Iltamiocel Autologous Cell Therapy for the Treatment of Female Stress Urinary Incontinence: A Double-Blind, Randomized, Stratified, Placebo-Controlled Trial

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 KarramScott Snyder, Emanuele Canestrari, Ron J. Jankowski, Michael B. Chancellor

Research output: Contribution to journalArticlepeer-review

Abstract

Aims: This study aimed to determine the efficacy and safety of iltamiocel investigational autologous muscle cell therapy in females with stress urinary incontinence (SUI). Methods: 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. Results: 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. Conclusion: 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. Trial Registration: ClinicalTrials.gov identifier: NCT01893138; EudraCT number: 2014-002919-41.

Original languageEnglish (US)
Pages (from-to)2290-2299
Number of pages10
JournalNeurourology and Urodynamics
Volume43
Issue number8
DOIs
StatePublished - Nov 2024

Keywords

  • autologous myoblasts
  • cell therapy
  • regenerative medicine
  • skeletal muscle cells
  • stress urinary incontinence
  • Double-Blind Method
  • Humans
  • Middle Aged
  • Urinary Incontinence, Stress/therapy
  • Treatment Outcome
  • Transplantation, Autologous
  • Cell- and Tissue-Based Therapy
  • Female
  • Adult
  • Aged

ASJC Scopus subject areas

  • Clinical Neurology
  • Urology

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