Clinical trial NCT01972334: Stool Transplant in Pediatric Patients With Recurring C. Difficile Infection
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Clostridium Difficile Colitis
Keywords: clostridium difficile
children
fecal transplant
Lead sponsor: MemorialCare Health System
Sponsor class: OTHER
Collaborators: name: Memorial Medical Center Foundation
First posted: 2013-10-30
Enrollment: 9
Sex: ALL
Minimum age (years): 1
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: The primary efficacy outcome of this randomized, controlled, double-blinded study is the time of recurrence of an infection
timeFrame: 12 months
Secondary outcomes:
measure: safety of fecal microbial transplant in children with recurrent clostridium difficile
timeFrame: 12 months
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Miller Children's Hospital
state: California
Study officials:
affiliation: MemorialCare Health Servies
name: Sonia Michail, MD
role: PRINCIPAL_INVESTIGATOR
References: