[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Lupin Ltd.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":85},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,40,62],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":29,"startDateStruct":32,"completionDateStruct":34,"leadSponsor":36,"locationsCount":39},"100556307","phase-3-the-efficacy-and-safety-of-once-daily-mexiletine-pr-in-patients-with-myotonic-dystrophy-type-1-and-type-2-100556307",false,"NCT06523400","The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2","A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients With Myotonic Dystrophy Type 1 and Type 2 (Phase 3)","Inclusion Criteria:\n\n1. DM1 or DM2 diagnosis confirmed genetically;\n2. Ability to comprehend and willingness to sign an informed consent (ICF) or ICF of the parent(s)\u002Flegal guardian and written assent from the patient (if patient \\\u003C 18 years of age);\n3. Ability to understand the study requirements including intention to stay in the study until the end-of-study visit at 26 weeks of treatment;\n4. Male or non-pregnant female ≥16 years of age;\n5. Body Mass Index (BMI) of 18.5 kg\u002Fm2 to 30 kg\u002Fm2, and weight ≥45 kg;\n6. Female patients of childbearing potential must be using a highly effective form of birth control for the duration of the study and for at least 7 days after last dose of study drug;\n7. No significant cardiac abnormalities as determined by a cardiologist's assessment;\n8. Have sufficient finger flexor strength to grasp the handle of the dynamometer used to measure myotonia;\n9. Presence of clinical handgrip myotonia (delayed relaxation of grip of ≥ 3 seconds after maximum voluntary contraction) at screening using VHOT;\n10. Be able to walk independently 10 meters (cane, walker, orthoses allowed);\n11. DM1 patients only - Muscular impairment rating scale (MIRS) score of 2, 3 or 4.-\n\nExclusion Criteria:\n\n1. Are pregnant or lactating;\n2. Have any one of the following medical conditions: uncontrolled diabetes mellitus, cancer other than skin cancer less than five years previously (e.g., basal-cell carcinoma (BCC) and squamous-cell carcinoma (SCC) of skin allowed), multiple sclerosis, seizure disorders, or other serious medical illness;\n3. Severe renal impairment (glomerular filtration rate (GFR) \\\u003C 30 mL\u002Fmin);\n4. Medical conditions which could interfere with muscle function such as infections, trauma, fractures, or planned surgery;\n5. Medical conditions that could affect hand functioning including but not limited to rheumatoid arthritis, Dupuytren's contracture, hand deformity, etc.;\n6. Severe arthritis or medical condition (other thanDM1\u002FDM2) that would significantly impact ambulation;\n7. High incidence of falls or fall-associated fractures (\\>5 falls during the past 12 months);\n8. Preexisting elevated liver function tests \\> 3 times the upper limit of normal (ULN) at screening (alanine transaminase (ALT)\u002Faspartate transaminase (AST), gamma-glutamyl transferase (GGT)) and\u002For any abnormal chemistry, hematology or urine lab considered clinically significant by investigator;\n9. Serum potassium values \\\u003C 3.5 mmol\u002FL or \\> 5.0 mmol\u002FL or serum magnesium values \\\u003C 1.7 mg\u002FdL. Electrolytic imbalance such as hypokalaemia, hyperkalaemia or hypomagnesaemia may increase the proarrhythmic effects of mexiletine. Electrolyte imbalances need to be corrected before administering mexiletine and will be monitored throughout treatment.\n10. Treatment with mexiletine within 4 weeks prior to baseline (Day 1);\n11. Intake of any anti-myotonic treatment within 4 weeks prior to baseline (Day 1) or 5 half-lives, whichever is longer such as metformin, such as propafenone, flecainide, lamotrigine, carbamazepine or any other channel-blocker\u002F anticonvulsive drugs;\n12. Use of any concomitant medications that could increase the cardiac risk;\n13. Known allergy to mexiletine or any local anesthetics;\n14. Participation in another interventional clinical study during the last 3 months or 5 half-lives of the investigational medicinal product, whichever is longer;\n15. Wheelchair-bound or bed-ridden;\n16. Any cardiac safety associated condition including any of the following criteria detected by screening cardiac evaluations including 24-hr Holter monitor, echocardiogram and clinical evaluations:\n\n    * PR interval ≥240 ms or QRS duration ≥120 ms on resting ECG\n    * Personal history of 3rd degree or 2nd degree type 2 atrioventricular block or sinus node dysfunction with pauses ≥3 seconds, complete bundle branch block, bifascicular and trifascicular block or any heart block susceptible to evolve to complete heart block\n    * Personal history of sustained atrial fibrillation, flutter or tachycardia (duration \\>30 seconds)\n    * Personal history of non-sustained (ventricular triplets or more) or sustained ventricular tachycardia\n    * Myocardial infarction (acute or past) or coronary artery stenosis \\>50%, presence of abnormal Q waves\n    * New York Heart Association (NYHA) Class II to IV heart failure\n    * Left ventricular systolic dysfunction with ejection fraction \\\u003C50%\n    * Sinus node dysfunction (including ECG sinus rate \\\u003C50 beats per minute (BPM))\n    * Co-administration of antiarrhythmics inducing torsades de pointes (class Ia: quinidine, procainamide, disopyramide, ajmaline; class Ic: encainide, flecainide, propafenone, moricizine; class III: amiodarone, sotalol, ibutilide, dofetilide, dronedarone, vernakalant)\n    * Co-administration of other classes of antiarrhythmics (class Ib: lidocaine, phenytoin, tocainide; class II: propranolol, esmolol, timolol, metoprolol, atenolol, carvedilol, bisoprolol, nebivolol; class IV: verapamil, diltiazem)\n    * Patients with implantable cardioverter defibrillators (ICDs) and pacemakers are excluded\n    * Presence of symptomatic coronary artery disease","ALL","16 Years",{"count":19,"type":20},176,"ESTIMATED","INTERVENTIONAL",[23],"PHASE3","A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES study)",[26],"Myotonic Dystrophy","RECRUITING","2026-02-05",{"date":30,"type":31},"2026-02-06","ACTUAL",{"date":33,"type":31},"2025-02-13",{"date":35,"type":20},"2026-04-22",{"name":37,"class":38},"Lupin Ltd.","INDUSTRY",7,{"id":41,"slug":42,"hasResults":11,"nctId":43,"briefTitle":44,"officialTitle":45,"acronym":4,"eligibilityCriteria":46,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":47,"targetDuration":4,"studyType":21,"phases":49,"briefSummary":50,"conditions":51,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":53,"lastUpdatePostDateStruct":54,"startDateStruct":56,"completionDateStruct":58,"leadSponsor":60,"locationsCount":61},"100600454","phase-3-an-open-label-randomized-cross-over-study-to-investigate-the-efficacy-and-safety-of-mexiletine-pr-compared-to-mexiletine-ir-100600454","NCT07097701","An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR Compared to Mexiletine IR","An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR Compared to Mexiletine IR in Patients With Non-Dystrophic Myotonias (ACHILLES Study)","Inclusion Criteria\n\n1. Ability to comprehend and willingness to sign an informed consent (ICF) or ICF of the parent(s)\u002Flegal guardian and written assent from the patient (if patient \\\u003C 18 years of age);\n2. Non-dystrophic myotonias including myotonia congenita (MC), paramyotonia congenita (PC) and sodium channel myotonia (SCM) confirmed genetically;\n3. Male or non-pregnant female ≥16 years and older at screening;\n4. Body Mass Index (BMI) of 18.5 kg\u002Fm2 to 30 kg\u002Fm2, and weight ≥45 kg;\n5. Female patients of childbearing potential must be using a highly effective form of birth control for the duration of the study and for at least 30 days after last dose of study drug. Male patients must use birth control for the duration of the study and for at least 30 days after last dose of study drug;\n6. No significant cardiac abnormalities as determined by a cardiologist including electrocardiogram (ECG) and echocardiogram not older than 3 months prior to study entry;\n7. Participants with myotonic symptoms severe enough to justify treatment (in the opinion of the study investigator);\n8. Presence of clinical handgrip myotonia (delayed relaxation of grip of ≥ 3 second using a stopwatch) at screening (naïve patients only) and on Day 1 (pre-dose) (patients naïve and previously treated with mexiletine).\n\nExclusion Criteria\n\n1. Are pregnant or lactating;\n2. Have any one of the following medical conditions: uncontrolled diabetes mellitus, cancer other than skin cancer less than five years previously (e.g., basal-cell carcinoma (BCC) and squamous-cell carcinoma (SCC) of skin allowed), multiple sclerosis, seizure disorders, or other serious medical illness or has any other condition, which in the opinion of the Investigator, precludes the participant's participation in the study or the participant is unlikely to comply with the protocol-defined procedures and therefore is unlikely to complete the study;\n3. Severe renal impairment (glomerular filtration rate (GFR) \\\u003C 30 mL\u002Fmin);\n4. Medical conditions which could interfere with muscle function such as infections, trauma, fractures, or planned surgery;\n5. Medical conditions that could affect hand functioning including but not limited to rheumatoid arthritis, Dupuytren's contracture, hand deformity, etc.;\n6. Severe arthritis or medical condition (other than NDM) that would significantly impact ambulation;\n7. Severe hepatic impairment or preexisting elevated liver function tests \\> 3 times the upper limit of normal (ULN) at screening (alanine transaminase (ALT)\u002Faspartate transaminase (AST), gamma-glutamyl transferase (GGT)) and\u002For any abnormal chemistry, hematology or urine lab considered clinically significant by the investigator;\n8. Serum potassium values \\\u003C 3.5 mmol\u002FL or \\> 5.0 mmol\u002FL or serum magnesium values \\\u003C 1.7 mg\u002FdL. Electrolytic imbalance such as hypocalcaemia, hypercalcaemia, hypokalaemia, hyperkalaemia or hypomagnesaemia may increase the proarrhythmic effects of mexiletine. Electrolyte imbalances need to be corrected before administering mexiletine and will be monitored throughout treatment.\n9. Intake of any other anti-myotonic treatment within 4 weeks prior to baseline (Day 1) or 5 half-lives, whichever is longer (e.g., metformin, propafenone, flecainide, lamotrigine, carbamazepine or any other channel-blocker\u002Fanticonvulsive drugs;\n10. Use of any concomitant medications that could increase the cardiac risk or increases the risk of adverse reactions (see Section 6.8 for a complete list of prohibited concomitant medications);\n11. Known allergy to mexiletine or any of the excipients or any local anesthetics;\n12. Participation in another interventional clinical study during the last 3 months or 5 half-lives of the investigational medicinal product, whichever is longer;\n13. Wheelchair-bound or bedridden;\n14. Any cardiac safety-associated condition including any of the following criteria detected by screening cardiac evaluations including ECG, echocardiogram and clinical evaluations (see protocol Section 5.3 for a detailed list);\n15. Current smokers (within one month of the screening visit) (eg, cigarettes, cigars, vape\u002Fe-cigarette products, etc.);\n16. Subgroup 'mexiletine-naïve': patients with previous treatment with mexiletine are excluded. Subgroup 'previous mexiletine treatment': patients with treatment with mexiletine within 1 week prior to baseline (Day 1) are excluded",{"count":48,"type":20},24,[23],"An Open-Label, Randomized, Cross-Over Study to Investigate the Efficacy and Safety of Mexiletine PR compared to Mexiletine IR in Patients with Non-Dystrophic Myotonias (ACHILLES study)",[52],"Myotonia","2025-12-15",{"date":55,"type":31},"2025-12-22",{"date":57,"type":31},"2025-09-03",{"date":59,"type":20},"2027-02-22",{"name":37,"class":38},1,{"id":63,"slug":64,"hasResults":11,"nctId":65,"briefTitle":66,"officialTitle":67,"acronym":4,"eligibilityCriteria":68,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":69,"enrollmentInfo":70,"targetDuration":72,"studyType":73,"phases":4,"briefSummary":74,"conditions":75,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":77,"lastUpdatePostDateStruct":78,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":61},"100604833","prospective-long-term-observational-study-patient-registry-of-paediatric-myotonic-disorders-100604833","NCT07154654","Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders","Prospective, Long Term, Observational Study (Patient Registry) of Paediatric Myotonic Disorders From Birth to Less Than Six Years of Age Who Are Treated With Mexiletine (PEGASUS Study).","Inclusion Criteria:\n\n1. Male or female patients from birth to less than 6 years\n2. A genetically confirmed diagnosis of NDM or DM (DM1or DM2), as per the treating clinician.\n3. Presence of clinical symptoms of myotonia (hand grip myotonia, myotonia in the leg muscles, any other myotonia symptoms) to be confirmed by the treating clinician.\n4. Patients already receiving mexiletine treatment or who are clinically considered for mexiletine treatment as per the treating physician judgement.\n5. No history of or significant cardiac abnormalities as determined by a cardiologist's assessment of the ECG and echocardiogram performed prior to enrolment in the study or as per the treating physician standard of care (NaMuscla SmPC, 2023)\n6. No known history or signs and symptoms of any significant liver disorder as per treating physician.\n7. No known clinically relevant abnormal laboratory investigations for haematology, biochemistry, and urinalysis values at screening (or based on values obtained within 3 months prior to screening in patient's medical record) that could affect the study objectives as judged by the treating physician.\n8. Parent or legal guardian able to provide consent\u002Fassent to study participation and to sign the written informed consent or non-opposition as per local regulatory requirements prior to study entry and perform any study-related activity. -\n\nExclusion Criteria:\n\n1. Any contraindication to mexiletine as listed in the Namuscla Summary of Product Characteristics (SmPC) (NaMuscla SmPC, 2023)\n\n   1. Hypersensitivity to the active substance, or to any of the excipients\n   2. Hypersensitivity to any local anaesthetic\n   3. Ventricular tachyarrhythmia\n   4. 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\u002For wide QRS complex (≥ 120 ms), second-degree atrioventricular block, bundle branch block, bifascicular and trifascicular block),\n   5. QT interval \\> 450ms\n   6. Myocardial infarction (acute or past), or abnormal Q-waves\n   7. Symptomatic coronary artery disease\n   8. Heart failure with ejection fraction \\\u003C50%\n   9. Atrial tachyarrhythmia, fibrillation or flutter\n   10. Sinus node dysfunction (including sinus rate \\\u003C 50 bpm)\n   11. Co-administration with medicinal products inducing torsades de pointes.\n   12. Co-administration with medicinal products with narrow therapeutic index\n2. Any other neurological or psychiatric condition that might affect the study assessments, as per the treating clinician.\n3. Any clinically significant illness, laboratory findings, ECG, or other clinical symptoms, which in the opinion of the treating physician could affect the patient's optimal participation in the study\n4. Receiving strong inducers or inhibitors of CYP2D6 or CYP1A2 or planned to receive them, during the subject participation (See section 4.1.5.1 Prohibited medications).\n5. Any concurrent illness, or medications which could affect the muscle function, and confound the results according to the treating physician.\n6. Seizure disorder, diabetes mellitus requiring treatment by insulin.","6 Years",{"count":71,"type":20},10,"2 Years","OBSERVATIONAL","This is a prospective, open-label, multi-centre, single arm, registry study to collect standard relevant clinical and epidemiological data during routine medical evaluation and treatment in paediatric patients with myotonic disorders who are being treated with mexiletine therapy according to the physician.",[76],"Myotonic Disorders","2025-09-22",{"date":79,"type":31},"2025-09-23",{"date":81,"type":20},"2025-09-30",{"date":83,"type":20},"2028-02-22",{"name":37,"class":38},""]