Urodynamics/Lower Urinary Tract Dysfunction/Female Pelvic Medicine: Female Incontinence: Therapy I (PD06): Podium 6: Friday, September 10, 2021
PD06-02 AN EVALUATION OF WOMEN WITH PERSISTENT OR RECURRENT STRESS URINARY INCONTINENCE (SUI) FOLLOWING SURGERY IN A DOUBLE-BLIND, RANDOMIZED, CONTROLLED TRIAL COMPARING SAFETY AND EFFICACY OF AUTOLOGOUS MUSCLE DERIVED CELLS FOR URINARY SPHINCTER REPAIR (AMDC-USR) WITH PLACEBO (PBO)
INTRODUCTION AND OBJECTIVE:
Women with persistent or recurrent SUI following prior surgical intervention have an unmet medical need with symptoms which substantially impact daily functioning and quality of life (QOL).
Data provided are a stratified subset from a Phase 3 study (NCT01893138). 75 women with average 19.8±11.3 stress leaks over 3 days were randomized 2:1 (150 x 106 AMDC-USR:vehicle PBO) at 22 sites. AMDC-USR was manufactured from skeletal muscle tissue harvested from the vastus lateralis via outpatient biopsy and injected into the urinary sphincter during a subsequent outpatient procedure. SUI was monitored by 3-day diaries of stress incontinence episode frequency (SIEF) and QOL questionnaires during blinded follow-up through 12 months.
All women completed 12-month visits (50 AMDC-USR; 25 PBO). Treatment effect at ≥75% SIEF reduction was robust and significant. SIEF reduction in AMDC-USR correlated with improvement in all QOL scores at 12 months (p≤0.002) and exceeded clinically meaningful levels of ≥ 10-point improvement in I-QOL score at ≥ 75% reduction in SIEF. There were significant differences in QOL changes between responders and non-responders in the AMDC-USR group only. There were no AMDC-USR-related serious adverse reactions.
Women with chronic SUI symptoms (average > 12 years) sought treatment despite undergoing as many as five prior surgeries at the time of enrollment. A single injection of AMDC-USR may be a safe and effective treatment in this challenging refractory population. Based in part on this evidence to address unmet medical need and the recognized seriousness of the condition, AMDC-USR has been granted the expedited Regenerative Medicine Advanced Therapy (RMAT) designation by the United States Food and Drug Administration for this population.
Source of Funding:
Cook MyoSite Incorporated© 2021 by American Urological Association Education and Research, Inc.