EVALUATION OF THE SAFETY AND EFFICACY OF ADJUSTABLE CONTINENT THERAPY IN CHILDREN WITH SPINAL DYSRAPHISM. (BPUA)
Primary Purpose
Neurogenic Bladder Due to Spinal Dysraphism
Status
Not yet recruiting
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Implantation of ACT balloon, Uromedica (Irvine, CA, USA) periurethrally at the bladder neck
Sponsored by
About this trial
This is an interventional treatment trial for Neurogenic Bladder Due to Spinal Dysraphism
Eligibility Criteria
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
Sites / Locations
- Assistance Publique Hopitaux de Marseille
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Children with neurogenic incontinence with spinal dysraphism
Arm Description
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
Evaluation of the quality of life with the PIN-Q score
The child will be asked to answer some items
Full Information
NCT ID
NCT03351634
First Posted
August 25, 2017
Last Updated
November 21, 2017
Sponsor
Assistance Publique Hopitaux De Marseille
1. Study Identification
Unique Protocol Identification Number
NCT03351634
Brief Title
EVALUATION OF THE SAFETY AND EFFICACY OF ADJUSTABLE CONTINENT THERAPY IN CHILDREN WITH SPINAL DYSRAPHISM.
Acronym
BPUA
Official Title
EVALUATION OF THE SAFETY AND EFFICACY OF ADJUSTABLE CONTINENT THERAPY FOR THE MANAGEMENT OF NEUROGENIC INCONTINENCE IN CHILDREN WITH SPINAL DYSRAPHISM.
Study Type
Interventional
2. Study Status
Record Verification Date
November 2017
Overall Recruitment Status
Not yet recruiting
Study Start Date
December 30, 2017 (Anticipated)
Primary Completion Date
October 30, 2023 (Anticipated)
Study Completion Date
October 30, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique Hopitaux De Marseille
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
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neurogenic Bladder Due to Spinal Dysraphism
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Children with neurogenic incontinence with spinal dysraphism
Arm Type
Experimental
Intervention Type
Device
Intervention Name(s)
Implantation of ACT balloon, Uromedica (Irvine, CA, USA) periurethrally at the bladder neck
Intervention Description
The ACT system is a permanent implant designed for the correction of incontinence in patients.
Primary Outcome Measure Information:
Title
Measurement of urinary continence with the Schulte-Baukloh score and 24hours pad-test
Description
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
Time Frame
5 YEARS
Secondary Outcome Measure Information:
Title
Evaluation of the quality of life with the PIN-Q score
Description
The child will be asked to answer some items
Time Frame
5 YEARS
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Alice FAURE
Phone
04.91.96.81.41
Email
alice.faure@ap-hm.fr
First Name & Middle Initial & Last Name or Official Title & Degree
Claire MORANDO
Email
claire.morando@ap-hm.fr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Urielle DESALBRES
Organizational Affiliation
ASSISTANTE PUBLIQUE HOPITAUX DE MARSEILLE
Official's Role
Study Director
Facility Information:
Facility Name
Assistance Publique Hopitaux de Marseille
City
Marseille
ZIP/Postal Code
13354
Country
France
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alice FAURE
Phone
04.91.96.81.41
Email
alice.faure@ap-hm.fr
First Name & Middle Initial & Last Name & Degree
Claire MORANDO
Email
claire.morando@yahoo.fr
12. IPD Sharing Statement
Learn more about this trial
EVALUATION OF THE SAFETY AND EFFICACY OF ADJUSTABLE CONTINENT THERAPY IN CHILDREN WITH SPINAL DYSRAPHISM.
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