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

NCT ID: NCT04729582

Last Updated: 2023-08-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-06-30

Study Completion Date

2026-10-31

Brief Summary

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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

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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.

Conditions

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Urinary Incontinence Epispadias, Male

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This is a randomized placebo-controlled clinical trial
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Verum Group

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

Group Type EXPERIMENTAL

Primary human muscle stem cells (Satori-01)

Intervention Type 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 group

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Placebo is the injection solution without muscle stem cells.

Interventions

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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.

Intervention Type BIOLOGICAL

Placebo

Placebo is the injection solution without muscle stem cells.

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

3 Years

Maximum Eligible Age

17 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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German Federal Ministry of Education and Research

OTHER_GOV

Sponsor Role collaborator

Simone Spuler, MD

OTHER

Sponsor Role lead

Responsible Party

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Simone Spuler, MD

Principal investigator

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Anne K Ebert, Prof. Dr.

Role: PRINCIPAL_INVESTIGATOR

Pediatric Urology, Department for Urology University of Ulm, Germany

Locations

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

Regensburg, , Germany

Site Status

Pediatric Urology, Department for Urology University of Ulm

Ulm, , Germany

Site Status

Countries

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Germany

Central Contacts

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Simone Spuler, Prof. Dr.

Role: CONTACT

+49 (0) 30 450 540 501

Facility Contacts

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Wolfgang H Rösch, Prof. Dr.

Role: primary

+49 (0) 941 369 5453

Anne K Ebert, Prof. Dr.

Role: primary

+49 (0) 731 500 58000

Other Identifiers

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MUST

Identifier Type: -

Identifier Source: org_study_id

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