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)
Journal of Urology
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.
Chancellor MB, Peters KM, Benson KD, Thomas S, Bennett JB, Dmochowski RR, Heit MH, et al. 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). J Urol. 2021 Sep;206(3 suppl):e99-100.