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NCT Number: NCT04729582

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

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.

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Key information

Conditions

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

Age range

3 year–17 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

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

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Placebo

Other

Placebo is the injection solution without muscle stem cells.

Primary outcomes

  1. Prevalence of Intervention-related Adverse Events

    Time frame: Upto 12 months post-intervention

    Characterization of type, incidence, severity, and duration of adverse events

  2. Change in Leak Point Pressure (LPP)

    Time frame: Six months post-intervention

    Change in LPP is calculated from baseline measurement

Study contacts

Contact information is provided by the study sponsor or research team.

Simone Spuler, Prof. Dr.

CONTACT

[email protected]

+49 (0) 30 450 540 501

Sponsors and collaborators

Lead sponsor

Simone Spuler, MD

Other

Collaborators

  • German Federal Ministry of Education and Research

Registry information

Official study 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

Acronym: MUST

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Jan 28, 2021
Registry last updated
Aug 3, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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