This study aimed to determine the efficacy and safety of iltamiocel investigational autologous muscle cell therapy in females with stress urinary incontinence (SUI).
Adult females were randomized 2:1 to iltamiocel (150 × 106 cells) or placebo and stratified by severity and prior SUI surgery. The primary objective was efficacy based on the frequency of stress incontinence episodes (SIE) recorded in a 3-day diary at 12 months posttreatment. After 12 months, placebo participants could elect to receive open-label iltamiocel. Efficacy and safety analyses were performed using all patients as treated populations.
The study enrolled 311 patients, 297 were randomized to either iltamiocel (n = 199) or placebo (n = 98). Of the 295 participants that completed 12 months blinded follow-up, the proportion achieving the primary endpoint of ≥ 50% SIE reduction was not statistically different between treatment groups (52% vs. 53.6%; p = 0.798). A significantly greater proportion of iltamiocel participants in the prior SUI surgery stratum group achieved ≥ 75% SIE reduction compared with placebo, (40% vs. 16%; p = 0.037). Treatment response was maintained at 24 months in 78.4% and 64.9% of iltamiocel participants who achieved ≥ 50% and ≥ 75% SIE reduction, respectively, at Month 12. Adverse events related to the treatment were reported in 19 (9.5%) iltamiocel participants and 6 (6.1%) placebo participants.
The study did not meet its primary endpoint however, iltamiocel cell therapy is safe and may be ideally suited to female patients who have undergone prior surgery for SUI. Additional study in this group of patients with high unmet medical needs is warranted.
ClinicalTrials.gov identifier: NCT01893138; EudraCT number: 2014-002919-41.
Neurourology and urodynamics. 2024 Sep 16 [Epub ahead of print]
Melissa R Kaufman, Howard B Goldman, Christopher J Chermansky, Roger Dmochowski, Michael J Kennelly, Kenneth M Peters, Lieschen H Quiroz, Jason B Bennett, Sherry Thomas, Charles G Marguet, Kevin D Benson, Una J Lee, Eric R Sokol, Christopher E Wolter, Daniel M Katz, Christopher M Tarnay, Danielle Antosh, Michael H Heit, Christian Rehme, Mickey Karram, Scott Snyder, Emanuele Canestrari, Ron J Jankowski, Michael B Chancellor
Vanderbilt University Medical Center, Nashville, Tennessee, USA., Lerner College of Medicine Cleveland Clinic, Cleveland, Ohio, USA., University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, USA., Vanderbilt University Medical Center, Pittsburgh, Pennsylvania, USA., Atrium Health, Carolinas Medical Center, Charlotte, North Carolina, USA., Corewell Health System, Oakland University William Beaumont School of Medicine, Rochester, Minnesota, USA., University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma, USA., Mercy Health Saint Mary's Campus, Grand Rapids, Michigan, USA., Keck School of Medicine, University of Southern California, Los Angeles, California, USA., Prisma Health Regional Urology, Greenville, South Carolina, USA., Sanford Health/University of South Dakota School of Medicine, Sioux Falls, South Dakota, USA., Virginia Mason Franciscan Health, Seattle, Washington, USA., Stanford University School of Medicine, Stanford, California, USA., Mayo Clinic Arizona, Phoenix, Arizona, USA., Premier Medical Group of the Hudson Valley, Poughkeepsie, New York, USA., David Geffen School of Medicine at UCLA, Los Angeles, California, USA., Houston Methodist Hospital, Houston, Texas, USA., Emory University School of Medicine, Atlanta, Georgia, USA., Universitätsklinikum Essen, Essen, Germany., Christ Hospital, Cincinnati, Ohio, USA., Cook Research Incorporated, West Layfette, Indiana, USA., Cook MyoSite Incorporated, Pittsburgh, Pennsylvania, USA.