TY - JOUR
T1 - Iltamiocel Autologous Cell Therapy for the Treatment of Female Stress Urinary Incontinence
T2 - A Double-Blind, Randomized, Stratified, Placebo-Controlled Trial
AU - Kaufman, Melissa R.
AU - Goldman, Howard B.
AU - Chermansky, Christopher J.
AU - Dmochowski, Roger
AU - Kennelly, Michael J.
AU - Peters, Kenneth M.
AU - Quiroz, Lieschen H.
AU - Bennett, Jason B.
AU - Thomas, Sherry
AU - Marguet, Charles G.
AU - Benson, Kevin D.
AU - Lee, Una J.
AU - Sokol, Eric R.
AU - Wolter, Christopher E.
AU - Katz, Daniel M.
AU - Tarnay, Christopher M.
AU - Antosh, Danielle
AU - Heit, Michael H.
AU - Rehme, Christian
AU - Karram, Mickey
AU - Snyder, Scott
AU - Canestrari, Emanuele
AU - Jankowski, Ron J.
AU - Chancellor, Michael B.
N1 - Publisher Copyright:
© 2024 The Author(s). Neurourology and Urodynamics published by Wiley Periodicals LLC.
PY - 2024/11
Y1 - 2024/11
N2 - 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.
AB - 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.
KW - autologous myoblasts
KW - cell therapy
KW - regenerative medicine
KW - skeletal muscle cells
KW - stress urinary incontinence
KW - Double-Blind Method
KW - Humans
KW - Middle Aged
KW - Urinary Incontinence, Stress/therapy
KW - Treatment Outcome
KW - Transplantation, Autologous
KW - Cell- and Tissue-Based Therapy
KW - Female
KW - Adult
KW - Aged
UR - http://www.scopus.com/inward/record.url?scp=85204100338&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85204100338&partnerID=8YFLogxK
U2 - 10.1002/nau.25588
DO - 10.1002/nau.25588
M3 - Article
C2 - 39282854
AN - SCOPUS:85204100338
SN - 0733-2467
VL - 43
SP - 2290
EP - 2299
JO - Neurourology and Urodynamics
JF - Neurourology and Urodynamics
IS - 8
ER -