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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04729582
Recruitment Status : Not yet recruiting
First Posted : January 28, 2021
Last Update Posted : December 7, 2022
Sponsor:
Collaborator:
German Federal Ministry of Education and Research
Information provided by (Responsible Party):
Simone Spuler, MD, Charite University, Berlin, Germany

Study Description
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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.

Condition or disease Intervention/treatment Phase
Urinary Incontinence Epispadias, Male Biological: Primary human muscle stem cells (Satori-01) Other: Placebo Phase 1 Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 21 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: This is a randomized placebo-controlled clinical trial
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Estimated Study Start Date : May 2023
Estimated Primary Completion Date : August 2026
Estimated Study Completion Date : October 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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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.


Outcome Measures
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Primary Outcome Measures :
  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


Eligibility Criteria
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Ages Eligible for Study:   3 Years to 17 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04729582


Contacts
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Contact: Simone Spuler, Prof. Dr. +49 (0) 30 450 540 501 simone.spuler@charite.de

Locations
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Germany
Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwig Hospital
Regensburg, Germany, 93049
Contact: Wolfgang H Rösch, Prof. Dr.    +49 (0) 941 369 5453    wolfgang.roesch@barmherzige-regensburg.de   
Sub-Investigator: Wolfgang H Rösch, Prof. Dr.         
Pediatric Urology, Department for Urology University of Ulm
Ulm, Germany, 89075
Contact: Anne K Ebert, Prof. Dr.    +49 (0) 731 500 58000    Anne-Karoline.Ebert@uniklinik-ulm.de   
Principal Investigator: Anne K Ebert, Prof. Dr.         
Sponsors and Collaborators
Simone Spuler, MD
German Federal Ministry of Education and Research
Investigators
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Principal Investigator: Anne K Ebert, Prof. Dr. Pediatric Urology, Department for Urology University of Ulm, Germany
More Information
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Responsible Party: Simone Spuler, MD, Principal investigator, Charite University, Berlin, Germany
ClinicalTrials.gov Identifier: NCT04729582    
Other Study ID Numbers: MUST
First Posted: January 28, 2021    Key Record Dates
Last Update Posted: December 7, 2022
Last Verified: December 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Simone Spuler, MD, Charite University, Berlin, Germany:
Isolated epispadias
Urinary incontinence
Muscle stem cell therapy
Autologous cell therapy
Additional relevant MeSH terms:
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Urinary Incontinence
Enuresis
Epispadias
Urination Disorders
Urologic Diseases
Lower Urinary Tract Symptoms
Urological Manifestations
 Behavioral Symptoms
Elimination Disorders
Mental Disorders
Urogenital Abnormalities
Urethral Diseases
Congenital Abnormalities