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.
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
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
21
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.
Placebo is the injection solution without muscle stem cells.
Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwig Hospital
Regensburg, Germany
Pediatric Urology, Department for Urology University of Ulm
Ulm, Germany
Prevalence of Intervention-related Adverse Events
Characterization of type, incidence, severity, and duration of adverse events
Time frame: Upto 12 months post-intervention
Change in Leak Point Pressure (LPP)
Change in LPP is calculated from baseline measurement
Time frame: Six months post-intervention
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