"A Pilot Trial Comparing the Effects of Onabotulinumtoxina and Standard" by Sumit Dave
Electronic Thesis and Dissertation Repository

Degree

Master of Science

Program

Epidemiology and Biostatistics

Supervisor

Dr George Rodrigues

Abstract

Research question

Is it feasible to conduct a phase III RCT to compare OnabotulinumtoxinA injections to oxybutynin as primary therapy in pediatric neurogenic bladder?

Methods

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.

Results

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.

Conclusion

It is feasible and safe to conduct a phase III trial to investigate the efficacy of primary OnabotulinumtoxinA compared to oxybutynin therapy.

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