NCT04786574撤回3 期
A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 12 Weeks of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 入组人数
- 20
- 主要终点
- The percentage of subjects that will have Renal Replacement Therapy (RRT) by 1 year of age.
研究概览
简要总结
The primary objective of this study is to evaluate the effect of tolvaptan on the need for renal replacement therapy in pediatric subjects with autosomal recessive polycystic kidney disease (ARPKD)
详细描述
Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 3), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV).
The trial will be the first trial of tolvaptan in a pediatric ARPKD population.
Participants in this study will be assigned to tolvaptan for 24 months and closely monitored over the course of the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Weeks 至 12 Weeks（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects between 28 days and < 12 weeks of age, inclusive at the time of enrollment.
- •Must have clinical and imaging features that are consistent with a diagnosis of ARPKD with all the following characteristics:
- •Nephromegaly (> 2 standard deviations from age-appropriate standard via ultrasound)
- •Multiple renal cysts
- •History of oligohydramnios or anhydramnios
- •Ability for parent or guardian to provide written, informed consent prior to initiation of any trial-related procedures, and ability, in the opinion of the principal investigator, to comply with all the requirements of the trial.
排除标准
- •Premature birth (≤ 32 weeks gestational age)
- •Anuria or RRT, defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation
- •Evidence of syndromic conditions associated with renal cysts (other than ARPKD)
- •Abnormal liver function tests including ALT and AST, > 1.2 × ULN
- •Parents with renal cystic disease
- •Need for chronic diuretic use
- •Cannot be monitored for fluid balance
- •Has or at risk of having sodium and potassium electrolyte imbalances
- •Has or at risk of having significant hypovolemia as determined by investigator
- •Clinically significant anemia, as determined by investigator
- •Severe systolic dysfunction defined as ejection fraction < 14%
- •Serum sodium levels < 130 mmol/L or >145 mmol/L
- •Taking any other experimental medications
- •Require ventilator support
- •Taking medications known to induce CYP3A4
- •Having an infection including viral that would require therapy disruptive to IMP dosing
- •Platelet count <50,000 µL
- •Significant Portal Hypertension
- •Bladder dysfunction or difficulty voiding
- •Taking vasopressin agonist
- •Having concomitant illness or taking medications that are likely to confound endpoint assessments.
- •History of cholangitis
- •Received or scheduled to receive a liver transplant
研究组 & 干预措施
Tolvaptan (OPC-41061)
Experimental
干预措施: Tolvaptan (OPC-41061) (Drug)
结局指标
主要结局
The percentage of subjects that will have Renal Replacement Therapy (RRT) by 1 year of age.
时间窗: From Enrollment to 1 year of age
次要结局
- Palatability of the suspension formulation as assessed by a parent questionnaire immediately after and within 15-20 minutes after the first oral dose(From Enrollment to 2 years of age)
- Rate of change of eGFR by Schwartz formula from pre-treatment to after 2 years of treatment(From Enrollment to 2 years of age)
- Acceptance of the suspension formulation as assessed by a parent questionnaire immediately after and within 15-20 minutes after the first oral dose(From Enrollment to 2 years of age)
研究者
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