Autologous Muscle Stem Cell Therapy for Treatment of Congenital Urinary Incontinence in Epispadias Patients
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Primary human muscle stem cells (Satori-01), Placebo.
- Who it may be relevant to
- Registry conditions: Urinary Incontinence, Epispadias, Male. Basic parameters: 3 years — 17 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Germany
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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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
Overview
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
- 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.
Primary outcome measures
- Prevalence of Intervention-related Adverse Events [Time frame: Upto 12 months post-intervention]
- Change in Leak Point Pressure (LPP) [Time frame: Six months post-intervention]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
Germany · 2 centers
- Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwi — Regensburg
- Pediatric Urology, Department for Urology University of Ulm — Ulm
Identifiers
NCT: NCT04729582 · MUST