A double-blind, randomized, placebo-controlled clinical trial evaluating the safety and efficacy of autologous muscle derived cells in female subjects with stress urinary incontinence.
International urology and nephrology
PURPOSE: The purpose of the study was to assess safety and efficacy of autologous muscle derived cells for urinary sphincter repair (AMDC-USR) in female subjects with predominant stress urinary incontinence.
METHODS: A randomized, double-blind, multicenter trial examined intra-sphincteric injection of 150 × 10
RESULTS: AMDC-USR was safe and well-tolerated with no product-related serious adverse events or discontinuations due to adverse events. Interim analysis revealed an unexpectedly high placebo response rate (90%) using the composite primary outcome which prevented assessment of treatment effect as designed and thus enrollment was halted at 61% of planned subjects. Post hoc analyses suggested that more stringent endpoints lowered placebo response rates and revealed a possible treatment effect.
CONCLUSIONS: Although the primary efficacy finding was inconclusive, these results inform future trial design of AMDC-USR to identify clinically meaningful efficacy endpoints based on IEF reduction, understanding of placebo response rate, and refinement of subject selection criteria to more appropriately align with AMDC-USR's proposed mechanism of action.
Jankowski RJ, Tu LM, Carlson C, Robert M, Carlson K, Quinlan D, Eisenhardt A, Chen M, Snyder S, Pruchnic R, Chancellor M, Dmochowski R, Kaufman MR, Carr L. A double-blind, randomized, placebo-controlled clinical trial evaluating the safety and efficacy of autologous muscle derived cells in female subjects with stress urinary incontinence. Int Urol Nephrol. 2018 Dec;50(12):2153-2165. doi: 10.1007/s11255-018-2005-8. Epub 2018 Oct 15. PMID: 30324580.