EUCTR2019-003757-28-FR进行中（未招募）1 期
An Open-label, non-Comparative Study to Evaluate the Steady-State Pharmacokinetics, Safety, and Efficacy of Mexiletine in Adolescents and Children with Myotonic Disorders
upin Europe GmbH0 个研究点目标入组 14 人开始时间: 2020年11月20日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 14
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
- •1. Male or female patients aged = 6 and < 18 years who are able to comply with the study conditions;
- •2. A genetically confirmed diagnosis of NDM or DM (DM1or DM2);
- •3. Presence of clinical symptoms of myotonia (hand grip myotonia, myotonia in the leg muscles, any other myotonia symptoms);
- •4. No significant cardiac abnormalities as determined by a cardiologist’s assessment of the ECG and echocardiogram performed within 3 months prior to enrolment in the study. (if not done within 3 months before trial, ECG and echocardiogram assessments will be performed at screening);
- •5. No history of any significant liver disorder;
- •6. Patients receiving mexiletine treatment agree to stop treatment at least 7 days prior to initiation of treatment with Namuscla;
- •7. Patients receiving other antimyotonic treatment agree to stop treatment for at least 7 times the half-life of respective drug;
- •8. Laboratory investigations for haematology, biochemistry, and urinalysis at screening are within normal range, or showing no clinically relevant abnormal values, as judged by the Investigator;
- •9. Female patients of childbearing potential must be using an acceptable form of birth control as determined by the Investigator (e.g., oral contraception, implantable, injectable/transdermal hormonal contraception, intrauterine device (IUD), barrier methods), tubal ligation or have a vasectomized partner or are practicing abstinence;
- •10. Patients able to provide assent to study participation and a parent or legal guardian able to sign written informed consent prior to study entry.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 14
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Any contra-indication to mexiletine as listed in the Namuscla Summary of Product Characteristics (SmPC):
- •1.a. Hypersensitivity to the active substance, or to any of the excipients;
- •1.b. Hypersensitivity to any local anaesthetic;
- •1.c. Ventricular tachyarrhythmia;
- •1.d. Complete heart block (i.e., third-degree atrioventricular block) or any heart block susceptible to evolve to complete heart block (first-degree atrioventricular block with markedly prolonged PR interval (= 200 ms) and/or wide QRS complex (= 120 ms), second-degree atrioventricular block, bundle branch block, bifascicular and trifascicular block);
- •1.e. QT interval > 450ms;
- •1.f. Myocardial infarction (acute or past), or abnormal Q-waves;
- •1.g. Symptomatic coronary artery disease;
- •1.h. Heart failure with ejection fraction <50%;
- •1.i. Atrial tachyarrhythmia, fibrillation or flutter;
- •1.j. Sinus node dysfunction (including sinus rate < 50 bpm);
- •1.k. Co-administration with medicinal products inducing torsades de pointes;
- •1.l. Co-administration with medicinal products with narrow therapeutic index;
- •2. Any other neurological or psychiatric condition that might affect the assessment of the study measurements;
- •3. Any clinically significant illness, laboratory findings, ECG, or other clinical symptoms, which in the opinion of the Investigator could affect the patient’s optimal participation in the study;
- •4. Strong inducer or inhibitor of CYP2D6 or CYP1A2 within 7 days prior to study drug administration;
- •5. Any concurrent illness, or medications which could affect the muscle function;
- •6. Seizure disorder, diabetes mellitus requiring treatment by insulin;
- •7. Pregnant or breastfeeding;
- •8. Concurrent participation in any other clinical trial.
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