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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Urinary Incontinence, Epispadias, Male

Intervention / Treatment
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* Biological: Primary human muscle stem cells (Satori-01)
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: This is a randomized placebo-controlled clinical trial
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Verum Group<br>Participants receive primary human muscle stem cells as one-time injection into the urethral sphincter region under visual control using cystoscopy. | Biological: Primary human muscle stem cells (Satori-01)<br>* 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.<br>|
| Placebo Comparator: Placebo group<br>Participants receive placebo solution as one-time injection into the external urethral sphincter region under visual control using cystoscopy. | Other: Placebo<br>* Placebo is the injection solution without muscle stem cells.<br>|

What is the study measuring?
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Primary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Isolated epispadias, Urinary incontinence, Muscle stem cell therapy, Autologous cell therapy