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

Simone Spuler, MD

Status and phase: Not yet enrolling; Phase 2; Phase 1

Conditions: Epispadias, Male; Urinary Incontinence

Treatments: Biological: Primary human muscle stem cells (Satori-01); Other: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT04729582

MUST

Details and patient eligibility

About

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.

Full 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.

Enrollment: 21 estimated patients

Sex: Male

Ages: 3 to 17 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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21 participants in 2 patient groups, including a placebo group

Verum Group

Experimental group

Description:

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

Treatment:

Biological: Primary human muscle stem cells (Satori-01)

Placebo group

Placebo Comparator group

Description:

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

Treatment:

Other: Placebo