b013e318292fa0d.
- Conte MP, Schippa S, Zamboni I, Penta M, Chiarini F, Seganti L, Osborn J, Falconieri P, Borrelli O, Cucchiara S. Gut-associated bacterial microbiota in paediatric patients with inflammatory bowel disease. Gut. 2006 Dec;55(12):1760-7. doi: 10.1136/gut.2005.078824. Epub 2006 Apr 28.
- 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.
- Haberman Y, Tickle TL, Dexheimer PJ, Kim MO, Tang D, Karns R, Baldassano RN, Noe JD, Rosh J, Markowitz J, Heyman MB, Griffiths AM, Crandall WV, Mack DR, Baker SS, Huttenhower C, Keljo DJ, Hyams JS, Kugathasan S, Walters TD, Aronow B, Xavier RJ, Gevers D, Denson LA. Pediatric Crohn disease patients exhibit specific ileal transcriptome and microbiome signature. J Clin Invest. 2014 Aug;124(8):3617-33. doi: 10.1172/JCI75436. Epub 2014 Jul 8. Erratum In: J Clin Invest. 2015 Mar 2;125(3):1363.
- Lobaton T, Bessissow T, De Hertogh G, Lemmens B, Maedler C, Van Assche G, Vermeire S, Bisschops R, Rutgeerts P, Bitton A, Afif W, Marcus V, Ferrante M. The Modified Mayo Endoscopic Score (MMES): A New Index for the Assessment of Extension and Severity of Endoscopic Activity in Ulcerative Colitis Patients. J Crohns Colitis. 2015 Oct;9(10):846-52. doi: 10.1093/ecco-jcc/jjv111. Epub 2015 Jun 26.
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- 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.
- Tedelind S, Westberg F, Kjerrulf M, Vidal A. Anti-inflammatory properties of the short-chain fatty acids acetate and propionate: a study with relevance to inflammatory bowel disease. World J Gastroenterol. 2007 May 28;13(20):2826-32. doi: 10.3748/wjg.v13.i20.2826.
- 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.
- Sekirov I, Russell SL, Antunes LC, Finlay BB. Gut microbiota in health and disease. Physiol Rev. 2010 Jul;90(3):859-904. doi: 10.1152/physrev.00045.2009.
- Gevers D, Kugathasan S, Denson LA, Vazquez-Baeza Y, Van Treuren W, Ren B, Schwager E, Knights D, Song SJ, Yassour M, Morgan XC, Kostic AD, Luo C, Gonzalez A, McDonald D, Haberman Y, Walters T, Baker S, Rosh J, Stephens M, Heyman M, Markowitz J, Baldassano R, Griffiths A, Sylvester F, Mack D, Kim S, Crandall W, Hyams J, Huttenhower C, Knight R, Xavier RJ. The treatment-naive microbiome in new-onset Crohn's disease. Cell Host Microbe. 2014 Mar 12;15(3):382-392. doi: 10.1016/j.chom.2014.02.005.
- Putignani L, Del Chierico F, Petrucca A, Vernocchi P, Dallapiccola B. The human gut microbiota: a dynamic interplay with the host from birth to senescence settled during childhood. Pediatr Res. 2014 Jul;76(1):2-10. doi: 10.1038/pr.2014.49. Epub 2014 Apr 14.
- Nikhil Pai 2018. "PediCRaFT: Pediatric Crohn's Disease Fecal Transplant Trial". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03378167.
Frequently Asked Questions
What criteria makes an individual a viable candidate for this experiment?
"This trial has room for 45 qualified candidates with ileocolitis aged between 3 and 17. In addition, participants must have pediatric Crohn's disease or IBD-Unclassified favouring Crohn's Disease (as determined by the patient's primary gastroenterologist) as well as active symptoms." - Anonymous Online Contributor
Unverified Answer
Are individuals younger than 50 years old acceptable to enrol in this clinical research?
"This clinical trial has specific requirements for applicants, stipulating that they must be between 3 and 17 years old. There are 131 different trials available to youngsters under 18 while there are 430 studies directed towards seniors over 65." - Anonymous Online Contributor
Unverified Answer
What is the total participant quota for this research endeavor?
"Affirmative. According to clinicaltrials.gov, the research trial which first went live on December 1st 2018 is still accepting new participants. The study seeks 45 individuals from two separate medical locations and was last edited April 23rd 2022." - Anonymous Online Contributor
Unverified Answer
Has MICROBIOTA been granted official authorization from the FDA?
"MICROBIOTA's safety rating was given a score of 1 by Power due to the limited amount of data present that supports its efficacy and safety." - Anonymous Online Contributor
Unverified Answer
What are the chief objectives of this clinical experimentation?
"This 30-week trial will measure the dropout rates post enrolment and also assess certain clinical outcomes such as a remission in disease symptoms (PCDAI ≤ 10 at Week 6, Week 30), an improvement in mucosal inflammatory markers (decrease fecal calprotectin from baseline - Week 6, Week 30) and improved overall symptom management (PCDAI decrease ≥15 from baseline - week 6, week 30)." - Anonymous Online Contributor
Unverified Answer
Are researchers currently seeking participants for this experiment?
"The information accessible on clinicaltrials.gov attests that this medical research is actively seeking volunteers, which was initially published on December 1st 2018 and most recently edited on April 23rd 2022." - Anonymous Online Contributor
Unverified Answer