12-week, Multicenter, Open-label, Non-comparative Study to Investigate Pharmacodynamic and Safety of Alfuzosin 0.2 mg/kg/Day in the Treatment of Children and Adolescents 2 - 16 Years of Age With Hydronephrosis Associated With Elevated Detrusor Leak Point Pressure of Neuropathic Etiology Followed by a 40-week Open-label Extension
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Sanofi
- 入组人数
- 25
- 试验地点
- 2
- 主要终点
- Number of Participants With a Decrease From Baseline ≥ 1 in the Society of Fetal Urology (SFU) Grade of Hydronephrosis
研究概览
简要总结
Primary objective was to determine efficacy of Alfuzosin in the treatment of children and adolescents 2-16 years of age with newly diagnosed or progressive hydronephrosis due to elevated detrusor Leak Point Pressure [LPP] of neuropathic etiology.
Secondary objectives were:
- To investigate the safety and tolerability of alfuzosin 0.2 mg/kg/day in children and adolescents,
- To investigate the number of Urinary Tract Infection (UTI) episodes,
- To investigate the pharmacokinetics of Alfuzosin (population kinetics).
详细描述
The study consisted of 2 phases:
- a 12-week efficacy phase then,
- a 40-week safety extension phase.
All eligible subjects received alfuzosin 0.2 mg/kg/day. The formulation and the frequency was assigned by Interactive Voice Response System (IVRS) according to age group and ability to swallow tablets.
Patients who completed the 12-week open-label treatment period were offered to continue in the 40-week open-label safety extension study. The treatment was the same as in the 12-week efficacy phase.
All patients had a one-week follow-up period after the last dose intake.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children and adolescents of either gender 2 - 16 years of age with a detrusor Leak Point Pressure (LPP) of 40 cm water or greater and with newly diagnosed or progressive hydronephrosis either Society of Fetal Urology (SFU) grade 1, 2 or 3 due to neuropathic bladder dysfunction.
排除标准
- •Hydronephrosis of non-neuropathic etiology.
- •Urological surgery in the last 4 months prior to the study.
- •Urethral dilatation in the last 3 months prior to the baseline urodynamic assessment.
- •α-blocker therapy in the last 4 weeks prior to the baseline urodynamic assessment.
- •Detrusor injections of botulinum toxin in the last 6 months.
- •Urological diseases/conditions other than functional bladder obstruction of neuropathic etiology, that can lead to upper urinary tract dilatation (e.g., bladder anomalies, ureterocele).
- •History of intolerance to α-blocker therapy.
- •Orthostatic hypotension.
- •History of risk factors for Torsade de pointes (e.g., family history of Long QT Syndrome).
- •The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
研究组 & 干预措施
Alfuzosin solution - 2-7 years
Alfuzosin solution, daily dose divided in 3 doses given at breakfast, lunch and dinner to children 2-7 years of age.
干预措施: Alfuzosin (Drug)
Alfuzosin solution - 8-16 years
Alfuzosin solution, daily dose divided in 3 doses given at breakfast, lunch and dinner to children and adolescents 8-16 years of age who were not able to swallow tablets or preferred to take the solution or had a body weight < 30 kg.
干预措施: Alfuzosin (Drug)
Alfuzosin tablet - 8-16 years
Alfuzosin tablet, daily dose divided in 2 doses given at breakfast and dinner to children and adolescents 8-16 years of age who were able to swallow tablets and had a body weight ≥ 30 kg.
干预措施: Alfuzosin (Drug)
结局指标
主要结局
Number of Participants With a Decrease From Baseline ≥ 1 in the Society of Fetal Urology (SFU) Grade of Hydronephrosis
时间窗: baseline and 12 weeks (efficacy study phase)
Hydronephrosis was investigated by ultrasound and graded using SFU classification at each time point. 'Complete response' was assessed when bilateral hydronephrosis at baseline and grade decrease from baseline ≥ 1 for both kidneys, or, unilateral hydronephrosis at baseline and grade decrease from baseline ≥ 1 for the affected kidney without worsening of the other kidney. 'Partial response' was assessed when bilateral hydronephrosis at baseline and grade decrease from baseline ≥ 1 for one kidney without worsening of the other kidney.
次要结局
- Number of Participants With Symptomatic Urinary Tract Infection (UTI) Episodes(52 weeks (efficacy and extension study phases))