Efficacy of Intravesical Oxybutynin in Children With Neurogenic Bladder Dysfunction
POXIPEN
2 other identifiers
interventional
60
1 country
19
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. The hypothesis of this study 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, the investigators 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jan 2026
19 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 22, 2025
CompletedFirst Posted
Study publicly available on registry
June 18, 2025
CompletedStudy Start
First participant enrolled
January 15, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 1, 2028
April 14, 2026
January 1, 2026
2 years
May 22, 2025
April 9, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Evolution of maximal bladder capacity at 4 weeks of treatment (end of follow-up)
This parameter is considered by experts to be an objective marker of improvement in neurological bladder, in addition to the secondary clinical objective. It was used as the primary objective in the only prospective study in the literature. It is obtained by cystomanometry and is defined by the maximum volume (in mL) measured at the moment when permission to urinate is given to the patient, after filling via urethral catheter. In younger patients, urination occurs spontaneously, and this value is obtained a posteriori by curve analysis. If treatment is successful, maximum bladder capacity should increase.
4 weeks
Secondary Outcomes (15)
Evolution of maximal bladder pressure at 4 weeks of treatment (end of follow-up)
4 weeks
Urinary incontinence
4 weeks
Evaluation of the number of side effects
4 weeks
Proportion of responders at 4 weeks of treatment
4 weeks
Proportion of continent patients at 4 weeks of treatment
4 weeks
- +10 more secondary outcomes
Study Arms (2)
IntraVesical Oxybutynin (IVO)
EXPERIMENTALInstillation of oxybutynin conditioned in VESOXX® 10mg/10mL ready-to-use syringes (1mg/mL) at the end of the evacuation catheterization. Dose: 0.4mg/kg/day in 2 to 3 instillations per day, i.e. a maximum of 10mg per instillation. Maximum dose: 20mg/day for patients aged 6-11 years and 11 months, and 30mg/day for patients aged 12-17 years.
IntraVesical Placebo (PCB)
PLACEBO COMPARATORInstillation of saline solution conditioned in 10 mL ready-to-use syringes at the end of the evacuation catheterization. Dose equivalent to IVO in ml, i.e. always 0.4mg/kg/day, i.e. a maximum of 10 mL per instillation. Maximum dose: 20mL/day for patients aged 6-11 years and 11 months, and 30mL/day for patients aged 12 to 17 years.
Interventions
Pharmaceutical form: solution. Route of administration: Intravesical. Medicinal product unique ID: PRD8074745. EU active substance code: SUB03581MIG.
Pharmaceutical form: solution. Route of administration: Intravesical.
Eligibility Criteria
You may qualify if:
- Person affiliated to or beneficiary of a social security plan
- In failure of treatment with one or more anticholinergics defined by a response considered insufficient by the investigator after at least 4 weeks of optimal dose treatment, unable to take oral oxybutynin or intolerable adverse events
- Having performed renal ultrasonography less than 2 months ago
- Having performed cystomanometry less than 6 months ago including maximal bladder capacity and maximal bladder pressure (preferably not under oral oxybutynin treatment)
- Age between 6 and 17 years old
- Informed about study organization, having given consent to participate and each legal representative have signed the informed consent
- Having undergone the medical examination adapted to research
- Presenting overactive bladder due to spina bifida confirmed by urodynamic check-up of less than 6 months. Overactive bladder is defined according to International Children's Continence Society, (ICCS): "a urodynamic observation characterized by involuntary detrusor contractions during the filling phase which may be spontaneous or provoked"
- Carrying out intermittent catheterization for at least 6 weeks and at least three times a day
- Able and volunteer to perform intravesical catheterization and instillation (patient or parents).
You may not qualify if:
- Person displaying known allergy to one of the components of evaluated product (notably oxybutynin).
- Person with congestive cardiac failure
- Person with cardiac arrhythmia
- Person with tachycardia
- Person with uncontrolled hypertension
- Person under one of the following treatments : Bisphosphonates; Cytochrome P450 Inhibitors (such as ketoconazole and Erythromycin); Cholinesterase inhibitors.
- Person displaying a contraindication to evaluated product, in particular: Hypersensitivity to oxybutynin; Myasthenia; Angle-closure glaucoma; Functional or organic gastrointestinal obstruction including pyloric stenosis, paralytic ileus and intestinal atony; Serious gastro-intestinal disorders (e.g., severe ulcerative colitis and toxic megacolon); Patients who have undergone ileostomy, colostomy, severe hemorrhagic colectasis or rectocolitis; Subvesical obstruction (urethral stenosis, posterior urethra valve); Ongoing treatment with anticholinergic drugs for another indication that could not be stopped; Patient with polyuria of other origin (renal, heart, potomania); Concomitant oxygenotherapy.
- Woman of childbearing age without highly effective contraception (Sexual abstinence OR combined contraception by oral, intravaginal or transdermal ovulation inhibition OR progestin-only contraception by oral, injectable or implantable ovulation inhibition OR Intrauterine device or hormonal IUD).
- Pregnant, parturient or breastfeeding woman.
- Person deprived of liberty for judicial or administrative decision.
- Person under psychiatric care as referred in articles L. 3212-1 and L. 3213-1.
- Intradetrusor injection of botulinum toxin less than 6 months before.
- Person with hyperthyroidism
- Person with coronary cardiac disease.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Central Hospital, Nancy, Francelead
- Ministry of Health, Francecollaborator
- FARCO PHARMAcollaborator
Study Sites (19)
Centre Hospitalier Universitaire de Besançon
Besançon, France
Centre Hospitalier Universitaire De Bordeaux
Bordeaux, France
Centre Hospitalier Régional Et Universitaire De Brest
Brest, France
Centre Hospitalier Universitaire De Caen Normandie
Caen, France
Centre Hospitalier Universitaire de Clermont-Ferrand
Clermont-Ferrand, France
Centre Hospitalier De Colmar
Colmar, France
Centre Hospitalier Universitaire Grenoble Alpes
Grenoble, France
Centre Hospitalier Universitaire de Lille
Lille, France
Centre Hospitalier Et Universitaire De Limoges
Limoges, France
Centre Hospitalier Régional De Marseille
Marseille, France
Fondation Lenval Nice
Nice, France
Hôpital Necker Enfants Malades
Paris, France
Hôpital Trousseau (chirurgie viscérale pédiatrique et néonatale)
Paris, France
Hôpital Trousseau (médecine physique et de réadaptation pédiatrique)
Paris, France
Centre Hospitalier Universitaire de Poitiers
Poitiers, France
Centre Hospitalier Universitaire de Rennes
Rennes, France
Centre Hospitalier Universitaire De Saint Etienne
Saint-Etienne, France
Les Hopitaux Universitaires De Strasbourg
Strasbourg, France
Centre Hospitalier Régional Et Universitaire de Nancy
Vandœuvre-lès-Nancy, France
Related Publications (1)
Buzzi M, Epstein J, Hatem Z, Juge N, Mazeaud C, Lemelle JL, Berte N. Intravesical oxybutynin for bladder capacity in children with spina bifida: the 'Place de l'OXybutynine Intravesicale chez le Patient Enfant Neurologique' (POXIPEN) trial protocol. BJU Int. 2026 Feb;137(2):390-398. doi: 10.1111/bju.70058. Epub 2025 Nov 3.
PMID: 41178302DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Principal investigator, pediatric surgeon
Study Record Dates
First Submitted
May 22, 2025
First Posted
June 18, 2025
Study Start
January 15, 2026
Primary Completion (Estimated)
January 1, 2028
Study Completion (Estimated)
June 1, 2028
Last Updated
April 14, 2026
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will not share