OXYBUTYNIN HYDROCHLORIDE for Spina bifida|Neurogenic bladder dysfunction

Inclusion criteria

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

Exclusion criteria

• {"criterion_text":"- Person displaying known allergy to one of the components of evaluated product (notably oxybutynin)."}
• {"criterion_text":"- Person with congestive cardiac failure"}
• {"criterion_text":"- Person with cardiac arrhythmia"}
• {"criterion_text":"- Person with tachycardia"}
• {"criterion_text":"- Person with uncontrolled hypertension"}
• {"criterion_text":"- Person under one of the following treatments : Bisphosphonates; Cytochrome P450 Inhibitors (such as ketoconazole and Erythromycin); Cholinesterase inhibitors."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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)."}
• {"criterion_text":"- Pregnant, parturient or breastfeeding woman."}
• {"criterion_text":"- Person deprived of liberty for judicial or administrative decision."}
• {"criterion_text":"- Person under psychiatric care as referred in articles L. 3212-1 and L. 3213-1."}
• {"criterion_text":"- Intradetrusor injection of botulinum toxin less than 6 months before."}
• {"criterion_text":"- Person with hyperthyroidism"}
• {"criterion_text":"- Person with coronary cardiac disease"}

Primary endpoints

• {"endpoint_text":"- Evolution of maximal bladder capacity at 4 weeks of treatment (end of follow-up).","definition_or_measurement_approach":"Measured by urodynamic assessment/cystomanometry of maximal bladder capacity at 4 weeks of treatment (urodynamic check-up described in inclusion criteria)."}

Secondary endpoints

• {"endpoint_text":"- Evolution of maximal bladder pressure at 4 weeks of treatment (end of follow-up).","definition_or_measurement_approach":"Measured by urodynamic assessment/cystomanometry of maximal bladder pressure at 4 weeks."}
• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Time-to-event within a 28-day timeframe; treatment failure defined as at least one of: treatment judged non-effective by patient/practitioner; urinary incontinence reduction <50% vs initial bladder diary; intolerable side effects as reported by patient."}
• {"endpoint_text":"- Evaluation of tolerance and side effects: digestive, psychiatric, neurological, cutaneous, urological, pain associated with instillation.","definition_or_measurement_approach":"Assessment of adverse events and tolerability across specified systems (digestive, psychiatric, neurological, cutaneous, urological) and instillation-related pain."}
• {"endpoint_text":"- Proportion of responders at 4 weeks of treatment (patients who had at least a 50% reduction in urinary incontinence episodes).","definition_or_measurement_approach":"Responder defined as ≥50% reduction in urinary incontinence episodes measured by bladder diary at 4 weeks."}
• {"endpoint_text":"- Proportion of continent patients at 4 weeks of treatment (patients who had a 100% reduction in urinary incontinence episodes).","definition_or_measurement_approach":"Continent patients defined as 100% reduction in urinary incontinence episodes measured by bladder diary at 4 weeks."}
• {"endpoint_text":"- Product usability measured with usability questionnaire (UMUX-LITE and specific questions).","definition_or_measurement_approach":"Usability assessed using UMUX-LITE questionnaire and additional study-specific questions."}
• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Quality of life measured by ICIQ-UI-SF and KIDSCREEN-10 scores; standardized difference between baseline and end of study."}
• {"endpoint_text":"- 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).","definition_or_measurement_approach":"Bladder diary metrics: number and volume of catheterizations over 72 hours in week before each visit (V1, V2, V3)."}
• {"endpoint_text":"- Evolution of the elements of the other urodynamic assessments at 4 weeks of treatment: Bladder compliance; Minimum filling volume causing uninhibited detrusor contraction.","definition_or_measurement_approach":"Urodynamic parameters such as bladder compliance and minimal filling volume triggering uninhibited detrusor contraction measured at 4 weeks."}
• {"endpoint_text":"- Evolution of renal ultrasonography at 4 weeks of treatment: Renal pelvis anteroposterior diameter; Ureters diameter.","definition_or_measurement_approach":"Renal ultrasound measures including renal pelvis anteroposterior diameter and ureter diameter assessed at 4 weeks."}

France

Earliest CTIS Part Ii Submission Date

19-12-2024

Latest Decision Or Authorization Date

19-03-2026

Processing Time Days

455

Number Of Sites

19

Number Of Participants

60

Primary sponsor

Full Name

Centre Hospitalier Regional Universitaire De Nancy

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France

Third parties

• {"country":"","full_name":"French Ministry of Health","duties_or_roles":"Source of monetary support","organisation_type":""}
• {"country":"","full_name":"FARCO PHARMA","duties_or_roles":"Source of monetary support","organisation_type":""}