An Observational Study to Describe the Long-term Safety and Effectiveness of Namuscla in the Symptomatic Management of Myotonia in Adult Patients With Non-dystrophic Myotonic Disorders
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Lupin Ltd.
- 入组人数
- 53
- 试验地点
- 12
- 主要终点
- Primary Outcome 1 Proportion of patients with treatment-emergent AEs
研究概览
简要总结
This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM.
详细描述
This is a non-interventional, prospective, observational, multicentre study to evaluate the long-term safety and effectiveness of Namuscla in adult patients with NDM. Namuscla should be prescribed as per the approved Summary of Product Characteristics (SmPC).
Adult patients with non-dystrophic myotonic disorders who have been prescribed Namuscla by the treating physician, and who meet the eligibility criteria will be enrolled in this study.
This includes:
- Patients newly initiated on Namuscla for the treatment of NDM (newly exposed)
- Patients already on Namuscla/ mexiletine at enrolment - For patients receiving mexiletine other than Namuscla, only those who switch to Namuscla will be included in the study.
Patients already being treated with Namuscla/ mexiletine at the time of enrolment will be considered for enrolment provided they meet the eligibility criteria.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult, male or female patients with non-dystrophic myotonic disorders planned to be started on Namuscla according to the approved SmPC
- •Patients already receiving Namuscla/mexiletine for the treatment of NDM; (for patients on mexiletine other than Namuscla, only those who switch to Namuscla will be enrolled).
- •Patients who understand and are willing to provide informed consent.
排除标准
- •Patients who are enrolled or participating in any other clinical trial for an investigational product. -
- •Hypersensitivity to mexiletine, or to any of the excipients of Namuscla, or hypersensitivity to any local anaesthetic
- •Ventricular tachyarrhythmia
- •Atrial tachyarrhythmia, fibrillation or flutter
- •Complete heart block (ie, third-degree atrioventricular block) or any heart block susceptible to evolve to complete heart block (first-degree atrioventricular block with markedly prolonged PR interval (≥ 240 ms) and/or wide QRS complex (≥ 120 ms), second-degree atrioventricular block, bundle branch block, bifascicular and trifascicular block),
- •Myocardial infarction (acute or past), or abnormal Q-waves
- •Symptomatic coronary artery disease
- •Heart failure with reduced ejection fraction <50%
- •Sinus node dysfunction (including sinus rate < 50 bpm)
- •Patients receiving drugs that can induce torsades de pointes
- •Patients receiving medicinal products with narrow therapeutic index (ie, theophylline, tizanidine, digoxin, lithium, phenytoin or warfarin)
- •Patients who are pregnant or lactating.
结局指标
主要结局
Primary Outcome 1 Proportion of patients with treatment-emergent AEs
时间窗: Approximately 3 years
Proportion of patients with treatment-emergent AEs (\[TEAEs\], including SAEs) from study enrolment to 6, 12, 24 and 36 months on Namuscla
Primary Outcome 2 Proportion of patients requiring dose reduction or treatment discontinuation
时间窗: Approximately 3 years
Proportion of patients requiring dose reduction or treatment discontinuation due to AEs (including SAEs).
次要结局
- Secondary Outcome Proportion of patients with AEs /SAEs/ Adverse Event of Special Interest (AESI)(Approximately 3 years)