Status: Funded - Open
Fecal microbiota transplantation for severe acute malnutrition: A pilot study
Majdi Osman, MD, MPH
BACKGROUND: Severe acute malnutrition (SAM) is a significant global health problem whose long-term sequelae, including stunting, neuro-developmental abnormalities, and immune dysfunction remain largely refractory to current therapeutic interventions of ready to use therapeutic food and antibiotics.
SAM affects approximately 19 million children under 5 years worldwide with no clinical response in approximately 36% of uncomplicated SAM cases treated by standard recommended WHO therapy and 1 million deaths in children annually.
GAP: In recent years, a growing body of evidence has shown that children with SAM exhibit impaired development of their gut microbiota and that microbiota immaturity is causally related to malnutrition and that a microbial therapeutic could play a role in treatment for SAM unresponsive to standard therapy.
Building off these insights, this study will assess whether fecal microbiotia transplantation (FMT) can enable recovery of children with SAM not responsive to standard therapy and potentially identify novel therapeutics for restoring the microbiome of children with SAM.
HYPOTHESIS: We hypothesize that FMT will be an effective and safe broad-spectrum microbial therapy for SAM.
METHODS: This randomized, placebo controlled study with will assess safety and tolerability of FMT in SAM and generate preliminary microbiological and clinical data on FMT in children with uncomplicated SAM refractory to standard WHO therapy.
20 children (10 treatment, 10 control) will be recruited from the Department of Pediatrics at the University of Cape Town, South Africa with the treatment arm receiving FMT by enema according to a standard protocol for FMT in children.
IMPACT: The findings from the pilot study will inform the design of a larger randomized controlled trial to assess fecal microbiotia transplantation for SAM with the view to identify particular strains for a novel microbial therapy for SAM.
Website Link: openbiome.org
United States, South Africa