PDF(Pubmed)
Abstract：
UNASSIGNED: A previous phase 3 trial of prucalopride in pediatric patients (6 months-18 years old) with functional constipation (FC) demonstrated no efficacy versus placebo. We designed an additional phase 3 trial to further assess the efficacy, long-term safety and tolerability of prucalopride in children and adolescents.
UNASSIGNED: This multicenter trial (ClinicalTrials.gov identifier: NCT04759833; EudraCT number: 2022-003221-22) comprises a 12-week, randomized, double-blind, placebo-controlled phase, followed by a 36-week, double-blind, safety extension phase. Approximately 240 toilet-trained patients aged 3-17 years will be randomized 1:1:1 to receive low- (0.04 mg/kg) or high-dose (0.08 mg/kg) prucalopride, or placebo once daily. Fifteen non-toilet-trained patients ≥6 months old with FC will be included in an exploratory efficacy and safety analysis.
UNASSIGNED: The efficacy endpoints used in this study will differ from those used in adults and in the previous pediatric phase 3 trial; they have been adapted to be more suitable for a wider age range of pediatric patients. Both study phases will be longer than in the previous pediatric study, providing a longer time period in which to assess the efficacy and safety of prucalopride. Study participants will be identified using the modified Rome IV criteria for FC, instead of the Rome III criteria, and non-toilet-trained patients will be included, which will broaden the population of pediatric patients assessed. Patients will undergo fecal disimpaction before randomization and undergo standardized continuous behavioral therapy throughout the trial. This pediatric study of prucalopride will aim to demonstrate the efficacy and long-term safety of this treatment.
UNASSIGNED: This multicenter trial (ClinicalTrials.gov identifier: NCT04759833; EudraCT number: 2022-003221-22) comprises a 12-week, randomized, double-blind, placebo-controlled phase, followed by a 36-week, double-blind, safety extension phase. Approximately 240 toilet-trained patients aged 3-17 years will be randomized 1:1:1 to receive low- (0.04 mg/kg) or high-dose (0.08 mg/kg) prucalopride, or placebo once daily. Fifteen non-toilet-trained patients ≥6 months old with FC will be included in an exploratory efficacy and safety analysis.
UNASSIGNED: The efficacy endpoints used in this study will differ from those used in adults and in the previous pediatric phase 3 trial; they have been adapted to be more suitable for a wider age range of pediatric patients. Both study phases will be longer than in the previous pediatric study, providing a longer time period in which to assess the efficacy and safety of prucalopride. Study participants will be identified using the modified Rome IV criteria for FC, instead of the Rome III criteria, and non-toilet-trained patients will be included, which will broaden the population of pediatric patients assessed. Patients will undergo fecal disimpaction before randomization and undergo standardized continuous behavioral therapy throughout the trial. This pediatric study of prucalopride will aim to demonstrate the efficacy and long-term safety of this treatment.
摘要：
■先前在功能性便秘（FC）的儿科患者（6个月至18岁）中使用普卢卡必利的3期试验表明与安慰剂相比没有疗效。我们设计了一个额外的3期试验来进一步评估疗效，儿童和青少年使用普鲁卡必利的长期安全性和耐受性。
■这项多中心试验（ClinicalTrials.gov标识符：NCT04759833；EudraCT编号：2022-003221-22）为期12周，随机化，双盲,安慰剂对照阶段，接下来是36周，双盲,安全扩展阶段。大约240名3-17岁的接受过厕所训练的患者将被随机分为1：1：1，接受低剂量（0.04mg/kg）或高剂量（0.08mg/kg）的普鲁卡必利，或安慰剂每天一次。15名非厕所训练≥6个月大的FC患者将被纳入探索性疗效和安全性分析。
■本研究中使用的疗效终点与成人和先前的儿科3期试验中使用的疗效终点不同；它们已被调整为更适合更广泛年龄范围的儿科患者。这两个研究阶段都将比以前的儿科研究更长，提供更长的时间来评估普鲁卡必利的疗效和安全性。研究参与者将使用FC的修改后的罗马IV标准进行识别，而不是罗马三世的标准，并将包括非如厕训练的病人,这将扩大评估的儿科患者的人群。患者将在随机分组前进行粪便分解，并在整个试验中进行标准化的连续行为治疗。这项普鲁卡洛必利的儿科研究旨在证明这种治疗的疗效和长期安全性。
■这项多中心试验（ClinicalTrials.gov标识符：NCT04759833；EudraCT编号：2022-003221-22）为期12周，随机化，双盲,安慰剂对照阶段，接下来是36周，双盲,安全扩展阶段。大约240名3-17岁的接受过厕所训练的患者将被随机分为1：1：1，接受低剂量（0.04mg/kg）或高剂量（0.08mg/kg）的普鲁卡必利，或安慰剂每天一次。15名非厕所训练≥6个月大的FC患者将被纳入探索性疗效和安全性分析。
■本研究中使用的疗效终点与成人和先前的儿科3期试验中使用的疗效终点不同；它们已被调整为更适合更广泛年龄范围的儿科患者。这两个研究阶段都将比以前的儿科研究更长，提供更长的时间来评估普鲁卡必利的疗效和安全性。研究参与者将使用FC的修改后的罗马IV标准进行识别，而不是罗马三世的标准，并将包括非如厕训练的病人,这将扩大评估的儿科患者的人群。患者将在随机分组前进行粪便分解，并在整个试验中进行标准化的连续行为治疗。这项普鲁卡洛必利的儿科研究旨在证明这种治疗的疗效和长期安全性。
