FMT in Pediatric Crohn's Disease

NCT03194529 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: CHLA-17-00221
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Crohn Disease; Pediatric Crohns Disease

Keywords

Crohn's Disease; PCDAI; 16s rRNA; Fecal Microbiota Transplantation; Gut Microbiome; FMT

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

  Safety will be closely monitored (during defined telephone calls and study visits) and adverse events will be documented, including mucosal inflammation episodes and standard of care laboratory test abnormalities.

  24 weeks

Secondary Outcomes (2)

• Crohn's disease activity

  24 weeks

• Changes in gut microbiome

  24 weeks

Study Arms (1)

FMT in children with disease remission

EXPERIMENTAL

All children (with Crohn's disease in remission) will receive the equivalent of 50 g of stools from a healthy donor (FMT) into the jejunum through upper endoscopy.

Biological: FMT

Interventions

FMT BIOLOGICAL

Fecal Microbiota Transplantation, single dose, 50g of stool, delivered via standard of care upper endoscopy into jejunum.

Also known as: Fecal Microbiota Transplantation

FMT in children with disease remission

Eligibility Criteria

• Age: 7 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age: 7-21 who have been diagnosed with Crohn's disease
• Remission of disease defined as PCDAI \<10
• Needs upper GI endoscopy

You may not qualify if:

• Unwilling to give informed consent/assent
• 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 Crohn's disease (such as neoplastic disease or organ transplantation), have received or are receiving chemotherapy, or have been diagnosed with HIV.
• Subjects with severe food allergies

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 (5)

• Rinawi F, Assa A, Hartman C, Mozer Glassberg Y, Friedler VN, Rosenbach Y, Silbermintz A, Zevit N, Shamir R. Incidence of Bowel Surgery and Associated Risk Factors in Pediatric-Onset Crohn's Disease. Inflamm Bowel Dis. 2016 Dec;22(12):2917-2923. doi: 10.1097/MIB.0000000000000937.

  PMID: 27755214 BACKGROUND

• Shi Y, Dong Y, Huang W, Zhu D, Mao H, Su P. Fecal Microbiota Transplantation for Ulcerative Colitis: A Systematic Review and Meta-Analysis. PLoS One. 2016 Jun 13;11(6):e0157259. doi: 10.1371/journal.pone.0157259. eCollection 2016.

  PMID: 27295210 BACKGROUND

• Sartor RB. Microbial influences in inflammatory bowel diseases. Gastroenterology. 2008 Feb;134(2):577-94. doi: 10.1053/j.gastro.2007.11.059.

  PMID: 18242222 BACKGROUND

• Bakhtiar SM, LeBlanc JG, Salvucci E, Ali A, Martin R, Langella P, Chatel JM, Miyoshi A, Bermudez-Humaran LG, Azevedo V. Implications of the human microbiome in inflammatory bowel diseases. FEMS Microbiol Lett. 2013 May;342(1):10-7. doi: 10.1111/1574-6968.12111. Epub 2013 Mar 15.

  PMID: 23431991 BACKGROUND

• D'Inca R, Annese V, di Leo V, Latiano A, Quaino V, Abazia C, Vettorato MG, Sturniolo GC. Increased intestinal permeability and NOD2 variants in familial and sporadic Crohn's disease. Aliment Pharmacol Ther. 2006 May 15;23(10):1455-61. doi: 10.1111/j.1365-2036.2006.02916.x.

  PMID: 16669960 BACKGROUND

Related Links

• fecal-microbial-transplant-program at CHLA

MeSH Terms

Conditions

Crohn Disease

Interventions

Fecal Microbiota Transplantation

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Officials

• Sonia Michail, MD

  Children's Hospital Los Angeles

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Primary outcome is safety in children with Crohn's disease in remission.

Study Record Dates

• First Submitted: June 19, 2017
• First Posted: June 21, 2017
• Study Start: October 9, 2017
• Primary Completion: June 30, 2019
• Study Completion: July 31, 2019
• Last Updated: March 13, 2020

Record last verified: 2020-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)