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

Phase 1

Not yet recruiting

• Conditions: Urinary Incontinence; Epispadias, Male
• Interventions: Biological: Primary human muscle stem cells (Satori-01); Other: Placebo

• Registration Number: NCT04729582
• Lead Sponsor: Simone Spuler, MD

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 Timeline

• Study First Submitted: 2021-01-21
• Study First Submitted QC: 2021-01-27
• Study First Posted: 2021-01-28
• Status Verified: 2023-08
• Last Update Submitted: 2023-08-02
• Last Update Posted: 2023-08-03
• Study Start: 2024-06
• Primary Completion: 2026-08
• Study Completion: 2026-10

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: Male
• Target Recruitment: 21

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (2)

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