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

Sponsor

Cook MyoSite (Industry)

Overall Status

Recruiting

CT.gov ID

NCT03104517

Collaborator

(none)

260

Enrollment

Locations

Arms

81.3

Anticipated Duration (Months)

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Stress Urinary Incontinence	Biological: AMDC-USR (iltamiocel) Other: Placebo	Phase 3

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 Design

Study Type:

Interventional

Anticipated Enrollment :

260 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

CELLEBRATE: An Adaptive, Two-Stage, 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

Actual Study Start Date :

Apr 23, 2019

Anticipated Primary Completion Date :

Jan 1, 2025

Anticipated Study Completion Date :

Feb 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: AMDC-USR (iltamiocel) Autologous muscle-derived cells for urinary sphincter repair (AMDC-USR; generic name: iltamiocel) is the study product.	Biological: AMDC-USR (iltamiocel) Autologous Muscle Derived Cells for Urinary Sphincter Repair (generic name: iltamiocel)
Placebo Comparator: Placebo Placebo control is the vehicle solution used for the study product.	Other: Placebo Placebo control is the vehicle solution used for the study product.

Arm

Intervention/Treatment

Experimental: AMDC-USR (iltamiocel)

Autologous muscle-derived cells for urinary sphincter repair (AMDC-USR; generic name: iltamiocel) is the study product.

Biological: AMDC-USR (iltamiocel)

Autologous Muscle Derived Cells for Urinary Sphincter Repair (generic name: iltamiocel)

Placebo Comparator: Placebo

Placebo control is the vehicle solution used for the study product.

Other: Placebo

Placebo control is the vehicle solution used for the study product.

Outcome Measures

Primary Outcome Measures

Number of leaks due to stress incontinence episodes, as recorded in a diary [12 months]
Stress leak frequency

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

Female

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UCLA Women's Health Clinical Research Unit/Department of OBGYN	Los Angeles	California	United States	90095
2	University of California, Davis	Sacramento	California	United States	95817
3	Stanford Hospital and Clinics	Stanford	California	United States	94305
4	University of Colorado Hospital	Aurora	Colorado	United States	80045
5	MedStar Georgetown Hospital Department of Urology	Washington	District of Columbia	United States	20007
6	Loyola University Medical Center	Maywood	Illinois	United States	60153
7	University of Kansas Health System	Kansas City	Kansas	United States	66160
8	Louisiana State University Health Sciences Center, Shreveport	Shreveport	Louisiana	United States	71103
9	Beaumont Hospital, Royal Oak	Royal Oak	Michigan	United States	48073
10	University of New Mexico Women's Care Clinic	Albuquerque	New Mexico	United States	87131
11	McKay Urology	Charlotte	North Carolina	United States	20207
12	Cleveland Clinic/Glickman Institute-Q10	Cleveland	Ohio	United States	44195
13	University of Oklahoma Physicians Building	Oklahoma City	Oklahoma	United States	73104
14	The Institute for Female Pelvic Medicine and Reconstructive Surgery	Allentown	Pennsylvania	United States	18103
15	Magee Women's Hospital of UPMC	Pittsburgh	Pennsylvania	United States	15213
16	Prisma Health, Upstate	Greenville	South Carolina	United States	29605
17	Sanford Female Pelvic Medicine and Reconstructive Surgery Clinic	Sioux Falls	South Dakota	United States	57105
18	Vanderbilt University Medical Center, Dept. of Urologic Surgery	Nashville	Tennessee	United States	37232
19	Urology Clinics of North Texas, PLLC	Dallas	Texas	United States	75231
20	UT Southwestern Medical Center	Dallas	Texas	United States	75390
21	Baylor Scott and White Medical Center, Temple	Temple	Texas	United States	76508
22	Urology of Virginia, PLLC	Virginia Beach	Virginia	United States	23462
23	Virginia Mason Medical Center	Seattle	Washington	United States	98101
24	Aurora Medical Center, West Allis	Milwaukee	Wisconsin	United States	53227
25	Derriford Hospital	Plymouth	Devon	United Kingdom	PL6 8DH
26	St. James's University Hospital	Leeds	Yorkshire	United Kingdom	LS9 7TF