Ignite Creation Date:
2025-12-25 @ 4:15 AM
Ignite Modification Date:
2026-01-02 @ 5:28 AM
Study NCT ID:
NCT07027020
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-06-18
First Post:
2025-05-22
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Efficacy of Intravesical Oxybutynin in Children With Neurogenic Bladder Dysfunction
Sponsor:
Central Hospital, Nancy, France
Organization:
Study Overview
Official Title:
Efficacy of Intravesical Oxybutynin in Children With Neurogenic Bladder Dysfunction: A Randomized, Prospective Controlled Multi-center Trial
Status:
NOT_YET_RECRUITING
Status Verified Date:
2025-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
POXIPEN
Brief Summary:
Malformation of the lumbosacral region (spina bifida) affects the innervation of the bladder in children. The usual evolution leads to a neurological bladder with small capacity, poor compliancy and overactivity, exposing to incontinence and obstruction to the evacuation of urine. It is responsible for renal failure requiring dialysis and transplantation. Current therapeutics aim to evacuate urine and reduce intravesical pressure. It gradually combines 1) intermittent catheterization, 2) anticholinergics, 3) botulinum toxin (Botox®) injection into the detrusor (bladder muscle) by cystoscopy and 4) surgery (vesicostomy, Bricker, enterocystoplasty). Oxybutynin relaxes the detrusor, improves continence and reduces intravesical pressure. It is usually administered per os, but there are contraindications (glaucoma, myasthenia), side effects (constipation, dry mouth). It can be difficult to swallow for children, and drug resistance may develop. It can lead to ineffective treatment requiring therapeutic escalation. The next step, intradetrusor Botox® injection, is invasive (cystoscopy), has a limited duration of action (6 months) and must be performed under general anesthesia in children.
Surgical treatments are effective but irreversible and responsible for morbidity and mortality. A randomized study was performed demonstrating the efficacy of intravesical oxybutynin compared to oral administration in adult patients. This study found a significant increase in bladder capacity and a significant decrease in side effects in the intravesical oxybutynin group. Due to the relative difficulties of intravesical oxybutynin delivery (preparation, cost) and the more invasive nature, it is not used as an alternative to oral oxybutynin.
Our hypothesis is that this treatment may have a legitimate place in the treatment of neurogenic bladder in patients with failure of anticholinergic treatment before a therapeutic escalation requiring an invasive procedure (Botox®, enterocystoplasty) especially in children for whom repeated general anesthesia for Botox® injection may interfere with brain development. In this way, we aim to extend the time to therapeutic escalation in the pediatric population.
The main objective of the trial is to compare the efficacy on maximal bladder capacity of intravesical oxybutynin instillation versus placebo in the treatment of children with overactive neurogenic bladder (spina bifida), performing intermittent catheterization, for whom oral anticholinergic treatment is ineffective or poorly tolerated.
Surgical treatments are effective but irreversible and responsible for morbidity and mortality. A randomized study was performed demonstrating the efficacy of intravesical oxybutynin compared to oral administration in adult patients. This study found a significant increase in bladder capacity and a significant decrease in side effects in the intravesical oxybutynin group. Due to the relative difficulties of intravesical oxybutynin delivery (preparation, cost) and the more invasive nature, it is not used as an alternative to oral oxybutynin.
Our hypothesis is that this treatment may have a legitimate place in the treatment of neurogenic bladder in patients with failure of anticholinergic treatment before a therapeutic escalation requiring an invasive procedure (Botox®, enterocystoplasty) especially in children for whom repeated general anesthesia for Botox® injection may interfere with brain development. In this way, we aim to extend the time to therapeutic escalation in the pediatric population.
The main objective of the trial is to compare the efficacy on maximal bladder capacity of intravesical oxybutynin instillation versus placebo in the treatment of children with overactive neurogenic bladder (spina bifida), performing intermittent catheterization, for whom oral anticholinergic treatment is ineffective or poorly tolerated.
Detailed Description:
Secondary objectives of the trial :
* Secondary efficacy objectives will be analyzed following a fixed sequence in a priori ordered hypotheses: to evaluate after treatment with intravesical oxybutynin (IVO) or placebo instillation: maximum bladder pressure evolution; incontinence: time to clinical treatment failure perceived by the patient. These parameters are considered by experts to be objective markers of neurological bladder improvement, in addition to the primary urodynamic objective.
* Evaluate and compare between groups the tolerance and adverse effects of treatment.
* Evaluate and compare between groups the number of responders and continent patients after treatment
* Evaluate and compare between groups the usability of Intravesical Oxybutynin.
* Evaluate and compare between groups the evolution of quality of life.
* Evaluate and compare between groups the evolution of bladder diary data.
* Evaluate and compare between groups the evolution of other urodynamic parameters.
* Evaluate and compare between groups the evolution of renal ultrasonography parameters.
* Identify factors, among the patient's characteristics, associated with a greater probability to be a responder or non-responder to intravesical oxybutynin.
* Secondary efficacy objectives will be analyzed following a fixed sequence in a priori ordered hypotheses: to evaluate after treatment with intravesical oxybutynin (IVO) or placebo instillation: maximum bladder pressure evolution; incontinence: time to clinical treatment failure perceived by the patient. These parameters are considered by experts to be objective markers of neurological bladder improvement, in addition to the primary urodynamic objective.
* Evaluate and compare between groups the tolerance and adverse effects of treatment.
* Evaluate and compare between groups the number of responders and continent patients after treatment
* Evaluate and compare between groups the usability of Intravesical Oxybutynin.
* Evaluate and compare between groups the evolution of quality of life.
* Evaluate and compare between groups the evolution of bladder diary data.
* Evaluate and compare between groups the evolution of other urodynamic parameters.
* Evaluate and compare between groups the evolution of renal ultrasonography parameters.
* Identify factors, among the patient's characteristics, associated with a greater probability to be a responder or non-responder to intravesical oxybutynin.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2022-501902-36-00 | CTIS | None | View |