Mexiletine for Myotonic disorders | Nondystrophic myotonia | Myotonic dystrophy type 1 | Myotonic dystrophy type 2

Inclusion criteria

• {"criterion_text":"- Patients previously completed the parent study PIP study 4 (MEX-NM-301) and tolerated the Mexiletine in the study.\n- 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.\n- Patients continue to meet inclusion criteria of parent study (MEX-NM-301)"}

Exclusion criteria

• {"criterion_text":"- Clinically significant laboratory abnormality, ECG or other clinical findings on physical examination indicative of a clinically significant exclusionary disease as determined by the investigator\n- Any contra-indication to mexiletine (as described in the Namuscla Summary of Product Characteristics [SmPC])\n- Co- administration with antiarrhythmics\n- Any other neurological or psychiatric condition that might affect the assessment of the study measurements\n- Any concurrent illness, or medications which could affect the muscle function\n- Seizure disorder, diabetes mellitus requiring treatment by insulin\n- Pregnant or breastfeeding\n- Concurrent participation in any other clinical trial."}

Primary endpoints

• {"endpoint_text":"- Number and frequency of adverse events (AEs)/serious adverse events (SAEs), throughout the study while on treatment with Namuscla","definition_or_measurement_approach":"Collection and counting of AEs/SAEs reported throughout the study while on treatment with Namuscla (safety monitoring during treatment)."}
• {"endpoint_text":"- Incidence of adverse events of special interest (AESI)","definition_or_measurement_approach":"Recording and incidence calculation of predefined adverse events of special interest."}
• {"endpoint_text":"- 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).","definition_or_measurement_approach":"Mean change from baseline in VAS (or Faces score for younger children) for muscle stiffness assessed every 3 months."}
• {"endpoint_text":"- 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).","definition_or_measurement_approach":"Quantitative handgrip myotonia score measured with a commercial grip dynamometer and computerized capture system under standardized conditions, assessed every 6 months."}

Secondary endpoints

• {"endpoint_text":"- 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).","definition_or_measurement_approach":"Mean change from baseline in VAS or Faces scores for muscle stiffness, pain, weakness and fatigue assessed every 3 months."}
• {"endpoint_text":"- 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)","definition_or_measurement_approach":"Mean change in time to open eyes after forced closure measured by stopwatch (when eyelid myotonia present), every 6 months."}
• {"endpoint_text":"- Clinical myotonia assessment (every 6 months) - Clinical improvement in flexor myotonia (right hand flexor muscles)","definition_or_measurement_approach":"Clinical assessment of improvement in right hand flexor myotonia performed every 6 months."}
• {"endpoint_text":"- 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)","definition_or_measurement_approach":"Mean change in time to perform TUG test for patients aged 6 to <18 years, assessed every 6 months."}
• {"endpoint_text":"- 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).","definition_or_measurement_approach":"Mean change from baseline in PedsQL score assessed every 6 months for patients aged 6 to <18 years."}
• {"endpoint_text":"- Clinical Global Impression (CGI) scores (efficacy and tolerability) evaluated by the patient, a parent or proxy and by the investigator. Measured every 6 months.","definition_or_measurement_approach":"CGI scores for efficacy and tolerability as rated by patient, parent/proxy and investigator, assessed every 6 months."}
• {"endpoint_text":"- Mean change in Myotonia Behaviour Scale (MBS) scores (for patients aged 6 years to < 18 years; measured every 6 months).","definition_or_measurement_approach":"Mean change in MBS scores from baseline, measured every 6 months for patients aged 6 to <18 years."}
• {"endpoint_text":"- Long-term safety of mexiletine - Changes in vital signs (every 3 months).","definition_or_measurement_approach":"Monitoring and reporting of changes in vital signs every 3 months as part of long-term safety assessment."}
• {"endpoint_text":"- Long-term safety of mexiletine - Changes in clinical laboratory values (every 3 months).","definition_or_measurement_approach":"Monitoring and reporting of changes in clinical laboratory values every 3 months as part of long-term safety assessment."}

France

Earliest CTIS Part Ii Submission Date

05-11-2024

Latest Decision Or Authorization Date

19-11-2024

Processing Time Days

14

Number Of Sites

2

Number Of Participants

12

Primary sponsor

Full Name

Lupin Europe GmbH

Organisation Type

Pharmaceutical company

Country Of Registered Address

Germany