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临床试验/NL-OMON52925
NL-OMON52925已完成2 期

A Phase IIb open-label, multi-centre, extension study to explore the long-term safety and efficacy of KH176 in subjects with a genetically confirmed mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation who have completed the KHENERGYZE study KH176-202. - KHENEREXT

Khondrion B.V.0 个研究点目标入组 9 人开始时间: 待定最近更新:
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试验速览

阶段
2 期
状态
已完成
发起方
入组人数
9
研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

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. Having fulfilled all inclusion and exclusion criteria and completed the full
  • •treatment period of study KH176-202.
  • •4. 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.
  • •5. Objectified Left Ventricular Ejection Fraction (LVEF) >=45%
  • •(echocardiography, or otherwise).
  • •6. Left Ventricular (LV) wall thickness <=15 mm.
  • •7. Left atrium dilatation <= 40 mL/m2. Note: No need to test LV parameters
  • •(criteria #5, #6, #7) if favourable echocardiography (or otherwise) results
  • •dated less than 13 months prior to Screening are available.
  • •8.Women of childbearing potential must be willing to use highly effective
  • •contraceptive methods during the entire study, i.e. combined (estrogen and
  • •progestogen containing) oral, intravaginal or transdermal hormonal
  • •contraception associated with inhibition of ovulation; oral, injectable or
  • •implantable progestogen-only hormonal contraception associated with inhibition
  • •of ovulation; use of an intrauterine device; an intrauterine hormone releasing
  • •system, bilateral tubal occlusion and vasectomy of the partner. Any hormonal
  • •contraception method must be supplemented with a barrier method (preferably
  • •male condom). Vasectomised partner is considered a highly effective birth
  • •control method provided that partner is the sole sexual partner of the subject
  • •and that the vasectomised partner has received medical assessment of the
  • •surgical success.
  • •Sexual abstinence is considered a highly effective method only if defined as
  • •refraining from heterosexual intercourse during the entire period of risk
  • •associated with the study treatments. Reliability of sexual abstinence needs to
  • •be evaluated in relation to the duration of the clinical trial and the
  • •preferred and usual lifestyle of the subject. 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
  • •this study.
  • •Note 2: To be considered not of childbearing potential, potential female
  • •subjects must be post-menopausal for at least two years, or have been
  • •surgically sterilised (bilateral tubal ligation, hysterectomy or bilateral
  • •oophorectomy) for at least 6 months prior to Screening.
  • •Note 3: Sonlicromanol has been shown non-genotoxic judged from the Ames test,
  • •Chromosomal Aberration test and in vivo Micronucleus test. Moreover,
  • •appreciable systemic exposure from the exposure to (~2.5 mL) semen is extremely
  • •unlikely. However, until reproductive toxicology studies have confirmed that
  • •sonlicromanol does not adversely affect normal reproduction in adult males and
  • •females, as well as causing developmental toxicity in the offspring, the
  • •following contraceptive precautions must be adhered to:
  • •male subjects with female partners of childbearing potential must be willing
  • •to use condoms during the entire study.
  • 另有 1 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 (except sonlicromanol) 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), PVC burden
  • •>=5% or daytime Mobitz II AV block on any of the Holter assessments in the
  • •KH176-202 study or in the medical history.
  • •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 blood pressure > 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; Mean QTc of triplicate ECG recording > 450 ms for male subjects; mean
  • •QTc of triplicate ECG recording > 470 ms for female subjects (Diagram-read),
  • •T-top inversion in >1 consecutive lead.
  • •7. Serum hyperkalemia (> 5.0 mEq/L).
  • •8. Serum hypokalemia (< 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. History of hypersensitivity or idiosyncrasy to any of the components of the
  • •investigational drug.
  • •14. Medical history of drug abuse (illegal drugs such as cannabinoids,
  • •amphetamines, cocaine, opiates or problematic use of prescription drugs such as
  • •benzodiazepines, opiates).
  • •15. 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, Vitamin E, riboflavin, and antioxidant
  • •supplements (including, but not limited to idebenone/EPI-743, mitoQ or
  • •alternative names for similar products); 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.
  • 另有 3 项未显示

研究者

发起方
Khondrion B.V.

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