EVALUATION OF THE SAFETY AND EFFICACY OF ADJUSTABLE CONTINENCE THERAPY BALLOONS IN BLADDER EXSTROPHY AND INCONTINENT EPISPADIAS PATIENTS

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to evaluate the effectiveness and safety of a minimally invasive surgical procedure with ACT (Adjustable Continence Therapy) balloons implantation for the treatment of urinary incontinence in children with bladder exstrophy or isolated epispadias. The ACT therapy consists of two small adjustable silicone balloons connected with a tubing to a port, surgically placed around the bladder neck, one on each side of the urethra.

Official Title
-----------------
PROSPECTIVE STUDY EVALUATING THE SAFETY AND EFFECTIVENESS OF ADJUSTABLE CONTINENCE THERAPY (ACT) BALLOONS FOR THE MANAGEMENT OF URINARY INCONTINENCE IN CHILDREN WITH BLADDER EXSTROPHY OR INCONTINENT EPISPADIAS

Conditions
-----------------
Bladder Exstrophy

Intervention / Treatment
-----------------
* Device: ACT


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Boys and girls > 5 years with bladder exstrophy or isolated epispadias; sphincteric incontinence (leak point pressure < 45 cm d'H20, open bladder neck during filling, stress urinary incontinence); normal renal function (eGFR > 90ml/min); no (or stable) upper urinary tract dilatation in ultrasound. Exclusion Criteria: under 5 years or more than 18 years old; renal insufficiency (acute or chronic); evolutive deterioration of the upper urinary tract (hydronephrosis); unmanageable detrusor instability; residual volume greater than 100 ml after voiding; bleeding disorders.

Ages Eligible for Study
-----------------
Minimum Age: 5 Years
Maximum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Study Arm 1<br>Children with bladder exstrophy or isolated epispadias | Device: ACT<br>* Implantation of ACT balloon, Uromedica (Irvine, CA, USA) periurethrally at the bladder neck. The ACT system is a permanent implant designed for the correction of incontinence in patients.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluation of the clinical impact of ACT balloon on urinary continence with pad weight | Therapeutic success will be based on whether patients demonstrate at least a 50% reduction in 24 hour Pad weight at 6, 12 and 24 months follow-up compared to the pad weight results at baseline. | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of pads per day (voiding diary) | Change of number of peds changed per day from baseline to 6, 12 and 24 months (the best : 0 pads changed a day). | 6, 12 and 24 months | 
| Number of incontinence episodes per day (voiding diary) | Change in number of incontinence episodes per day from baseline to 6, 12 and 24 months (the best : 0) | 6, 12 and 24 months | 
| PIN-Q questionnaire | Change on validated incontinence quality of life questionnaire from baseline to 6, 12 and 24 months | 6, 12 and 24 months | 
| Incidence of urethral stricture and device erosion after ACT implantation | Cumulative incidence of ACT related clinically relevant urethral structure and device erosions (the best : 0) | 6, 12 and 24 months |