Protocol for a double-blind, randomised, placebo-controlled pilot study for assessing the feasibility and efficacy of faecal microbiota transplant in a paediatric Crohn’s disease population: PediCRaFT Trial

What is it about?

Crohn's Disease (CD) is an incurable disease that causes severe inflammation of the intestines. Canada has one of the highest rates of CD in the entire world, particularly affecting children under 10 years old. Childhood-onset CD is extremely aggressive. Children experience poor growth, severe unpredictable abdominal pain, frequent school absences, and symptoms that are both debilitating and embarrassing. Treatment involves lifelong medications, which are not always effective and may cause infections and cancers. Longterm medication needs in children only increases their lifetime risks of developing these side effects. No one knows why Crohn's disease happens, but one theory is due to the types of bacteria in a person's intestine. In 2015, a team of McMaster University researchers showed that transferring healthy bacteria into adults with ulcerative colitis is safe, and effective. This treatment, known as fecal microbiota transplant (FMT), may offer a novel alternative to medication therapies. FMT has never been studied for the treatment of CD in a formal clinical trial. We seek to change that. The aim of this study is to test whether FMT is an effective treatment in children with active CD. To answer this, we will assign children with CD to receive either FMT treatments from a healthy donor, or plain salt water (placebo). The treatment will be given first during a colonoscopy, then as specialized FMT pills taken by mouth for another 6 weeks. We will recruit participants from two different hospitals, and will monitor symptoms, stool samples and bloodwork to know if they improve. A study like this has never been performed, and our strategy of using a combination of colonoscopy + oral pills to deliver FMT treatments is new. Therefore, our trial is called a “pilot.” If we can successfully run our study at two different hospitals, we will be able to justify, and adjust our trial in the future to include many more hospitals and patients. Therefore, we will measure how patients are responding to FMT, and whether our trial is running well. Our team recently completed the first study in the world of FMT in children with Ulcerative Colitis. We will leverage 3 years of experience for another first. We strive to find new treatments for children and adults struggling with this life-altering disease. This trial, across two hospitals, will give us invaluable data to launch a future, Canada-wide study to assess the role of FMT in young Canadian CD patients.

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