NL-OMON49029已完成2 期
A Phase IIb double-blind, randomised, placebo-controlled, multi-centre, confirmative three-way cross-over study on cognitive function with two doses of KH176 in subjects with a genetically confirmed mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation. - KHENERGYZE
Khondrion B.V.0 个研究点目标入组 9 人开始时间: 待定最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 9
研究概览
简要总结
Trial is onging in other countries
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Males and females aged 18 years or older at screening.
- •2. Ability and willingness to provide written Informed Consent prior to
- •screening evaluations.
- •3. Confirmed mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation (heteroplasmy >=
- •20%, urinary epithelial cells).
- •4. Positive NMDAS score >10 at Screening.
- •5. Three or more clinical features, with no other causative unifying
- •diagnosis, found to commonly occur in subjects with a m.3243A>G mutation:
- •- Deafness
- •- Developmental delay
- •- Diabetes Mellitus
- •- Epilepsy
- •- Gastrointestinal complaints
- •- Progressive External Ophtalmoplegia (PEO) and retinopathy
- •- Exercise intolerance
- •- Migraine (with or without aura), specified by at least five attacks
- •fulfilling diagnostic criteria B-D:
- •B. Headache attacks lasting 4-72 hours (untreated or unsuccessfully treated)
- •C. Headache has at least two of the following four characteristics:
- •1. unilateral location
- •2. pulsating quality
- •3. moderate or severe pain intensity
- •4. aggravation or causing avoidance of routine physical activity (e.g.
- •walking or climbing stairs)
- •D. During headache at least one of the following:
- •1. nausea and/or vomiting
- •2. photophobia and phonophobia
- •6. Attentional dysfunction score (Cogstate Identification test) >= 0.2 standard
- •deviations poorer than healthy controls at Screening.
- •7. Disease appropriate physical and mental health as established at Screening
- •by medical history, physical examination, ECG and vital signs recording, and
- •results of clinical chemistry and haematology testing as judged by the
- •investigator.
- •8. Objectified Left Ventricular Ejection Fraction (LVEF) >=45%
- •(echocardiography, or otherwise).
- •9. Left Ventricular (LV) wall thickness <=15 mm.
- •10. Left atrium dilatation <= 40 mL/m2.
- •Note: No need to test LV parameters (criteria #8, #9, #10) if favorable
- •echocardiography (or otherwise) results dated less than 6 months prior to
- •Screening are available.
- •11. Women of childbearing potential must be willing to use adequate
- •contraceptive methods during the entire study, i.e., a hormonal contraceptive
- •method (pill, vaginal ring, patch, implant, injectable, hormone-medicated
- •intrauterine device) or an intrauterine device. Sexual abstinence is an
- •acceptable contraceptive method only as true abstinence: when this is in line
- •with the preferred and usual lifestyle of the patient. [Periodic abstinence
- •(e.g., calendar, ovulation, symptothermal, post-ovulation methods) and
- •withdrawal are not acceptable methods of contraception].
- •Note 1: Natural family planning methods, female condom, cervical cap or
- •diaphragm are not considered adequate contraceptive methods in the context of
- 另有 10 项未显示
排除标准
- •1. Surgery of gastro-intestinal tract that might interfere with absorption.
- •2. Treatment with an investigational product within 3 months or 5 times the
- •half-life of the investigational product (whichever is longer) prior to the
- •first dose of the study medication.
- •3. Documented history of ventricular tachycardia (HR>110 beats/min).
- •4. History of acute heart failure, (family) history of unexplained syncope or
- •congenital long and short QT syndrome or sudden death.
- •5. Clinically relevant abnormal laboratory, vital signs or physical or mental
- •a) Aspartate aminotransferase (ASAT) or alanine aminotransferase (ALAT) > 3 x
- •upper limit of normal (ULN), or bilirubin > 3 x ULN at screening. If a patient
- •has ASAT or ALAT > 3 x ULN but < 3.5 x ULN, re-assessment is allowed at the
- •investigator*s discretion.
- •b) Estimated glomerular filtration rate <= 60 mL/min according to the CKD-EPI
- •formula at screening.
- •c) Systolic Bloodpressure > 150 mmHg at screening or baseline.
- •d) All other clinically relevant parameters at screening or baseline as judged
- •by the Investigator.
- •6. Clinically relevant abnormal ECG or cardiac functioning, defined as
- •ST-segment elevation >1 mm in I, II, III, aVL, aVF ,V3 ,V4 ,V5 ,V6; >2 mm in
- •V1, V2; QTc >450 ms for male subjects; QTc > 470ms for female subjects (local,
- •machine read), T-top inversion in >1 consecutive lead.
- •7. Serum Hyper-potassium (> 5.0 mEq/L).
- •8. Serum Hypo-potassium (< 3.5 mEq/L).
- •9. History of ischemic heart disease.
- •10. Symptomatic heart failure.
- •11. Clinically relevant aorta and/or mitralis valvular defect as judged by the
- •investigator.
- •12. Pregnancy or breast feeding (females).
- •13. Poor nutritional state as judged by the investigator.
- •14. History of hypersensitivity or idiosyncrasy to any of the components of the
- •investigational drug.
- •15. Medical history of drug abuse (illegal drugs such as cannabinoids,
- •amphetamines, cocaine, opiates or problematic use of prescription drugs such as
- •benzodiazepines, opiates).
- •16. The use of any of the following medication and/or supplements within 4
- •weeks or 5 times the half-life (whichever is longer) prior to the first dosing
- •of the study medication:
- •a. (multi)vitamins, co-enzyme Q10, Vitamine E, riboflavin, and anti-oxidant
- •supplements (including, but not limited to idebenone/EPI-743, mitoQ); unless
- •stable for at least one month before first dosing and remaining stable
- •throughout the study.
- •b. any medication negatively influencing mitochondrial functioning (including
- •but not limited to valproic acid, glitazones, statins, anti-virals, amiodarone,
- •and non-steroidal anti-inflammatory drugs (NSAIDs)), unless stable for at least
- •one month before first dosing and remaining stable throughout the study.
- •Note: thus, mitoQ and any medication negatively influencing mitochondrial
- •functioning are allowed as long as the dose has been stable for at least one
- •month prior to first dosing and remains stable throughout the study.
- •c. any strong Cytochrome P450 (CYP)3A4 inhibitors (all *conazoles-
- •anti-fungals*, HIV antivirals, grapefruit).
- 另有 5 项未显示
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