一项随机安慰剂对照、双盲二期研究，旨在探索 sonlicromanol 在患有遗传性线粒体疾病和运动症状的儿童中的安全性、疗效和药代动力学（“KHENERGYC”）。
A randomised placebo-controlled, double-blind phase II study to explore the safety, efficacy, and pharmacokinetics of sonlicromanol in children with genetically confirmed mitochondrial disease and motor symptoms ("KHENERGYC").
机构信息
Khondrion BV, Transistorweg 5C, M Building, 6534, AT, Nijmegen, The Netherlands.
Radboud Center for Mitochondrial Medicine, Department of Pediatrics, Radboud University Medical Center Nijmegen, Geert Grooteplein Zuid 10, 6500 HB, Nijmegen, The Netherlands.
出版信息
BMC Neurol. 2022 Apr 27;22(1):158. doi: 10.1186/s12883-022-02685-3.
BACKGROUND
METHODS: The KHENERGYC trial will be a phase II, randomised, double-blinded, placebo-controlled (DBPC), parallel-group study in the paediatric population (birth up to and including 17 years). The study will be recruiting 24 patients suffering from motor symptoms due to genetically confirmed PMD. The trial will be divided into two phases. The first phase of the study will be an adaptive pharmacokinetic (PK) study with four days of treatment, while the second phase will include randomisation of the participants and evaluating the efficacy and safety of sonlicromanol over 6 months.
DISCUSSION
Effective novel therapies for treating PMDs in children are an unmet need. This study will assess the pharmacokinetics, efficacy, and safety of sonlicromanol in children with genetically confirmed PMDs, suffering from motor symptoms.
TRIAL REGISTRATION
clinicaltrials.gov: NCT04846036 , registered April 15, 2021. European Union Clinical Trial Register (EUDRACT number: 2020-003124-16 ), registered October 20, 2020. CCMO registration: NL75221.091.20, registered on October 7, 2020.
背景
方法：KHENERGYC 试验将是一项针对儿科人群（出生至 17 岁）的 II 期、随机、双盲、安慰剂对照（DBPC）、平行组研究。该研究将招募 24 名患有遗传性 PMD 导致运动症状的患者。该试验将分为两个阶段。第一阶段是为期四天的适应性药代动力学（PK）研究，第二阶段将包括参与者的随机分组，并评估 sonlicromanol 在 6 个月内的疗效和安全性。
讨论
治疗儿童 PMD 的有效新型疗法是未满足的需求。本研究将评估 sonlicromanol 在患有运动症状的遗传性 PMD 儿童中的药代动力学、疗效和安全性。
试验注册
clinicaltrials.gov：NCT04846036，于 2021 年 4 月 15 日注册。欧盟临床试验注册处（EUDRACT 编号：2020-003124-16），于 2020 年 10 月 20 日注册。CCMO 注册：NL75221.091.20，于 2020 年 10 月 7 日注册。
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