Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed MEX-NM-301.
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Number and frequency of adverse events (AEs)/serious adverse events (SAEs), throughout the study while on treatment with Namuscla
研究概览
简要总结
The aim of this study is to obtain additional information regarding the long-term safety and efficacy of mexiletine for the symptomatic treatment of myotonia in paediatric subjects who have completed the initial paediatric study MEX-NM-301.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Patients previously completed the parent study PIP study 4 (MEX-NM-301) and tolerated the Mexiletine in the study.
- •Able and willing to provide assent to study participation and a parent or legal guardian willing to sign written informed consent prior to study entry.
- •Patients continue to meet inclusion criteria of parent study (MEX-NM-301)
排除标准
- •Clinically significant laboratory abnormality, ECG or other clinical findings on physical examination indicative of a clinically significant exclusionary disease as determined by the investigator
- •Any contra-indication to mexiletine (as described in the Namuscla Summary of Product Characteristics [SmPC])
- •Co- administration with antiarrhythmics
- •Any other neurological or psychiatric condition that might affect the assessment of the study measurements
- •Any concurrent illness, or medications which could affect the muscle function
- •Seizure disorder, diabetes mellitus requiring treatment by insulin
- •Pregnant or breastfeeding
- •Concurrent participation in any other clinical trial.
结局指标
主要结局
Number and frequency of adverse events (AEs)/serious adverse events (SAEs), throughout the study while on treatment with Namuscla
Number and frequency of adverse events (AEs)/serious adverse events (SAEs), throughout the study while on treatment with Namuscla
Incidence of adverse events of special interest (AESI)
Incidence of adverse events of special interest (AESI)
Mean change in Visual Analogue Scale (VAS) or Faces score for muscle stiffness (myotonia severity) for children and adolescents aged 6 years to < 18 years (assessed every 3 months).
Mean change in Visual Analogue Scale (VAS) or Faces score for muscle stiffness (myotonia severity) for children and adolescents aged 6 years to < 18 years (assessed every 3 months).
Score of handgrip myotonia as quantitatively measured using a commercially available grip dynamometer and computerized capture system in standardized conditions for children and adolescents aged 6 years to < 18 years (every 6 months).
Score of handgrip myotonia as quantitatively measured using a commercially available grip dynamometer and computerized capture system in standardized conditions for children and adolescents aged 6 years to < 18 years (every 6 months).
次要结局
- Mean change in VAS (8 to < 18 years) or Faces (6 to < 8 years) score for severity of muscle stiffness (if not a primary endpoint) pain, weakness and fatigue (every 3 months).
- Clinical myotonia assessment (every 6 months) - Mean change in time to open the eyes after forced eye closure as measured on a stopwatch (when eyelid myotonia present)
- Clinical myotonia assessment (every 6 months) - Clinical improvement in flexor myotonia (right hand flexor muscles)
- Clinical myotonia assessment (every 6 months) - Mean change in time to perform Timed-up and go (TUG) test (patients aged 6 to <18 years only)
- Mean change in health-related quality-of-life as measured by the Paediatric Quality of Life (PedsQL) score (secondary endpoint for patients aged 6 years to <18 years; every 6 months).
- Clinical Global Impression (CGI) scores (efficacy and tolerability) evaluated by the patient, a parent or proxy and by the investigator. Measured every 6 months.
- Mean change in Myotonia Behaviour Scale (MBS) scores (for patients aged 6 years to < 18 years; measured every 6 months).
- Long-term safety of mexiletine - Changes in vital signs (every 3 months).
- Long-term safety of mexiletine - Changes in clinical laboratory values (every 3 months).
研究者
Alla Zozulya Weidenfeller
Scientific
Lupin Europe GmbH