Autologous Muscle Derived Cells Compared to Placebo for Urinary Sphincter Repair in Post-surgical Female Stress Incontinence (CELLEBRATE)

• ClinicalTrials.gov Identifier: NCT03104517
• Recruitment Status: Recruiting
• First Posted: April 7, 2017
• Last Update Posted: May 7, 2024
• Sponsor: Cook MyoSite
• Information provided by (Responsible Party): Cook MyoSite

Tracking Information

• First Submitted Date: April 3, 2017
• First Posted Date: April 7, 2017
• Last Update Posted Date: May 7, 2024
• Actual Study Start Date: April 23, 2019
• Estimated Primary Completion Date: January 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 3, 2017)

Number of leaks due to stress incontinence episodes, as recorded in a diary [ Time Frame: 12 months ]

Stress leak frequency

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Autologous Muscle Derived Cells Compared to Placebo for Urinary Sphincter Repair in Post-surgical Female Stress Incontinence
• Official Title: CELLEBRATE: A Double-Blind, Randomized, Controlled Trial Comparing the Safety and Efficacy of AMDC-USR With Placebo in Female Subjects With Persistent or Recurrent Stress Urinary Incontinence Following Surgical Treatment
• Brief Summary: This study evaluates the efficacy and safety of Autologous Muscle Derived Cells for Urinary Sphincter Repair (AMDC-USR; generic name: iltamiocel) compared to a placebo in the reduction of stress incontinence episode frequency in adult female patients with post-surgical persistent or recurrent stress urinary incontinence (SUI). Half of the participants will receive AMDC-USR (injections with cells) and the other half will receive placebo.

Detailed Description

Stress urinary incontinence (SUI) is the accidental loss of urine due to physical activity, such as laughing, coughing, or sneezing. Autologous Muscle Derived Cells for Urinary Sphincter Repair (AMDC-USR; generic name: iltamiocel) involves a medical procedure in which a participant's own muscle cells are collected, processed, and then injected into the tissues of the urinary passage.

This is a double-blind randomized study, which means neither the participant, nor the study doctor will know which treatment group the participant will be in. Participants who are randomly chosen to receive injection with placebo will have the option to receive an injection with their cells after completion of the blinded portion of their study participation (12 months).

• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Stress Urinary Incontinence

Intervention

• Biological: AMDC-USR (iltamiocel)
  Autologous Muscle Derived Cells for Urinary Sphincter Repair (generic name: iltamiocel)
• Other: Placebo
  Placebo control is the vehicle solution used for the study product.

Study Arms

• Experimental: AMDC-USR (iltamiocel)
  Autologous muscle-derived cells for urinary sphincter repair (AMDC-USR; generic name: iltamiocel) is the study product.
  Intervention: Biological: AMDC-USR (iltamiocel)
• Placebo Comparator: Placebo
  Placebo control is the vehicle solution used for the study product.
  Intervention: Other: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: February 8, 2024): 96
• Original Estimated Enrollment (submitted: April 3, 2017): 156
• Estimated Study Completion Date: February 2026
• Estimated Primary Completion Date: January 2025 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Adult female patient ≥ 18 years of age who has primary and moderate-to-severe symptoms of SUI for at least 6 months, as confirmed by patient medical history and clinical symptoms, including a focused incontinence evaluation.
• History of previous surgery for treatment of SUI. Previous surgery could include midurethral sling, retropubic suspension, or bladder neck sling. Bulking agents alone are not considered previous surgery for treatment of SUI.
• Must be willing and able to comply with the study procedures, be mentally competent and able to understand all study requirements, and must agree to read and sign the informed consent form prior to any study-related procedures.
• Must have completed 100% of the screening 3-day diary evening reports.

Exclusion Criteria:

• Patient has symptoms of only urge incontinence as confirmed by basic evaluation of etiology from a patient medical history, including a focused incontinence history.
• Patient has symptoms of mixed urinary incontinence where urge incontinence is the predominant factor.
• Patient has had stress urinary incontinence symptoms less than 6 months prior to signing the informed consent.
• Patient has not previously attempted conservative treatment prior to signing the informed consent. (Examples of conservative treatment include behavior modifications, bladder exercises, biofeedback, pelvic floor muscle therapy, etc.)
• Patient BMI ≥ 35.
• Patient routinely has more than 2 episodes of awakening to void during normal sleeping hours.
• If taking a medication known to affect lower urinary tract function, including but not limited to, anticholinergics, beta 3 adrenergic receptor agonists, tricyclic antidepressants, serotonin-norepinephrine reuptake inhibitor (SNRI) or selective serotonin reuptake inhibitor (SSRI) antidepressants, diuretics, or alpha-adrenergic blockers, patient cannot be maintained on a stable dose and/or frequency of medication (including diuretics), cannot be maintained on a stable dose and/or frequency for at least 2 weeks prior to screening or is likely to change during the course of the study.
• History of cancer in pelvic organs, ureters, or kidneys.
• Patient is pregnant, lactating, or plans to become pregnant during the course of the study.

Sex/Gender

• Sexes Eligible for Study: Female

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Maria Jose Godino; +54 911 3913 0952; MariaJose.Godino@IQVIA.com

• Listed Location Countries: United Kingdom, United States

Administrative Information

• NCT Number: NCT03104517
• Other Study ID Numbers: 15-06
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Cook MyoSite
• Original Responsible Party: Same as current
• Current Study Sponsor: Cook MyoSite
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Melissa Kaufman, M.D., Ph.D.; Vanderbilt University Medical Center, Department of Urologic Surgery

• PRS Account: Cook MyoSite
• Verification Date: May 2024