Spina bifida Trials in Disease

Study on the Effectiveness of Oxybutynin for Children with Overactive Neurogenic Bladder Due to Spina Bifida

This clinical trial is investigating a specific treatment approach for children who experience neurogenic bladder as a result of spina bifida. Neurogenic bladder is a condition where the nerves controlling the bladder do not function properly, causing problems such as frequent urination or difficulty holding urine. The study focuses on children who have not responded well to standard oral medications.

Main inclusion criteria:

• Children between 6 and 17 years of age
• Confirmed diagnosis of overactive bladder due to spina bifida, verified by urodynamic testing performed within the last 6 months
• Previous unsuccessful treatment with anticholinergic medications taken by mouth for at least 4 weeks, or inability to tolerate these oral medications due to side effects
• Currently performing intermittent catheterization (using a tube to empty the bladder) at least three times daily for a minimum of 6 weeks
• Recent kidney ultrasound completed within the last 2 months
• Recent bladder function test (cystomanometry) completed within the last 6 months
• Parent or caregiver willing and able to perform the treatment procedure at home
• Covered by a social security plan

Main exclusion criteria:

• Children not performing intermittent catheterization regularly
• Children without an overactive neurogenic bladder
• Children whose condition is effectively managed with oral anticholinergic medications
• Children outside the specified age range
• Children without a confirmed diagnosis of spina bifida

Focus and goal of the trial:

The study tests a different method of delivering oxybutynin hydrochloride by placing the medication directly into the bladder through a catheter, rather than taking it by mouth. This approach is called intravesical instillation. The main goal is to determine whether this direct delivery method can increase the bladder's capacity to hold urine and reduce symptoms like frequent urination and incontinence in children who have not benefited from oral treatments.

The trial is designed as a comparison study where some children receive the oxybutynin treatment while others receive a placebo. The treatment period lasts for four weeks, during which researchers carefully monitor changes in bladder function and any side effects. Parents or caregivers are taught how to perform the instillation procedure at home, making it a practical option for ongoing management if proven effective.

Throughout the study, participants undergo regular check-ups to assess their bladder's ability to hold urine, the pressure within the bladder, and overall health. Researchers also evaluate how well children tolerate the treatment, watching for potential side effects that may affect digestion, neurological function, or skin. By the end of the four-week period, the study team conducts a final assessment to measure improvements in bladder capacity and reductions in incontinence episodes.

Investigational drug:

The medication being tested is oxybutynin hydrochloride, marketed as VESOXX 1 mg/ml solution. Oxybutynin belongs to a class of medications called anticholinergics, which work by blocking certain nerve signals that cause the bladder muscles to contract involuntarily. When delivered directly into the bladder, the medication can relax the bladder muscles locally, potentially increasing how much urine the bladder can hold and reducing the frequency of urination. This intravesical approach is being studied specifically for cases where taking oxybutynin by mouth has not worked well or has caused unacceptable side effects.