Myotonic disorders: Nondystrophic myotonias (NDM) or myotonic dystrophies (DM, type 1 or type 2)
Conditions
Brief summary
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), 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).
Detailed description
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).
Interventions
DRUGMexiletine 83 mg hard capsules
DRUGMexiletine 62 mg hard capsules
Sponsors
Lupin Europe GmbH
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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), 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). | — |
Secondary
| Measure | Time frame |
|---|---|
| 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) s | — |
Countries
France
Outcome results
None listed