Secondary Logo

Journal Logo

Urodynamics/Lower Urinary Tract Dysfunction/Female Pelvic Medicine: Female Incontinence: Therapy I (PD06): Podium 6: Friday, September 10, 2021

PD06-01 A DOUBLE-BLIND, RANDOMIZED, CONTROLLED TRIAL COMPARING SAFETY AND EFFICACY OF AUTOLOGOUS MUSCLE DERIVED CELLS FOR URINARY SPHINCTER REPAIR (AMDC-USR) WITH PLACEBO (PBO) IN WOMEN WITH STRESS URINARY INCONTINENCE (SUI)

Kaufman, Melissa R.; Peters, Kenneth M.; Chermansky, Christopher J.; Quiroz, Lieschen H.; Bennett, Jason B.; Thomas, Sherry; Goldman, Howard B.; Benson, Kevin D.; Dmochowski, Roger R.; Lee, Una J.; Sokol, Eric R.; Galloway, Niall T.M.; Wolter, Christopher E.; Kennelly, Michael J.; Tarnay, Christopher M.; Heit, Michael H.; Rehme, Christian; Jankowski, Ron J.; Chancellor, Michael B.

doi: 10.1097/JU.0000000000001974.01
  • Free

INTRODUCTION AND OBJECTIVE:

This multicenter Phase 3 study (NCT01893138) evaluated efficacy and safety of AMDC-USR compared to PBO for SUI treatment.

METHODS:

297 adult women with average 14.4 ± 11.4 stress leaks over 3 days were randomized 2:1 (150x106 AMDC-USR:vehicle PBO) and stratified by baseline severity and prior SUI surgery. AMDC-USR was manufactured from vastus lateralis harvest via outpatient biopsy and injected into the urinary sphincter at a subsequent outpatient procedure. SUI was monitored by 3-day diary of stress incontinence episode frequency (SIEF) and quality of life (QOL) questionnaires. Subjects were unblinded after 12 months, and PBO subjects could receive an open-label AMDC-USR treatment. Subjects were followed for 2 years post initial treatment.

RESULTS:

297 women were treated (199 AMDC-USR; 98 PBO), 99% completed 12 months (199 AMDC-USR; 96 PBO), and 85% have completed 2 years (167 AMDC-USR; 86 PBO). 93% PBO subjects opted to receive open-label AMDC-USR treatment. Percentage of SIEF responders by strata, endpoints and visits is presented below. SIEF reduction correlated with improvement in all QOL scores at 12 months (p < 0.001). Treatment-related serious adverse reactions were uncommon (< 1%) with no AMDC-USR safety signals detected.

CONCLUSIONS:

Single injection of AMDC-USR is safe and durable through 2 years with varying therapeutic effect. Consistent with our previous study, ≥ 50% SIEF reduction is not a sufficient endpoint. High variability in PBO response rates across strata impacts generalizability of the treatment response in the overall population. Nonetheless, there was encouraging response in subjects with > 10 baseline SIE, and a markedly greater percentage (> 2-fold) of women with prior surgery achieved statistical and clinically meaningful ≥ 75% SIEF reduction compared with PBO, further supporting that this population may be ideally suited for AMDC-USR therapy.

Source of Funding:

Cook MyoSite Incorporated

© 2021 by American Urological Association Education and Research, Inc.