Epispadias, Male, Urinary Incontinence
Conditions
Keywords
Isolated epispadias, Urinary incontinence, Muscle stem cell therapy, Autologous cell therapy
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.
Interventions
BIOLOGICALPrimary 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.
OTHERPlacebo
Placebo is the injection solution without muscle stem cells.
Sponsors
German Federal Ministry of Education and Research
Simone Spuler, MD
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention model description
This is a randomized placebo-controlled clinical trial
Eligibility
Sex/Gender
MALE
Age
3 Years to 17 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Prevalence of Intervention-related Adverse Events | Upto 12 months post-intervention | Characterization of type, incidence, severity, and duration of adverse events |
| Change in Leak Point Pressure (LPP) | Six months post-intervention | Change in LPP is calculated from baseline measurement |
Countries
Germany
Contacts
Primary ContactSimone Spuler, Prof. Dr.
Outcome results
None listed