2023-508218-41-00招募中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 18 years of Age with Autosomal Recessive Polycystic Kidney Disease (ARPKD).
Otsuka Pharmaceutical Development & Commercialization Inc.9 个研究点 分布在 4 个国家目标入组 7 人开始时间: 2024年5月6日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 7
- 试验地点
- 9
- 主要终点
- Safety assessments which will be summarized by descriptive statistics, and the endpoints will be • Adverse events • Vital signs • Clinical laboratory assessments
研究概览
简要总结
To evaluate the safety of tolvaptan in pediatric subjects with ARPKD
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Male or female subjects between 28 days and less than 18 years of age, with clinical features that are consistent with a diagnosis of ARPKD.
- •Ability for parent/legal 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. Ability to provide written informed assent from all subjects old enough per local laws to provide assent.
排除标准
- •Premature birth (≤ 32 weeks gestational age) for infants 28 days to < 12 weeks of age.
- •Has or at risk of having significant hypovolemia (eg, subjects that lack free access to water [inability to respond to thirst, depending on age], without adequate fluid monitoring and management) as determined by investigator.
- •Clinically significant anemia, as determined by investigator.
- •Platelets < 50000 µL.
- •Severe systolic dysfunction defined as ejection fraction < 14%.
- •Taking any other experimental medications.
- •Require ventilator support.
- •Taking medications known to induce CYP3A
- •Having an active infection including viral that would require therapy disruptive to IMP dosing.
- •Females who are breast-feeding or who have a positive pregnancy test result prior to receiving IMP.
- •Subjects with a history of substance abuse within the last 6 months (depending on age).
- •Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation.
- •Subjects who have bladder dysfunction and/or difficulty voiding.
- •Subjects taking a vasopressin agonist (eg, desmopressin).
- •Subjects with a history of persistent noncompliance with antihypertensive or other important medical therapy.
- •Subjects having concomitant illnesses or taking medications likely to confound endpoint assessments, including taking approved (ie, marketed) therapies for the purpose of affecting PKD cysts such as tolvaptan, vasopressin antagonists, anti-sense RNA therapies, rapamycin, sirolimus, everolimus, or somatostatin analogs (ie, octreotide, sandostatin).
- •Subjects who do not agree to remain abstinent or assent to use a combination of 2 of the following highly effective birth control methods for at least 28 days before the first dose of IMP, during the trial (including during IMP dose interruptions), and for at least 30 days after the last dose of IMP.
- •Received or are scheduled to receive a liver transplant.
- •History of cholangitis within the last 6 months.
- •Has findings consistent with clinically significant portal hypertension (eg, varices, variceal bleeding, hypersplenism indicated by thrombocytopenia).
- •Evidence of syndromic conditions associated with renal cysts (other than ARPKD).
- •Abnormal liver function tests including ALT and AST, > 1.2 × ULN.
- •Has splenomegaly or portal hypertension.
- •Parents with renal cystic disease.
- •Receiving chronic diuretic that could not be adjusted after tolvaptan initiation.
- •Cannot be monitored for fluid balance.
- •Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator.
结局指标
主要结局
Safety assessments which will be summarized by descriptive statistics, and the endpoints will be • Adverse events • Vital signs • Clinical laboratory assessments
Safety assessments which will be summarized by descriptive statistics, and the endpoints will be • Adverse events • Vital signs • Clinical laboratory assessments
Serum transaminase elevations for frequency (2 ×, 3 ×, 5 × and 10 × ULN), time to onset, time to peak levels, time of offset (< 3 ×, 2 ×, or 1 × ULN), response to de challenge and re-challenge and frequency of progression to Hy’s laboratory criteria (ALT or AST > 3 × ULN and BT, > 2 × ULN without alkaline phosphatase ≥2 × ULN) • Change from baseline in sNa+
Serum transaminase elevations for frequency (2 ×, 3 ×, 5 × and 10 × ULN), time to onset, time to peak levels, time of offset (< 3 ×, 2 ×, or 1 × ULN), response to de challenge and re-challenge and frequency of progression to Hy’s laboratory criteria (ALT or AST > 3 × ULN and BT, > 2 × ULN without alkaline phosphatase ≥2 × ULN) • Change from baseline in sNa+
次要结局
- Time to RRT
- Annual rate of change of eGFR (eGFR Schwartz formula = 0.413 × height [or length, cm] /serum creatinine mg/dL) from baseline to post‑treatment after 18 months of treatment.
- Change from baseline of eGFR (eGFR Schwartz formula = 0.413 × height [or length, cm] /serum creatinine mg/dL) while on treatment at Months 1, 6, 12, and 18.
- Percentage of subjects who receive RRT
- Percentage of change in kidney size of height adjusted TKV at every time point from baseline to 18 months on treatment via ultrasound using ellipsoid methodology.
- Change from baseline (last assessment prior to dosing) in growth percentile trajectories for height (stature), weight, and head circumference (for subjects ≤ 2 years or age) at 3 months, 6 months, 12 months, and 18 months.
- Age-appropriate assessment of palatability and acceptability of suspension formulation for applicable subjects.
- Proportions of each Tanner Staging by gender and age at baseline and every 6 months until Month 18/EoTx.
研究者
EU Clinical Trials Helpdesk
Scientific
Otsuka Pharmaceutical Development & Commercialization Inc.
研究点 (9)
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