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)

Document Type

Conference Proceeding

Publication Date

9-2021

Publication Title

Journal of Urology

Abstract

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).

METHODS:

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.

RESULTS:

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.

CONCLUSIONS:

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.

Volume

206

Issue

3 suppl

First Page

e99

Last Page

e100

Comments

The American Urological Association Annual Meeting, Virtual, September 10-13, 2021.

DOI

10.1097/JU.0000000000001974.02

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