EVALUATION OF THE SAFETY AND EFFICACY OF ADJUSTABLE CONTINENT THERAPY IN CHILDREN WITH SPINAL DYSRAPHISM. (BPUA)

EVALUATION OF THE SAFETY AND EFFICACY OF ADJUSTABLE CONTINENT THERAPY FOR THE MANAGEMENT OF NEUROGENIC INCONTINENCE IN CHILDREN WITH SPINAL DYSRAPHISM.

Urinary continence is still a difficult goal to achieve in neurogenic bladder patients. Conservative treatment of neurogenic urinary incontinence using clean intermittent catheterization in conjunction with pharmacological therapy, notably anticholinergic medication +/- adrenergic agents, may produce continence in a variable number of patients. In other patients, reconstructive surgery of the bladder and/or bladder neck is necessary to achieve urinary continence. Surgical treatment options to increase bladder outlet resistance in patients with neurogenic bladder include injection of bulking agents around the bladder neck, bladder neck reconstruction, fascial sling procedures and Artificial Urinary Sphincter.

The Adjustable Continence Therapy system (ACT) or periurethral adjustable balloons are a minimally invasive device consisting of two volume-adjustable balloons implanted periurethrally at the bladder neck as a method of augmenting titration for urethral coaptation. Adjustable means that such system would be adaptable to the individual clinical condition.

Originally conceived and developed as a treatment for female stress urinary incontinence, the technique has been then adapted and balloons were globally developed for the use in postprostatectomy incontinence. The published success rate in male after prostatectomy and in women was respectively 56 to 92% and 60 to 83%.

The investigators hypothesize that the use of ACT for treating incontinence in children secondary to neurogenic sphincter incontinence could compress the urethra or the bladder neck, acting as an extrinsic occlusive system increasing passive and dynamic urethral and bladder resistance.

Goal of the study:

To prospectively assess the efficacy and safety of periurethral adjustable balloons in the treatment of neurogenic incontinence in children with spinal dysraphism.

Material and methods :

A prospective study will be performed at La Timone Enfants hospital and La Nord hospital in Marseille, France. Boys and girls at least at school age (5 or 6 years) with neurogenic incontinence due to outlet issues (low detrusor leak point pressure and low stress leak point pressure) with spinal dysraphism will be recruited.

The ACT balloon is an implantable medical device developed and furnished for free by Uromedica (Irvine, CA, USA). The procedure is performed under general anaesthesia using the same implantation technique as published in adult population.

Study Overview

• Status: Not yet recruiting
• Conditions: Neurogenic Bladder Due to Spinal Dysraphism
• Intervention / Treatment: Device: Implantation of ACT balloon, Uromedica (Irvine, CA, USA) periurethrally at the bladder neck

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Claire MORANDO; Email: claire.morando@ap-hm.fr
• Study Contact Backup: Name: Alice FAURE; Phone Number: 04.91.96.81.41; Email: alice.faure@ap-hm.fr

Study Locations

• France
  • Marseille, France, 13354
    • Assistance Publique Hopitaux de Marseille
    • Contact: Alice FAURE; Phone Number: 04.91.96.81.41; Email: alice.faure@ap-hm.fr
    • Contact: Claire MORANDO; Email: claire.morando@yahoo.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Boys and girls > 5 years with spinal dysraphism (occult or open)
• Neurogenic sphincteric incontinence (leak point pressure < 45 cm d'H20, open bladder neck during filling, urinary incontinence)
• Normal renal function (eGFR > 90ml/min)
• No (or stable) upper urinary tract dilatation in ultrasound
• In case no compliante bladder ( bladder capacity < 50% for the age) a concomitant injection of botox endoscopically will be performed

Exclusion Criteria:

• under 5 years with an acquired neurogenic bladder
• renal insufficiency (acute or chronic)
• evolutive deterioration of the upper urinary tract (hydronephrosis)
• active systemic or urinary tract infections
• unmanageable detrusor instability
• reduced bladder compliance
• residual volume greater than 100 ml after voiding
• bleeding disorders
• urethral stenosis
• who refused CIC
• patent sacral bedsore

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Children with neurogenic incontinence with spinal dysraphism	Device: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Measurement of urinary continence with the Schulte-Baukloh score and 24hours pad-test	Evaluation of urinary continence with mictional schedule and 24hours pad-test (Patients will be classified as "dry" if they will be using no pads or a single ''security pad'' per day; "significantly improved" if a reduction >50% in pad usage (usually representing 2- 5 pads/day, depending on preoperative pad use); and little/no improvement (>5 pads/day and <50% improvement against baseline) and the Schulte-Baukloh score	5 YEARS

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of the quality of life with the PIN-Q score	The child will be asked to answer some items	5 YEARS

Collaborators and Investigators

• Sponsor: Assistance Publique Hopitaux De Marseille
• Investigators: Study Director: Urielle DESALBRES, ASSISTANTE PUBLIQUE HOPITAUX DE MARSEILLE

Study record dates

Study Major Dates

• Study Start (Anticipated): December 30, 2017
• Primary Completion (Anticipated): October 30, 2023
• Study Completion (Anticipated): October 30, 2024

Study Registration Dates

• First Submitted: August 25, 2017
• First Submitted That Met QC Criteria: November 21, 2017
• First Posted (Actual): November 24, 2017

Study Record Updates

• Last Update Posted (Actual): November 24, 2017
• Last Update Submitted That Met QC Criteria: November 21, 2017
• Last Verified: November 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Urologic Diseases; Urinary Bladder Diseases; Neurologic Manifestations; Congenital Abnormalities; Nervous System Malformations; Urinary Bladder, Neurogenic; Neural Tube Defects; Spinal Dysraphism
• Other Study ID Numbers: 2016-49; 2016-A01974-47 (Registry Identifier: ANSM)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No