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

Primary Purpose

Urinary Incontinence, Epispadias, Male

Status
Not yet recruiting
Phase
Phase 1
Locations
Germany
Study Type
Interventional
Intervention
Primary human muscle stem cells (Satori-01)
Placebo
Sponsored by
Simone Spuler, MD
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Urinary Incontinence focused on measuring Isolated epispadias, Urinary incontinence, Muscle stem cell therapy, Autologous cell therapy

Eligibility Criteria

3 Years - 17 Years (Child)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwig Hospital
  • Pediatric Urology, Department for Urology University of Ulm

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Verum Group

Placebo group

Arm Description

Participants receive primary human muscle stem cells as one-time injection into the urethral sphincter region under visual control using cystoscopy.

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

Outcomes

Primary Outcome Measures

Prevalence of Intervention-related Adverse Events
Characterization of type, incidence, severity, and duration of adverse events
Change in Leak Point Pressure (LPP)
Change in LPP is calculated from baseline measurement

Secondary Outcome Measures

Full Information

First Posted
January 21, 2021
Last Updated
August 2, 2023
Sponsor
Simone Spuler, MD
Collaborators
German Federal Ministry of Education and Research
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1. Study Identification

Unique Protocol Identification Number
NCT04729582
Brief Title
Autologous Muscle Stem Cell Therapy for Treatment of Congenital Urinary Incontinence in Epispadias Patients
Acronym
MUST
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
June 2024 (Anticipated)
Primary Completion Date
August 2026 (Anticipated)
Study Completion Date
October 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Simone Spuler, MD
Collaborators
German Federal Ministry of Education and Research

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Urinary Incontinence, Epispadias, Male
Keywords
Isolated epispadias, Urinary incontinence, Muscle stem cell therapy, Autologous cell therapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Model Description
This is a randomized placebo-controlled clinical trial
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
21 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Verum Group
Arm Type
Experimental
Arm Description
Participants receive primary human muscle stem cells as one-time injection into the urethral sphincter region under visual control using cystoscopy.
Arm Title
Placebo group
Arm Type
Placebo Comparator
Arm Description
Participants receive placebo solution as one-time injection into the external urethral sphincter region under visual control using cystoscopy.
Intervention Type
Biological
Intervention Name(s)
Primary human muscle stem cells (Satori-01)
Intervention Description
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.
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo is the injection solution without muscle stem cells.
Primary Outcome Measure Information:
Title
Prevalence of Intervention-related Adverse Events
Description
Characterization of type, incidence, severity, and duration of adverse events
Time Frame
Upto 12 months post-intervention
Title
Change in Leak Point Pressure (LPP)
Description
Change in LPP is calculated from baseline measurement
Time Frame
Six months post-intervention

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Simone Spuler, Prof. Dr.
Phone
+49 (0) 30 450 540 501
Email
simone.spuler@charite.de
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anne K Ebert, Prof. Dr.
Organizational Affiliation
Pediatric Urology, Department for Urology University of Ulm, Germany
Official's Role
Principal Investigator
Facility Information:
Facility Name
Clinic for Pediatric Urology in cooperation with University Hospital Regensburg, St. Hedwig Hospital
City
Regensburg
ZIP/Postal Code
93049
Country
Germany
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Wolfgang H Rösch, Prof. Dr.
Phone
+49 (0) 941 369 5453
Email
wolfgang.roesch@barmherzige-regensburg.de
First Name & Middle Initial & Last Name & Degree
Wolfgang H Rösch, Prof. Dr.
Facility Name
Pediatric Urology, Department for Urology University of Ulm
City
Ulm
ZIP/Postal Code
89075
Country
Germany
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anne K Ebert, Prof. Dr.
Phone
+49 (0) 731 500 58000
Email
Anne-Karoline.Ebert@uniklinik-ulm.de
First Name & Middle Initial & Last Name & Degree
Anne K Ebert, Prof. Dr.

12. IPD Sharing Statement

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

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