The Effect of Therapeutic Fecal Transplant on the Gut Microbiome in Children With Ulcerative Colitis

NCT02291523 | Unknown Phase 1 DMC FDA Drug

• 1 other identifier: CHLA-16-00050
• Study Type: interventional
• Target: 101
• Locations: 1 country
• Sites: 1

Brief Summary

Ninety Six patients with mild to moderate ulcerative colitis will be randomized to double blind, placebo controlled study. The safety and efficacy of the intervention will be closely monitored.

Conditions

Ulcerative Colitis

Keywords

Fecal transplant

Outcome Measures

Primary Outcomes (1)

• The primary endpoint is disease remission based on PUCAI scores (<10).

  The primary outcome including results of disease activity, and safety measures.

  12 Months

Secondary Outcomes (1)

• Secondary endpoints will include change in mucosal inflammation reflected on laboratory studies.

  12 Months

Study Arms (2)

Patient Stool Transplant

SHAM COMPARATOR

Arm 1 will get FMT (Fecal Microbial Transplant) placebo and high dose 5-ASA (Pentasa). The FMT is done through colonoscopy.

Biological: Fecal Microbial Transplant

Donor Stool Transplant

ACTIVE COMPARATOR

Arm 2 will get FMT (Fecal Microbial Transplant) with Healthy Donor Stool and high dose 5-ASA (Pentasa). The FMT is done through colonoscopy.

Biological: Fecal Microbial Transplant

Interventions

Fecal Microbial Transplant BIOLOGICAL

Fecal Transplant via Colonoscopy.

Also known as: FMT

Donor Stool Transplant; Patient Stool Transplant

Eligibility Criteria

• Age: 7 Years - 21 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age: 7-21 who have been diagnosed with ulcerative colitis
• Mild to moderate disease based on PUCAI with a score of 10-64
• Need for colonoscopy

You may not qualify if:

• Children who are known to be resistant to steroid therapy, immunomodulators and biologics, or on a steroid dose greater than 0.5 mg/kg/day (maximum 20 mg)
• Children with recent dose change of biologics (within 4 weeks), 5-ASA, steroids or immunomodulators (within 4 weeks)
• Allergy to or intolerance of mesalamine or 5-ASA products
• Any evidence of infectious colitis
• Concurrent infections that require anti-microbial therapy (such as abdominal abscess, pneumonia, etc…)
• Unable to give informed consent/assent
• Have received probiotic preparations ≤ 4 weeks prior to randomization
• Pregnancy and breast feeding in patient subjects of childbearing potential
• Subjects with significant renal and liver dysfunction (creatinine \> 2 mg/dl and direct bilirubin \> 2 mg/dl), Subjects with congenital or acquired immunodeficiency, or who are immunosuppressed due to conditions other than ulcerative colitis (such as neoplastic disease or organ transplantation), have received or are receiving chemotherapy, or have been diagnosed with HIV.

Sponsors & Collaborators

• Children's Hospital Los Angeles: lead

Study Sites (1)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Related Publications (4)

• Michail S, Bultron G, Depaolo RW. Genetic variants associated with Crohn's disease. Appl Clin Genet. 2013 Jul 16;6:25-32. doi: 10.2147/TACG.S33966. Print 2013.

  PMID: 23935379 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

• Hyams JS, Lerer T, Mack D, Bousvaros A, Griffiths A, Rosh J, Otley A, Evans J, Stephens M, Kay M, Keljo D, Pfefferkorn M, Saeed S, Crandall W, Michail S, Kappelman MD, Grossman A, Samson C, Sudel B, Oliva-Hemker M, Leleiko N, Markowitz J; Pediatric Infl ammatory Bowel Disease Collaborative Research Group Registry. Outcome following thiopurine use in children with ulcerative colitis: a prospective multicenter registry study. Am J Gastroenterol. 2011 May;106(5):981-7. doi: 10.1038/ajg.2010.493. Epub 2011 Jan 11.

  PMID: 21224840 BACKGROUND

• Le J, Hakimjavadi H, Parsana R, Chamala S, Michail S. Fecal Microbiota Transplantation Induces Sustained Gut Microbiome Changes in Pediatric Ulcerative Colitis: A Combined Randomized and Open-Label Study. Gastro Hep Adv. 2025 Jul 11;4(10):100741. doi: 10.1016/j.gastha.2025.100741. eCollection 2025.

  PMID: 40933007 DERIVED

MeSH Terms

Conditions

Colitis, Ulcerative

Condition Hierarchy (Ancestors)

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

Study Officials

• Sonia Michail, MD

  Children Hospital Los Angeles

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Clinical Pediatrics

Study Record Dates

• First Submitted: November 11, 2014
• First Posted: November 14, 2014
• Study Start: November 1, 2016
• Primary Completion: November 1, 2023
• Study Completion: November 1, 2023
• Last Updated: November 2, 2023

Record last verified: 2023-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)