Autologous Muscle Stem Cell Therapy for Treatment of Congenital Urinary Incontinence in Epispadias Patients (MUST)

• ClinicalTrials.gov Identifier: NCT04729582
• Recruitment Status: Not yet recruiting
• First Posted: January 28, 2021
• Last Update Posted: December 7, 2022
• Sponsor: Simone Spuler, MD
• Collaborator: German Federal Ministry of Education and Research
• Information provided by (Responsible Party): Simone Spuler, MD, Charite University, Berlin, Germany

Tracking Information

• First Submitted Date: January 21, 2021
• First Posted Date: January 28, 2021
• Last Update Posted Date: December 7, 2022
• Estimated Study Start Date: May 2023
• Estimated Primary Completion Date: August 2026 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 27, 2021)

• Prevalence of Intervention-related Adverse Events [ Time Frame: Upto 12 months post-intervention ]
  Characterization of type, incidence, severity, and duration of adverse events
• Change in Leak Point Pressure (LPP) [ Time Frame: Six months post-intervention ]
  Change in LPP is calculated from baseline measurement

• 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 Stem Cell Therapy for Treatment of Congenital Urinary Incontinence in Epispadias Patients
• Official Title: Combined Phase 1 and 1/2a Clinical Trial Evaluating the Safety and Efficacy of an Autologous Muscle Stem Cell Therapy in the Treatment of Urinary Incontinence in Isolated Epispadias
• Brief Summary: The aim of this trial is to evaluate the safety and efficacy of an autologous muscle stem cell therapy in the treatment of congenital urinary incontinence in isolated epispadias.

Detailed Description

Epispadias is the mildest form of exstrophy-epispadias complex (EEC); a congenital malformation disorder involving the midline abdominal and genitourinary structures. Patients with epispadias have a defect in the urethral sphincter resulting in urinary incontinence. This trial investigates the injection of autologous primary human muscle stem cells into the urethral sphincter with the aim of repairing the defect and restore the anatomic ability for continence.

Eligible participants will undergo muscle biopsy during a routine cystoscopy. Acquired muscle tissue is used for the isolation and expansion of muscle stem cells ex-vivo. Muscle stem cells are injected into the urethral sphincter under visual control using cystoscopy. Participants are assessed for safety and efficacy for a minimum of 12 months post-intervention.

• Study Type: Interventional
• Study Phase: Phase 1; Phase 2

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

This is a randomized placebo-controlled clinical trial

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment

Condition

• Urinary Incontinence
• Epispadias, Male

Intervention

• Biological: Primary human muscle stem cells (Satori-01)
  Primary human muscle stem cells are isolated from patient's muscle tissue and expanded ex-vivo. They are injected into the urethral sphincter region as a one-time autologous treatment.
• Other: Placebo
  Placebo is the injection solution without muscle stem cells.

Study Arms

• Experimental: Verum Group
  Participants receive primary human muscle stem cells as one-time injection into the urethral sphincter region under visual control using cystoscopy.
  Intervention: Biological: Primary human muscle stem cells (Satori-01)
• Placebo Comparator: Placebo group
  Participants receive placebo solution as one-time injection into the external urethral sphincter region under visual control using cystoscopy.
  Intervention: Other: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Not yet recruiting
• Estimated Enrollment (submitted: January 27, 2021): 21
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: October 2026
• Estimated Primary Completion Date: August 2026 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Male
• Isolated epispadias
• Aged ≥ 3 years
• Urinary incontinence as defined according to the International Children´s Continence Society
• Informed consent

Exclusion Criteria:

• Acute or chronic inflammatory local or systemic disease
• Coagulation Disorder
• Previous adverse reaction to anesthesia
• Congenital heart defect, cardiac arrhythmia

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: 3 Years to 17 Years (Child)
• Accepts Healthy Volunteers: No

Contacts

Contact: Simone Spuler, Prof. Dr.; +49 (0) 30 450 540 501; simone.spuler@charite.de

• Listed Location Countries: Germany

Administrative Information

• NCT Number: NCT04729582
• Other Study ID Numbers: MUST
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

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

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Simone Spuler, MD, Charite University, Berlin, Germany
• Original Responsible Party: Simone Spuler, Charite University, Berlin, Germany, Principal investigator
• Current Study Sponsor: Simone Spuler, MD
• Original Study Sponsor: Simone Spuler
• Collaborators: German Federal Ministry of Education and Research

Investigators

• Principal Investigator: Anne K Ebert, Prof. Dr.; Pediatric Urology, Department for Urology University of Ulm, Germany

• PRS Account: Charite University, Berlin, Germany
• Verification Date: December 2022