Autologous Muscle Stem Cell Therapy for Treatment of Congenital Urinary Incontinence in Epispadias Patients (MUST)
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|ClinicalTrials.gov Identifier: NCT04729582|
Recruitment Status : Not yet recruiting
First Posted : January 28, 2021
Last Update Posted : January 28, 2021
|Condition or disease||Intervention/treatment||Phase|
|Urinary Incontinence Epispadias, Male||Biological: Primary human muscle stem cells (Satori-01) Other: Placebo||Phase 1 Phase 2|
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 (Clinical Trial)|
|Estimated Enrollment :||21 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||This is a randomized placebo-controlled clinical trial|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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 :||April 2022|
|Estimated Primary Completion Date :||October 2024|
|Estimated Study Completion Date :||April 2025|
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.
Placebo is the injection solution without muscle stem cells.
- Prevalence of Intervention-related Adverse Events [ Time Frame: Upto 12 months post-intervention ]Characterization of type, incidence, severity, and duration of adverse events
- Change in Leak Point Pressure (LPP) [ Time Frame: Six months post-intervention ]Change in LPP is calculated from baseline measurement
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
|Contact: Simone Spuler, Prof. Dr.||+49 (0) 30 450 540 email@example.com|
|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 firstname.lastname@example.org|
|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.|
|Principal Investigator:||Anne K Ebert, Prof. Dr.||Pediatric Urology, Department for Urology University of Ulm, Germany|