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

NCT04729582 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: MUST
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 2

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.

Conditions

Urinary Incontinence; Epispadias, Male

Keywords

Isolated epispadias; Urinary incontinence; Muscle stem cell therapy; Autologous cell therapy

Outcome Measures

Primary Outcomes (2)

• 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

Study Arms (2)

Verum Group

EXPERIMENTAL

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)

Placebo group

PLACEBO COMPARATOR

Participants receive placebo solution as one-time injection into the external urethral sphincter region under visual control using cystoscopy.

Other: Placebo

Interventions

Primary human muscle stem cells (Satori-01) BIOLOGICAL

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.

Verum Group

Placebo OTHER

Placebo is the injection solution without muscle stem cells.

Placebo group

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male
• Isolated epispadias
• Aged ≥ 3 years
• Urinary incontinence as defined according to the International Children´s Continence Society
• Informed consent

You may not qualify if:

• Acute or chronic inflammatory local or systemic disease
• Coagulation Disorder
• Previous adverse reaction to anesthesia
• Congenital heart defect, cardiac arrhythmia

Sponsors & Collaborators

• Simone Spuler, MD: lead
• German Federal Ministry of Education and Research: collaborator

Study Sites (2)

Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwig Hospital

Regensburg, 93049, Germany

Location

Pediatric Urology, Department for Urology University of Ulm

Ulm, 89075, Germany

Location

MeSH Terms

Conditions

Urinary Incontinence; Epispadias

Condition Hierarchy (Ancestors)

Urination Disorders; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Lower Urinary Tract Symptoms; Urological Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Urogenital Abnormalities; Urethral Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Anne K Ebert, Prof. Dr.

  Pediatric Urology, Department for Urology University of Ulm, Germany

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Simone Spuler, Prof. Dr.

CONTACT

+49 (0) 30 450 540 501; simone.spuler@charite.de

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal investigator

Model Details: This is a randomized placebo-controlled clinical trial

Study Record Dates

• First Submitted: January 21, 2021
• First Posted: January 28, 2021
• Study Start: June 1, 2024
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: August 3, 2023

Record last verified: 2023-08

Locations

DE (2)