Stool Transplant in Pediatric Patients With Recurring C. Difficile Infection

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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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

Conditions
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Clostridium Difficile Colitis

Intervention / Treatment
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* Biological: FMT
* Biological: placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age: 1-21. Recurrent C. difficile infection defined as the occurrence of more than two infections Exclusion Criteria: Inflammatory bowel disease Immune-deficiency. Allergy to oral vancomycin. Children colonized with Clostridium difficile without evidence of symptoms to suggest colitis such as diarrhea and/or rectal bleeding. Concurrent infections that require anti-microbial therapy. Unable to give informed consent/assent. Pregnancy.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: FMT or fecal microbial transplant<br>intervention is fecal microbial transplant done through endoscopy, subjects will be randomized 1:1 to receive either FMT or placebo | Biological: FMT<br>* as explained in study arm<br>* Other names: stool transplant;|
| Placebo Comparator: placebo<br>1:1 randomization to FMT versus placebo (which is saline or salt water) | Biological: placebo<br>* salt water or saline will be given as placebo<br>* Other names: salt water;|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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clostridium difficile, children, fecal transplant