2024-514970-27-00招募中3 期
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)
Lupin Atlantis Holdings S.A.6 个研究点 分布在 4 个国家目标入组 20 人开始时间: 2025年1月31日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 6
- 主要终点
- Incidence of treatment emergent Adverse Events (TEAEs), treatment-related TEAEs, serious AEs, AEs of Special Interest (AESIs), and patient discontinuation rate between mexiletine PR and mexiletine IR after 12 weeks of treatment.
研究概览
简要总结
To compare the safety of mexiletine PR vs mexiletine IR for the symptomatic treatment of myotonia in adult patients with non-dystrophic myotonias (myotonia congenita (MC), paramyotonia congenita (PC) and sodium channel myotonia (SCM)).
入排标准
- 年龄范围
- 0 years 至 65+ years（18-64 Years, 65+ Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Non-dystrophic myotonias including myotonia congenita (MC), paramyotonia congenita (PC) and sodium channel myotonia (SCM) confirmed genetically;
- •Male or non-pregnant female ≥16 years and older at screening;
- •Body Mass Index (BMI) of 18.5 kg/m2 to 30 kg/m2, and weight ≥45 kg;
- •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;
- •No significant cardiac abnormalities as determined by a cardiologistincludingcardiologist including electrocardiogram (ECG) and echocardiogram not older than 3 months prior to study entry;
- •Participants with myotonic symptoms severe enough to justify treatment;
- •Ability to comprehend and willingness to sign an informed consent (ICF) or ICF of the parent(s)/legal guardian and written assent from the patient (if patient < 18 years of age);
排除标准
- •Are pregnant or lactating;
- •Medical conditions which could interfere with muscle function such as infections, trauma, fractures, or planned surgery;
- •Medical conditions that could affect hand functioning including but not limited to rheumatoid arthritis, Dupuytren's contracture, hand deformity, etc.;
- •Severe arthritis or medical condition (other than NDM) that would significantly impact ambulation;
- •Severe hepatic impairment or preexisting elevated liver function tests > 3 times the upper limit of normal (ULN) at screening (alanine transaminase (ALT)/aspartate transaminase (AST), gamma-glutamyl transferase (GGT)) and/or any abnormal chemistry, hematology or urine lab considered clinically significant by the investigator;
- •Serum potassium values < 3.5 mmol/L or > 5.0 mmol/L or serum magnesium values < 1.7 mg/dL. 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.
- •Current smokers (within one month of the screening visit) (eg, cigarettes, cigars, vape/e-cigarette products, etc.);
- •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/anticonvulsive drugs;
- •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);
- •Known allergy to mexiletine or any of the excipients or any local anesthetics;
- •Participation in another interventional clinical study during the last 3 months or 5 half-lives of the investigational medicinal product, whichever is longer;
- •Wheelchair-bound or bedridden;
- •Any cardiac safety-associated condition including any of the following criteria detected by screening cardiac evaluations including 24-hour Holter monitoring, ECG, echocardiogram and clinical evaluations (see protocol Section 5.3 for a detailed list).
- •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;
- •Severe renal impairment (glomerular filtration rate (GFR) < 30 mL/min);
研究组 & 干预措施
Mexiletine granules for prolonged-release oral suspension 333 mg, Mexiletine granules for prolonged-release oral suspension 167 mg, Mexiletine granules for prolonged-release oral suspension 500 mg
Test
干预措施: Mexiletine granules for prolonged-release oral suspension 333 mg (Drug)
Namuscla 167 mg hard capsules
Comparator
干预措施: Namuscla 167 mg hard capsules (Drug)
Mexiletine granules for prolonged-release oral suspension 333 mg, Mexiletine granules for prolonged-release oral suspension 167 mg, Mexiletine granules for prolonged-release oral suspension 500 mg
Test
干预措施: Mexiletine granules for prolonged-release oral suspension 167 mg (Drug)
Mexiletine granules for prolonged-release oral suspension 333 mg, Mexiletine granules for prolonged-release oral suspension 167 mg, Mexiletine granules for prolonged-release oral suspension 500 mg
Test
干预措施: Mexiletine granules for prolonged-release oral suspension 500 mg (Drug)
结局指标
主要结局
Incidence of treatment emergent Adverse Events (TEAEs), treatment-related TEAEs, serious AEs, AEs of Special Interest (AESIs), and patient discontinuation rate between mexiletine PR and mexiletine IR after 12 weeks of treatment.
Incidence of treatment emergent Adverse Events (TEAEs), treatment-related TEAEs, serious AEs, AEs of Special Interest (AESIs), and patient discontinuation rate between mexiletine PR and mexiletine IR after 12 weeks of treatment.
次要结局
- Clinical Global Impression (CGI) – Tolerability Index
- Video-recording of hand opening time (VHOT)
- Individualized Neuromuscular Quality of Life Questionnaire (INQoL)
- Myotonia Behavior Scale (MBS)
- Timed “Up & Go” (TUG) Test
- Visual Analog Scale (VAS) for myotonia/stiffness
- Clinical Global Impression (CGI) – Efficacy
研究者
Alla Zozulya Weidenfeller
Scientific
Lupin Atlantis Holdings S.A.
研究点 (6)
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