Clinical Trials (Clinician Perspective)

Pediatric patients with IBD are increasingly candidates for future clinical trials. Until now, very few RCTs have been conducted in pediatric populations with IBD. The main challenge will be the use of new therapies for treating active disease as well as for inducing and maintaining remission. However, accurate choice of the patient phenotype to be enrolled in the clinical trials is crucial for reaching both meaningful results and conclusions. Due to the heterogeneous nature of IBD, homogeneous populations of patients should be studied and compared even if this could prolong patient recruitment time and make the screening process more complex. The principal variables that should be taken into account when patients with IBD are evaluated for clinical trial are: disease type; severity; topographic distribution; complications (i.e., presence of fistulas in CD); weight and linear growth pattern; serological markers for activity, nutrition, and absorption; and genotype. The latter will be increasingly included among variables capable to influence the outcome of different treatments as more insights into genotype–phenotype correlations will be gained and other genes of susceptibility for IBD will be described.