Efficacy of intravesical oxybutynin in children with neurogenic bladder dysfunction: A randomized, prospective controlled multi-center trial.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Centre Hospitalier Regional Universitaire De Nancy

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

15 Jan 2026 → ongoing

Decision date (initial)

2025-01-09

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

French Ministry of Health · FARCO PHARMA

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy

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.

Secondary objectives 9

1. 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.
2. Evaluate and compare between groups the tolerance and adverse effects of treatment.
3. Evaluate and compare between groups the number of responders and continent patients after treatment
4. Evaluate and compare between groups the usability of Intravesical Oxybutynin.
5. Evaluate and compare between groups the evolution of quality of life.
6. Evaluate and compare between groups the evolution of bladder diary data.
7. Evaluate and compare between groups the evolution of other urodynamic parameters.
8. Evaluate and compare between groups the evolution of renal ultrasonography parameters.
9. Identify factors, among the patient’s characteristics, associated with a greater probability to be a responder or non-responder to intravesical oxybutynin.

Conditions and MedDRA coding

spina bifida

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. Person affiliated to or beneficiary of a social security plan.
2. Having performed renal ultrasonography less than 2 months ago.
3. Having performed cystomanometry less than 6 months ago including maximal bladder capacity and maximal bladder pressure (preferably not under oral oxybutynin treatment).
4. Age between 6 and 17 years old.
5. Informed about study organization, having given consent to participate and each legal representative have signed the informed consent.
6. Having undergone the medical examination adapted to research.
7. 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”.
8. Carrying out intermittent catheterization for at least 6 weeks and at least three times a day.
9. Able and volunteer to perform intravesical catheterization and instillation (patient or parents).
10. 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.

Exclusion criteria 14

1. Person displaying known allergy to one of the components of evaluated product (notably oxybutynin).
2. 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.
3. 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 OR tubal ligation OR male partner with vasectomy).
4. Pregnant, parturient or breastfeeding woman.
5. Person deprived of liberty for judicial or administrative decision.
6. Person under psychiatric care as referred in articles L. 3212-1 and L. 3213-1.
7. Intradetrusor injection of botulinum toxin less than 6 months before.
8. Person with hyperthyroidism
9. Person with coronary cardiac disease
10. Person with congestive cardiac failure
11. Person with cardiac arrhythmia
12. Person with tachycardia
13. Person with uncontrolled hypertension
14. Person under one of the following treatments : Bisphosphonates; Cytochrome P450 Inhibitors (such as ketoconazole and Erythromycin); Cholinesterase inhibitors.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Evolution of maximal bladder capacity at 4 weeks of treatment (end of follow-up).

Secondary endpoints 10

1. Evolution of maximal bladder pressure at 4 weeks of treatment (end of follow-up).
2. Time to clinical treatment failure defined by criteria found in literature and marketing authorization of Botox® in a 28-day time frame (at least one of the 3 criteria): Treatment judged as non-effective by either the patient or the practitioner; Reduction of urinary incontinence to less than 50% of the initial occurrences measured in the initial bladder diary; Intolerable side effects reported by the patient.
3. Evaluation of tolerance and side effects: digestive, psychiatric, neurological, cutaneous, urological, pain associated with instillation.
4. Proportion of responders at 4 weeks of treatment (patients who had at least a 50% reduction in urinary incontinence episodes).
5. Proportion of continent patients at 4 weeks of treatment (patients who had a 100% reduction in urinary incontinence episodes).
6. Product usability measured with usability questionnaire (UMUX-LITE and specific questions).
7. Standardized difference in patient quality of life calculated according to the ICIQ-UI-SF and KIDSCREEN-10 score between the beginning and the end of the study.
8. Evolution of the elements of the bladder diary at 4 weeks of treatment: Number and volume of urinary catheterizations over 72 hours during the week preceding each visit (V1, V2 and V3).
9. Evolution of the elements of the other urodynamic assessments at 4 weeks of treatment: Bladder compliance; Minimum filling volume causing uninhibited detrusor contraction.
10. Evolution of renal ultrasonography at 4 weeks of treatment: Renal pelvis anteroposterior diameter; Ureters diameter.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Regional Universitaire De Nancy

Sponsor organisation

Centre Hospitalier Regional Universitaire De Nancy

Address

Co N°34, 29 Av Du Mal De Lattre De Tassigny 29 Av Du Mal De Lattre De Tassigny

City

Nancy Cedex

Postcode

54035

Country

France

Scientific contact point

Organisation

Centre Hospitalier Regional Universitaire De Nancy

Contact name

Lemelle Jean-Louis

Public contact point

Organisation

Centre Hospitalier Regional Universitaire De Nancy

Contact name

Lemelle Jean-Louis

Locations

1 EU/EEA country · 19 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 15 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications