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.

Document Type

Article

Publication Date

12-1-2018

Publication Title

International urology and nephrology

Abstract

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.

Volume

50

Issue

12

First Page

2153

Last Page

2165

DOI

10.1007/s11255-018-2005-8

ISSN

1573-2584

PubMed ID

30324580

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