Efficacy, Neural Repair Mechanism, and Health Economics of a Multimodal Treat-to-Target Strategy Incorporating Vitamin D for Pediatric Overactive Bladder-Wet: A Randomized Controlled Trial
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 180
- 试验地点
- 1
- 主要终点
- Lower urinary tract symptom score
研究概览
简要总结
The purpose of this clinical trial is to investigate whether adding high-dose vitamin D (2,400 IU daily) to standard medical treatment (solifenacin combined with behavioral therapy) is more effective than standard treatment alone for children with overactive bladder-wet (OAB-wet).
OAB-wet causes sudden urinary urges and frequent daytime or incontinence, which significantly impacts a child's quality of life and increases the family's caregiving burden. While solifenacin is a standard medication used to calm the bladder, many children do not achieve complete dryness. This study introduces the "Treat-to-Target" (T2T) approach, where clinicians and families set personalized "functional goals" (such as zero leakage) and monitor progress closely to adjust care.
The study aims to answer the following questions:
Does adding vitamin D help more children achieve their goal of "zero leakage" compared to standard treatment? Does vitamin D help repair bladder-related nerves, as measured by a specific marker in the urine? Does this combined approach reduce the family's expenses (like laundry costs and diaper use) and improve the child's self-esteem?
Participants will be randomly assigned to one of two groups for 12 weeks:
Intervention Group: Standard care (solifenacin + behavioral therapy) plus daily vitamin D (2,400 IU).
Control Group: Standard care (solifenacin + behavioral therapy) alone. Researchers will evaluate symptoms, vitamin D levels, and nerve repair markers at 6 and 12 weeks to determine the best treatment strategy for these children.
详细描述
This randomized controlled trial utilizes a "Treat-to-Target" (T2T) framework to evaluate the incremental value of high-dose vitamin D supplementation as an add-on therapy to standard anticholinergic treatment (solifenacin) for pediatric idiopathic overactive bladder-wet (OAB-wet).
This study shifts the focus from simple symptom reduction to achieving predefined, patient-centered functional goals. While solifenacin targets the muscarinic receptors to inhibit detrusor overactivity (symptomatic relief), vitamin D is hypothesized to act as a neuromodulator that promotes neural repair-specifically the remyelination of pelvic nerves involved in bladder control.The study will enroll children with OAB-wet and documented vitamin D insufficiency/deficiency. The intervention group receives a multimodal start consisting of solifenacin, standard behavioral therapy (SU), and 2,400 IU/day of vitamin D3. The control group receives standard-of-care (solifenacin + SU).Key components of the study include:Goal Setting: At Week 0, clinicians and caregivers will establish a specific clinical target.Monitor and Adjust: At Week 6, serum 25(OH)D levels and voiding diaries will be reviewed to assess the trajectory toward the clinical target and the biochemical threshold of 40 ng/mL.Mechanism Validation: Urinary Myelin Basic Protein (uMBP) corrected by creatinine will be measured at baseline and Week6&12 to provide evidence of structural neural repair.Value-Based Assessment: A health economic evaluation will compare the total societal cost (medication costs vs. reduced caregiver burden and laundry expenses) between the two strategies.
By comparing these two pathways, the study aims to demonstrate that a multimodal T2T strategy incorporating vitamin D offers superior clinical achievement rates and better long-term functional outcomes than conventional pharmacological approaches.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children older than or equal to 5 years of age with a diagnosis of wet OAB (the diagnosis followed the latest guidelines of ICCS) attending the outpatient clinic of the Department of Urology of the Affiliated Children's Hospital of Chongqing Medical University,
- •children with serum vitamin D levels below 35 ng/ml as indicated by the tests conducted by the hospital
- •children whose guardians have given their informed consent, are able to ensure compliance and have signed a paper-based informed consent form.
排除标准
- •Those with urinary malformations or serious diseases (e.g., hypospadias, cryptorchidism, posterior urethral valvulae, vesicoureteral reflux, neurogenic bladder, urinary tumors, urinary stones, bladder and urethral injuries, etc.)
- •Those with neurological disorders (e.g., epilepsy, spinal cord injuries, spinal dysplasia, spinal embolism syndrome, multiple sclerosis, and autism spectrum disorders, etc.)
- •People with serious heart disease, abnormal liver and kidney function, lung disease, bone deformity, serious digestive tract disease, genetic metabolic disease
- •People with history of gastrointestinal surgery and urological surgery
- •People with dry stools and long-term constipation
- •People who are taking anticonvulsant and antiepileptic drugs, hormones, and anti-tuberculosis drugs
- •People with history of hypercalcemia, hyperphosphatemia with renal rickets
- •People who have had unexplained hematuria and hematuria with renal rickets within the last year
- •People who have been suffering from severe heart disease or chronic diabetes.
- •participation in other clinical studies at the time of consultation or during the follow-up period of other clinical studies
- •unwillingness to participate in this study
研究组 & 干预措施
Standard-of-care
Standard Urotherapy plus Solinasine succinate 5mg once daily with a maximum dose of 10mg/day
干预措施: urotherapy (Behavioral)
Multimodal strategy
A multimodal start consisting of solifenacin, standard behavioral therapy (SU), and 2,400 IU/day of vitamin D3
干预措施: urotherapy (Behavioral)
Multimodal strategy
A multimodal start consisting of solifenacin, standard behavioral therapy (SU), and 2,400 IU/day of vitamin D3
干预措施: Vitamin D3 (Drug)
Standard-of-care
Standard Urotherapy plus Solinasine succinate 5mg once daily with a maximum dose of 10mg/day
干预措施: Solifenacin Succinate (Drug)
结局指标
主要结局
Lower urinary tract symptom score
时间窗: week 1, week6，week 12
Lower urinary tract symptom scale score
urinary incontinence frequency
时间窗: week 1, week6，week 12
The number of daily occurrences of incontinence recorded through a bladder diary
Clinical Target Achievement Rate at 12 Weeks
时间窗: week 12
Percentage of participants who achieve their pre-defined personalized clinical goal set at Week 0.
次要结局
- Frequency of urination per day(week 1, week6，week 12)
- Qol score(week 1, week6，week 12)
- Change in Weekly Urinary Incontinence (UI) Frequency(Baseline, 6 weeks, 12 weeks.)
- Change in Pediatric Incontinence Questionnaire (PIN-Q) Score(Baseline, 12 weeks.)
- Change in Pediatric Lower Urinary Tract Symptom (PLUTS) Score(Baseline, 6 weeks, 12 weeks.)
- Health Economics: Total Household Burden Cost(12 weeks)
- Change in Urinary Myelin Basic Protein (uMBP)/Creatinine Ratio(Baseline, 12 weeks.)
研究者
Xing Liu
Professor, Doctor
Children's Hospital of Chongqing Medical University