Stool Transplant in Pediatric Patients With Recurring C. Difficile Infection

A Randomized, Placebo-controlled Pilot Trial to Administer Fecal Microbial Therapy (Stool Transplant) or Placebo in Children Ages 8 to 18 With Recurrent C. Difficile Infection

The incidence of C. difficile infection (CDI) has alarmingly increased over the past several years and the affected population has expanded to include those previously at low risk, such as children. The annual US financial burden associated with this infection is great and estimated to exceed $1.8 billion. C. difficile infection arises when the gut microbial ecology is disrupted during interventions notorious for perturbing the delicate microbial balance. A well known and common example is the use of antibiotics. Fecal microbiota transplant (FMT) has been introduced several decades ago in an attempt to restore the gut microbial balance. To this date there have been a great number of reports of success in eliminating recurrent C. difficile infections and restoring the gut microbial profile to resemble that of the healthy donor. While over 300 cases have been described in the literature, there has been no pediatric controlled studies performed to compare its efficacy to placebo. Therefore, there is a strong need to determine their safety and efficacy in pediatric randomized controlled studies. The investigators hypothesize that children with recurrent C. difficile infection will respond to fecal transplant therapy which will modify their gut microbial profile. The investigators propose a randomized, placebo controlled, pilot study of fecal microbial transplant in children with recurrent C. difficile infection to evaluate the safety and efficacy of fecal microbial transplant in children in preventing recurrent C. difficile infection. The investigators anticipate that fecal microbial transplant in children with recurrent C. difficile infection will be safe and efficacious and will provide these children with a great alternative to a disease that is difficult to treat. Results of this study will establish the major role of the gut microbiome in this disease and demonstrate the viability of gut microbial transplant in recipients.

Study Overview

• Status: Completed
• Conditions: Clostridium Difficile Colitis
• Intervention / Treatment: Biological: FMT; Biological: placebo

Detailed Description

Forty six children with recurrent C. difficle infection will be randomized 1:1 to receive either fecal microbial transplant or placebo. The safety and efficacy of this intervention will be monitored for one year.

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90806
      • Miller Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 21 years (ADULT, CHILD)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Age: 1-21.
2. Recurrent C. difficile infection defined as the occurrence of more than two infections

Exclusion Criteria:

1. Inflammatory bowel disease
2. Immune-deficiency.
3. Allergy to oral vancomycin.
4. Children colonized with Clostridium difficile without evidence of symptoms to suggest colitis such as diarrhea and/or rectal bleeding.
5. Concurrent infections that require anti-microbial therapy.
6. Unable to give informed consent/assent.
7. Pregnancy.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: FMT or fecal microbial transplant; intervention is fecal microbial transplant done through endoscopy, subjects will be randomized 1:1 to receive either FMT or placebo	Biological: FMT; as explained in study arm; Other Names: stool transplant; fecal microbial transplant
PLACEBO_COMPARATOR: placebo; 1:1 randomization to FMT versus placebo (which is saline or salt water)	Biological: placebo; salt water or saline will be given as placebo; Other Names: saline; salt water

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The primary efficacy outcome of this randomized, controlled, double-blinded study is the time of recurrence of an infection	secondary outcome measures are focused on the safety of Fecal microbial transplant in this population and all subjects will be closely monitored for developing adverse events for 12 months	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
safety of fecal microbial transplant in children with recurrent clostridium difficile	All subjects will be closely monitored for adverse events for 12 months after fecal transplant. Subjects will be asked to report any adverse events as they occur and will also be monitored during each visits especially fever, bloating, abdominal pain, vomiting, diarrhea, and rectal bleeding.	12 months

Collaborators and Investigators

• Sponsor: MemorialCare Health System
• Collaborators: Memorial Medical Center Foundation
• Investigators: Principal Investigator: Sonia Michail, MD, MemorialCare Health Servies

Study record dates

Study Major Dates

• Study Start: October 1, 2013
• Primary Completion (ACTUAL): July 1, 2016
• Study Completion (ACTUAL): July 1, 2016

Study Registration Dates

• First Submitted: October 18, 2013
• First Submitted That Met QC Criteria: October 24, 2013
• First Posted (ESTIMATE): October 30, 2013

Study Record Updates

• Last Update Posted (ESTIMATE): September 1, 2016
• Last Update Submitted That Met QC Criteria: August 30, 2016
• Last Verified: August 1, 2016

More Information

Terms related to this study

• Keywords: children; fecal transplant; clostridium difficile
• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Colonic Diseases; Intestinal Diseases; Colitis
• Other Study ID Numbers: 093-12

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED