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

August 2, 2023 updated by: Simone Spuler, MD

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

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

21

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Germany
      • Regensburg, Germany, 93049
        • Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwig Hospital
        • Contact:
        • Sub-Investigator:
          • Wolfgang H Rösch, Prof. Dr.
      • Ulm, Germany, 89075
        • Pediatric Urology, Department for Urology University of Ulm
        • Contact:
        • Principal Investigator:
          • Anne K Ebert, Prof. Dr.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 17 years (Child)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Verum Group
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)
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 Comparator: Placebo group
Participants receive placebo solution as one-time injection into the external urethral sphincter region under visual control using cystoscopy.
Other: Placebo
Placebo is the injection solution without muscle stem cells.

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Simone Spuler, MD

Collaborators

German Federal Ministry of Education and Research

Investigators

  • Principal Investigator: Anne K Ebert, Prof. Dr., Pediatric Urology, Department for Urology University of Ulm, Germany

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

June 1, 2024

Primary Completion (Estimated)

August 1, 2026

Study Completion (Estimated)

October 1, 2026

Study Registration Dates

First Submitted

January 21, 2021

First Submitted That Met QC Criteria

January 27, 2021

First Posted (Actual)

January 28, 2021

Study Record Updates

Last Update Posted (Actual)

August 3, 2023

Last Update Submitted That Met QC Criteria

August 2, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MUST (Other Identifier: Alias Study Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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