Addressing an unmet need: CELLEBRATE trial explores cell-based therapy for recurrent SUI

The phase 3 CELLEBRATE trial (NCT03104517) is exploring the safety and efficacy of autologous muscle-derived cells (iltamiocel) vs placebo for the treatment of adult women with post-surgical stress urinary incontinence (SUI). The design and methodology of the trial were shared during the 2025 American Urological Association (AUA) Annual Meeting in Las Vegas, Nevada.¹

Iltamiocel is “an investigational autologous cell therapy product comprised of muscle progenitor cells,” according to the authors of the study.

The investigational therapy received a Regenerative Medicine Advanced Therapy designation by the FDA for SUI based on the results of 2 completed, randomized-controlled trials (NCT01382602 and NCT01893138), the authors also noted. These studies showed that transurethral injection of iltamiocel significantly reduced SUI episodes, improved quality of life, and was safe.

The CELLEBRATE trial is aiming to provide additional evidence to support the safety and efficacy of this cell therapy in patients with recurrent or persistent SUI. In the following interview, Melissa R. Kaufman, MD, PhD, FACS, breaks down the design and potential implications of this study for clinical practice.

Melissa R. Kaufman, MD, PhD, FACS

Kaufman is the Patricia and Rodes Hart professor in urologic surgery, as well as a professor of urology and chief of the division of reconstructive urology and pelvic health at Vanderbilt University Medical Center in Nashville, Tennessee.

Urology Times®: Could you describe the background/rationale for the CELLEBRATE trial?

Kaufman: This is a study for female stress urinary incontinence, which is a very prevalent and impactful urologic condition. Women who experience recurrent or persistent incontinence despite having undergone a prior surgical intervention face a profound impact on their daily functioning and quality of life. There are procedures out there; they're often surgical or interventional for women who have recurrent or persistent incontinence, but they, of course, carry higher rates of complications. There is a pressing need for an effective, safe, minimally invasive treatment to address this unmet medical need.

In fact, this technology, which is called iltamiocel, was granted a Regenerative Medicine Advanced Therapy designation from the FDA based on some preliminary results from the trials that preceded the CELLEBRATE trial. This was granted because this is a serious problem and an unmet medical need.

Urology Times: How is this trial designed? What are the key end points?

Kaufman: The CELLEBRATE trial was initiated to provide additional evidence to support efficacy of iltamiocel and establish this innovative cell therapy as that option that's safe and minimally invasive for women with recurrent and persistent stress incontinence. We have enrollment of 96 participants with a 1:1 randomization at 17 sites. We just completed our final enrollment in January of 2025. We had a number of key inclusion and exclusion criteria that helped define that specific population of recurrent or persistent incontinence. The patients underwent outpatient muscle biopsy from their thigh, then these were grown at the main [manufacturing facility. Patients then received a single dose of iltamiocel or placebo.]

These patients are followed for 12 months. The primary end point is going to be a 75% reduction in stress incontinence episodes over a 3-day diary at 12 months. We're [also] looking at adverse events and lots of quality-of-life parameters.

Urology Times: What is the expected timeline for completion of the trial?

Kaufman: We hope to have all these primary results completed sometime near the end of 2026, probably after the AUA in 2026 we'll have the data ready to present. Then, we’ll hopefully be able to submit for a biologics license [application] with the FDA to get this iltamiocel product out to all the providers, all the urologists who treat female stress urinary incontinence.

Urology Times: What are the potential implications of this trial, if positive?

Kaufman: This is truly a transformational technology that has potentially a very broad application across a lot of different disease processes. These frontiers in cell medicine and cell-based therapies are being implemented. In fact, the company Cook MyoSite, that is sponsoring the CELLEBRATE trial, additionally has investigator-initiated trials and is sponsoring other trials for other processes, like fecal incontinence [and oropharyngeal] dysphagia. We have done early phase trials for male stress incontinence after prostatectomy. The applications for the technology may be very broad and transform the way that we treat patients and deliver therapies in the future.

REFERENCE

1. Kaufman M, Canestrari E, Nisha P, Jankowski R. CELLEBRATE: A double-blind, randomized, controlled study assessing the efficacy and safety of the iltamiocel cell therapy in the treatment of women with persistent or recurrent stress urinary incontinence following surgical treatment. Presented at: 2025 American Urological Association Annual Meeting. Las Vegas, Nevada. April 25-29, 2025. https://www.auajournals.org/doi/10.1097/01.JU.0001110436.86964.27