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Clinical Trials/NCT02423967
NCT02423967CompletedPhase 1

A Comparison of Efficacy of Fresh Versus Frozen Donor Stool for Fecal Microbial Transplant in Children With Recurrent Clostridium Difficile Infections

Mayo Clinic2 sites in 1 country28 target enrollmentStarted: April 2015Last updated:
Conditions
Interventions

Trial Snapshot

Phase
Phase 1
Status
Completed
Enrollment
28
Locations
2
Primary Endpoint
Clostridium difficile (CD) Eradication; percentage of patients with negative CD toxin in stool following Fecal Microbiota Transplant

Study Overview

Brief Summary

The primary goal of this study will be to assess whether stool collected and frozen from anonymous screened unrelated donors can be as effective as stool freshly collected from recipient's parents when used in Fecal Microbial Transplant for the eradication of recurrent Clostridium difficile infections in children. In the current protocols, which are more than 90% effective, each child who is receiving a fecal transplant has to provide their own donor stool, usually from a parent or close relative. This requires considerable screening costs for each case and is logistically complicated as the donor must be present and must stool just prior to the transplant. The investigators hope to show that a small number of healthy donors can provide stool samples which can be frozen and banked and then thawed for use in numerous patients. The primary goal is to show that Clostridium difficile will be eradicated as effectively (Greater than 90% success) when using the stool from the frozen donors.

The study will also evaluate the inflammatory response and intestinal microbiome in young children aged 1-3 years with Clostridium difficile infections to better predict which ones will respond to fecal transplantation and which ones have incidental infections. For this question the investigators will gather stool samples to check for lactoferrin, calprotectin, and alpha1antitrypsin, and 16s ribosomal RNA analysis in children before and after the fecal transplants. The goal is to see if there is an intestinal microbiome that predisposes some children to getting sick from Clostridium difficile versus just having it incidentally.

Detailed Description

The study is designed to enroll forty children aged 1-18 with recurrent Clostridium difficile Infection (CDI). Recurrent CDI is defined as an infection that persists after three rounds of appropriate antibiotics. Enrollees will be randomly assigned to receive Fecal Microbial Transplant using stool from either a screened relative as per current protocols versus using frozen stool that has been collected from healthy volunteers.

Primary outcome will be the eradication of the Clostridium difficile as defined by elimination of diarrhea with a negative Clostridium difficile toxin stool test.

Secondary outcomes will include a measure of quality of life pre and post Fecal Microbial Transplant using a validated quality of life survey instrument, the Health Act Child Health Questionnaire. The investigators will also assess for inflammation in the stool using lactoferrin and calprotectin and evaluate the stool microbiome in recipients pre and post transplant using sequencing to look for any pattern that predicts successful eradication.

Study Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
1 Year to 18 Years (Child, Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Not provided

Exclusion Criteria

  • Age under 1 or over 18
  • No diarrhea

Arms & Interventions

Transplant uses frozen anonymous stool

Experimental

Undergoes Fecal Microbial Transplant using stool collected from screened anonymous donor that has been frozen until time of Fecal Microbial Transplant Followed up and assessed for eradication of Clostridium difficile Quality of life assessed Stool assessed for inflammation and microbiome

Intervention: Transplant uses frozen anonymous stool (Biological)

Transplant uses fresh familial stool

Active Comparator

Undergoes Fecal Microbial Transplant using fresh stool from a screened family member Followed up and assessed for eradication of Clostridium difficile Quality of life assessed Stool assessed for inflammation and microbiome

Intervention: Transplant uses fresh familial stool (Biological)

Outcomes

Primary Outcomes

Clostridium difficile (CD) Eradication; percentage of patients with negative CD toxin in stool following Fecal Microbiota Transplant

Time Frame: 4 weeks

Will analyze stool sample collected 4 weeks after fecal microbiota transplant (FMT) for presence or absence of Clostridium difficile toxin. In addition, will note whether presenting symptoms improved or not after FMT.

Secondary Outcomes

  • Changes in quality of life questionnaire scores after Fecal Microbiota Transplant(12 weeks)
  • percentage of patients with stool Calprotectin levels exceeding 50 mcg/g after Fecal Microbiota Transplant(4 weeks)
  • percentage of patients with positive Lactoferrin inflammatory marker in stools after Fecal Microbiota Transplant(4 weeks)

Investigators

Sponsor Class
Other
Responsible Party
Principal Investigator
Principal Investigator

Mark G. Bartlett, M.D.

PI

Mayo Clinic

Study Sites (2)

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