iltamiocel (AMDC-USR) / Cook MyoSite - LARVOL DELTA

DigniFI: Iltamiocel Compared to Placebo for Chronic Fecal Incontinence in Females With Obstetric Injury (clinicaltrials.gov) - P3 | N=200 | Active, not recruiting | Sponsor: Cook MyoSite | Recruiting ➔ Active, not recruiting

Enrollment closed • Obstetrics

DigniFI Trial Update: A Phase 3, Two-stage, RCT Assessing the Efficacy and Safety of Iltamiocel in the Treatment of ChronicFfecal Incontinence Sponsored by Cook MyoSite (ASCRS 2025) - No abstract available

Clinical • P3 data

CELLEBRATE: Autologous Muscle Derived Cells Compared to Placebo for Urinary Sphincter Repair in Post-surgical Female Stress Incontinence (clinicaltrials.gov) - P3 | N=96 | Active, not recruiting | Sponsor: Cook MyoSite | Trial completion date: Feb 2026 ➔ Sep 2027 | Trial primary completion date: Feb 2025 ➔ Sep 2026

Trial completion date • Trial primary completion date • Urinary Incontinence • Urology

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 (AUA 2025) - P3 | "After the 12-month follow-up visit, participants originally randomized to placebo could elect to receive open-label iltamiocel injection.The primary efficacy endpoint is the percentage of participants achieving ≥75% reduction in SIE recorded in a 3-day diary at 12 months compared to baseline. Secondary efficacy endpoints include the percentage of participants achieving restored continence (0-1 SIE), and clinically meaningful improvement of QoL (≥10-point improvement in I-QOL participant-reported questionnaire, and ≥75% SIE reduction) at 12 months.The study is expected to have comprehensive primary results available in Q3 2026."

Clinical • CNS Disorders • Urinary Incontinence • Urology

CLBT RGST: Observational Registry for Iltamiocel Compared to Placebo for Urinary Sphincter Repair in Post-surgical Female Stress Incontinence (clinicaltrials.gov) - P=N/A | N=96 | Enrolling by invitation | Sponsor: Cook MyoSite | Not yet recruiting ➔ Enrolling by invitation

Enrollment open • Urinary Incontinence • Urology

Fecal incontinence stem cell trial opens at UCI Health (UCI Health) - "UCI Health has launched a clinical trial for a novel stem cell-derived therapy that shows promise for restoring anal sphincter function in patients with obstetric injury-related bowel incontinence, said colorectal surgeon Dr. Joseph C. Carmichael, principal investigator of the study....The phase 3 trial, designed to test the effectiveness and safety of iltamiocel, a cellular treatment made from a patient’s own muscle cells, is being conducted under the auspices of the UCI Alpha Clinic, the clinical trial arm of the UC Irvine Sue & Bill Gross Stem Cell Research Center."

New P3 trial • Urinary Incontinence

CELLEBRATE: Autologous Muscle Derived Cells Compared to Placebo for Urinary Sphincter Repair in Post-surgical Female Stress Incontinence (clinicaltrials.gov) - P3 | N=96 | Active, not recruiting | Sponsor: Cook MyoSite | Recruiting ➔ Active, not recruiting

Enrollment closed • Urinary Incontinence • Urology

CLBT RGST: Observational Registry for Iltamiocel Compared to Placebo for Urinary Sphincter Repair in Post-surgical Female Stress Incontinence (clinicaltrials.gov) - P=N/A | N=96 | Not yet recruiting | Sponsor: Cook MyoSite

New trial • Urinary Incontinence • Urology

Iltamiocel Autologous Cell Therapy for the Treatment of Female Stress Urinary Incontinence: A Double-Blind, Randomized, Stratified, Placebo-Controlled Trial. (PubMed, Neurourol Urodyn) - P3 | "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."

Journal • Urinary Incontinence • Urology

Japanese Bridging Study of Autologous Muscle Derived Cells Compared to Placebo for Female Urinary Sphincter Repair(JPN1) (clinicaltrials.gov) - P3 | N=0 | Withdrawn | Sponsor: Cook MyoSite | N=24 ➔ 0 | Trial completion date: Jun 2026 ➔ Jun 2027 | Not yet recruiting ➔ Withdrawn | Trial primary completion date: Jun 2025 ➔ Jun 2026

Enrollment change • Trial completion date • Trial primary completion date • Trial withdrawal • Urinary Incontinence • Urology

CELLEBRATE: Autologous Muscle Derived Cells Compared to Placebo for Urinary Sphincter Repair in Post-surgical Female Stress Incontinence (clinicaltrials.gov) - P3 | N=96 | Recruiting | Sponsor: Cook MyoSite | N=260 ➔ 96

Enrollment change • Urinary Incontinence • Urology

CELLEBRATE: Adaptive Design Study of Autologous Muscle Derived Cells Compared to Placebo for Female Urinary Sphincter Repair for Persistent or Recurrent Stress Urinary Incontinence Following Surgical Treatment (clinicaltrials.gov) - P3 | N=260 | Recruiting | Sponsor: Cook MyoSite

Trial completion date • Trial primary completion date • Urinary Incontinence • Urology

DigniFI: Iltamiocel Compared to Placebo for Chronic Fecal Incontinence in Females With Obstetric Injury (clinicaltrials.gov) - P3 | N=200 | Recruiting | Sponsor: Cook MyoSite | Not yet recruiting ➔ Recruiting | Initiation date: Sep 2023 ➔ Dec 2023

Enrollment open • Trial initiation date • Obstetrics

Japanese Bridging Study of Autologous Muscle Derived Cells Compared to Placebo for Female Urinary Sphincter Repair(JPN1) (clinicaltrials.gov) - P3 | N=24 | Not yet recruiting | Sponsor: Cook MyoSite | Trial completion date: Jun 2025 ➔ Jun 2026 | Trial primary completion date: Jun 2024 ➔ Jun 2025

Trial completion date • Trial primary completion date • Urinary Incontinence • Urology

Safety and Efficacy of Iltamiocel Cellular Therapy for the Treatment of Fecal Incontinence. Results of a Phase 1/2 Study. (PubMed, Ann Surg) - "The administration of iltamiocel cellular therapy is safe. Iltamiocel shows promise for significantly improving fecal incontinence symptoms and quality of life."

Journal • P1/2 data • Inflammation

DigniFI: Iltamiocel Compared to Placebo for Chronic Fecal Incontinence in Females With Obstetric Injury (clinicaltrials.gov) - P3 | N=200 | Not yet recruiting | Sponsor: Cook MyoSite

New P3 trial • Obstetrics

[VIRTUAL] An Evaluation of Women with Persistent or Recurrent Stress Urinary Incontinence (SUI) Following Surgery in a Double-blind, Randomized, Controlled Trial Comparing Safety and Efficacy of Autologous Muscle Derived Cells for Urinary Sphincter Repair (AMDC-USR) with Placebo (PBO) (AUA 2021) - P3 | "Women with chronic SUI symptoms (average > 12 years) sought treatment despite undergoing as many as five prior surgeries at the time of enrollment. A single injection of AMDC-USR may be a safe and effective treatment in this challenging refractory population. Based in part on this evidence to address unmet medical need and the recognized seriousness of the condition, AMDC-USR has been granted the expedited Regenerative Medicine Advanced Therapy (RMAT) designation by the United States Food and Drug Administration for this population."

Clinical • Urinary Incontinence • Urology

[VIRTUAL] 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) (AUA 2021) - P3 | "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."

Clinical • Urinary Incontinence • Urology

CELLEBRATE: Adaptive Design Study of Autologous Muscle Derived Cells Compared to Placebo for Female Urinary Sphincter Repair for Persistent or Recurrent Stress Urinary Incontinence Following Surgical Treatment (clinicaltrials.gov) - P3 | N=260 | Recruiting | Sponsor: Cook MyoSite | Trial completion date: Sep 2023 ➔ Feb 2026 | Trial primary completion date: Sep 2022 ➔ Jan 2025

Trial completion date • Trial primary completion date • Urinary Incontinence • Urology

Japanese Bridging Study of Autologous Muscle Derived Cells Compared to Placebo for Female Urinary Sphincter Repair(JPN1) (clinicaltrials.gov) - P3 | N=24 | Not yet recruiting | Sponsor: Cook MyoSite | Trial completion date: Dec 2024 ➔ Jun 2025 | Trial primary completion date: Dec 2023 ➔ Jun 2024

Trial completion date • Trial primary completion date • Urinary Incontinence • Urology