Autologous Muscle Derived Cells for Female Urinary Sphincter Repair

• ClinicalTrials.gov Identifier: NCT01893138
• Recruitment Status: Completed
• First Posted: July 8, 2013
• Results First Posted: January 5, 2023
• Last Update Posted: January 5, 2023
• Sponsor: Cook MyoSite
• Information provided by (Responsible Party): Cook MyoSite

Tracking Information

• First Submitted Date: July 2, 2013
• First Posted Date: July 8, 2013
• Results First Submitted Date: September 12, 2022
• Results First Posted Date: January 5, 2023
• Last Update Posted Date: January 5, 2023
• Actual Study Start Date: November 21, 2013
• Actual Primary Completion Date: November 1, 2019 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: December 12, 2022): Participants With ≥ 50% Reduction in Stress Incontinence Episode Frequency From Baseline to 12 Months Post-treatment; as Assessed by 3 Day Diary [ Time Frame: Baseline and 12 months ]
• Original Primary Outcome Measures (submitted: July 5, 2013): Number of leaks due to stress incontinence episodes, as recorded in a diary [ Time Frame: 12 months ]

Current Secondary Outcome Measures (submitted: December 12, 2022)

• Participants With at ≥ 75% Reduction in Stress Incontinence Episodes From Baseline at 12 Months [ Time Frame: Baseline and 12 months ]
• Participants With 0 or 1 Stress Incontinence Episodes Based on 3 Day Diary Records at 12 Months [ Time Frame: Baseline and 12 months ]
• Change in the Frequency of Stress Incontinence Episodes From Baseline at 12 Months [ Time Frame: Baseline and 12 months ]

• Original Secondary Outcome Measures (submitted: July 5, 2013): Stress leak frequency and pad weight composite endpoint [ Time Frame: 12 months ]

Current Other Pre-specified Outcome Measures (submitted: December 12, 2022)

• Association of Change in Quality of Life Scores With Change in Stress Incontinence Episode Frequency [ Time Frame: Baseline and 12 months ]
  Spearman's correlation used for analysis
• Treatment Durability at 24 Months [ Time Frame: Baseline, 12 months, and 24 months after injection with iltamiocel ]
  Treatment durability defined as iltamiocel-treated participants with reduction in stress incontinence episode frequency (SIEF) at 12 months who maintained that response at 24 months

• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Autologous Muscle Derived Cells for Female Urinary Sphincter Repair
• Official Title: A Double-blind, Randomized, Controlled Trial Comparing the Safety and Efficacy of AMDC-USR With Placebo in Female Subjects With Stress Urinary Incontinence
• Brief Summary: This randomized, double-blind, placebo-controlled, multicenter, confirmatory study will evaluate the efficacy and safety of Cook MyoSite Incorporated Autologous Muscle-Derived Cells (generic name Iltamiocel) compared to a placebo (vehicle) control dose in the treatment of stress urinary incontinence (SUI) in adult female patients.
• Detailed Description: Study comparing intrasphincteric injection of iltamiocel with placebo. Subjects unblinded after 12 month visits, but followed for up to 2 years. Subjects randomized to placebo could elect to receive open-label iltamiocel after completing 12 month visit.
• 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

• Other: Placebo
  Placebo control is the vehicle solution used for the study product.
• Biological: Iltamiocel
  AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intraurethral injection of 150 x 10^6 cells.
  Other Name: Autologous muscle-derived cells (AMDC)

Study Arms

• Placebo Comparator: Placebo
  Placebo control is the vehicle solution used for the study product.
  Intervention: Other: Placebo
• Experimental: Iltamiocel
  AMDC is the study product (autologous muscle-derived cells). The generic name is iltamiocel. Single intraurethral injection of 150 x 10^6 cells.
  Intervention: Biological: Iltamiocel

• Publications *: Antosh DD, High R, Brown HW, Oliphant SS, Abed H, Philip N, Grimes CL. Feasibility of prophylactic salpingectomy during vaginal hysterectomy. Am J Obstet Gynecol. 2017 Nov;217(5):605.e1-605.e5. doi: 10.1016/j.ajog.2017.07.017. Epub 2017 Jul 20.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 23, 2018): 311
• Original Estimated Enrollment (submitted: July 5, 2013): 267
• Actual Study Completion Date: November 10, 2020
• Actual Primary Completion Date: November 1, 2019 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Female patient has primary symptoms of SUI, as confirmed by patient medical history and clinical symptoms, including a focused incontinence evaluation.

Exclusion Criteria:

• Patient has symptoms of pure 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, etc.)
• Patient has more than 2 episode of awakening to void during normal sleeping hours.
• Patient cannot be maintained on a stable dose and/or frequency of medication (including diuretics) known to affect lower urinary tract function, including but not limited to, anticholinergics, tricyclic antidepressants or alpha-adrenergic blockers, for at least 2 weeks prior to screening or is likely to change during the course of the study.
• Patient is pregnant, lactating, or plans to become pregnant during the course of the study.
• Patient refuses to provide written informed consent.
• Patient is not at least 18 years of age.
• Patient is not available for the follow-up evaluations as required by the protocol.

Sex/Gender

• Sexes Eligible for Study: Female

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Belgium, Germany, United States
• Removed Location Countries: Netherlands

Administrative Information

• NCT Number: NCT01893138
• Other Study ID Numbers: 13-003
• Has Data Monitoring Committee: Not Provided

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: Cook Group Incorporated
• Current Study Sponsor: Cook MyoSite
• Original Study Sponsor: Cook Group Incorporated
• 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: December 2022