EUCTR2016-001696-79-NL进行中（未招募）1 期
A double-blind, randomized, placebo-controlled, single-center, two-way cross-over study with KH176 in patients with the mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation and clinical signs of mitochondrial disease - The KHENERGY study
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Khondrion BV
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Participants in the trial will be patients with a mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation and clinical signs of mitochondrial disease, including but not limited to MELAS, MIDD and mixed types.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 20
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.Motoric abnormalities other than related to the mitochondrial disease interfering with the outcome parameters.
- •2.CPEO patients with clinical signs and symptoms restricted to the eye only
- •3.Heteroplasmy level as measured in urine < 20%
- •4.Poor nutritional state as judged by the investigator
- •5.Body Mass Index (BMI) not within 18.0-30.0 kg/m2 at screening.
- •6.History of cancer
- •7.Surgery or active illness of gastro-intestinal tract that might interfere with absorption.
- •8.Participation in a trial of an investigational product in the preceding 3 months prior to the first dose or during this trial.
- •9.Positive drug, alcohol or cotinine test at screening and/or admission (Day 1 of the first dosing period).
- •10.Clinically relevant abnormal laboratory, ECG recordings, cardiac echo (within 1 year prior to screening), vital signs or physical or mental findings at screening as judged by the Investigator.
- •11.Clinically relevant abnormal ECG or cardiac functioning as judged by a cardiologist.
- •12.ECG: QTc > 450 ms, abnormal T-wave
- •13.Symptomatic heart failure or signs of ischemic heart disease
- •14.Left Ventricular Ejaction Fraction <45%
- •15.History or family history of congenital Long QT syndrome
- •16.Increased or decreased potassium (local laboratory normal range)
- •17.Inadequate contraception use, pregnancy or breast feeding (females)
- •18.Clinically significant presence or history of allergy as judged by the Investigator.
- •19.History of hypersensitivity or idiosyncrasy to any of the components of the investigational drug.
- •20.Within 4 weeks prior to dosing, the use of (multi)vitamins, co-enzyme Q10, Vitamine E, riboflavin, and anti-oxidant supplements (and idebenone/EPI-743), as well as any medication negatively influencing mitochondrial functioning (including but not limited to valproic acid, glitazones, statins, anti-virals, amiodarone, and NSAID’s) as well as any strong Cytochrome P450 inhibitors (all ‘conazoles-anti-fungals’, HIV antivirals, grapefruit) and strong Cytochrome P450 inducers (a.o. carbamazepine, phenobarbital, phenytoin, rifampicine, St Johns wort, pioglitazone, troglitazone) as well as any medication known to affect cardiac repolarization (all anti-psychotics, several anti-depressants: nor/amytriptilline, fluoxetine, anti-emetics: domperidone (motilium) granisetron, ondansetron)
研究者
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