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

Urinary Incontinence Clinical Trial

— MUST  

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04729582
• Other study ID #: MUST
• Status: Not yet recruiting
• Phase: Phase 1/Phase 2
• Start date: June 2024
• Est. completion date: October 2026

Study information

• Verified date: August 2023
• Source: Charite University, Berlin, Germany
• Contact: Simone Spuler, Prof. Dr.
• Phone: +49 (0) 30 450 540 501
• Email: simone.spuler[@]charite.de
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 21
• Est. completion date: October 2026
• Est. primary completion date: August 2026
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 3 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Enuresis
• Epispadias, Male
• Urinary Incontinence

Intervention

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.

Locations

Country	Name	City	State
Germany	Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwig Hospital	Regensburg
Germany	Pediatric Urology, Department for Urology University of Ulm	Ulm

Sponsors (2)

Lead Sponsor	Collaborator
Simone Spuler, MD	German Federal Ministry of Education and Research

Country where clinical trial is conducted

Germany, 

Outcome

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