Master of Science
Epidemiology and Biostatistics
Dr George Rodrigues
Is it feasible to conduct a phase III RCT to compare OnabotulinumtoxinA injections to oxybutynin as primary therapy in pediatric neurogenic bladder?
Patients on a stable oxybutynin regimen were recruited for a pilot RCT and underwent randomization to either OnabotulinumtoxinA or continuation of oxybutynin. Primary outcomes included an a priori defined feasibility and acceptability assessment. Secondary outcomes included continence, urodynamic parameters, side effects and QOL.
The study enrolled 8 subjects in the OnabotulinumtoxinA group and 6 in the oxybutynin group. The recruitment rate was 75 % and the dropout rate was 6.6 %. There were 2 minor protocol deviations. There were no side effects in the botulinum group compared to 66.7% in the oxybutynin group (p=0.02). The clinical and QOL outcomes were comparable.
It is feasible and safe to conduct a phase III trial to investigate the efficacy of primary OnabotulinumtoxinA compared to oxybutynin therapy.
Dave, Sumit, "A Pilot Trial Comparing the Effects of Onabotulinumtoxina and Standard Oxybutynin Therapy as First Line Treatment for the Poorly Compliant Pediatric Neurogenic Bladder" (2012). Electronic Thesis and Dissertation Repository. 860.