WithdrawnPhase 3

Study to Investigate the Efficacy and Safety of Mexiletine in Patients With Myotonic Dystrophy Type 1 and Type 2

Stopped: Assessing novel alternative study designs and regulatory pathways

0Started 2021-09-03

Plain-language summary

A Randomized, Double-blind, Placebo-controlled, Multi-center Study to Investigate the Efficacy and Safety of Mexiletine During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 \[The MIND Study\]

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. DM1 or DM2 diagnosis confirmed genetically;
✓. Ability to provide informed consent;
✓. Ability to understand the study requirements including intention to stay in the study until the end-of-study visit at 26 weeks of treatment;
✓. Male or non-pregnant female ≥18 years of age;
✓. Female patients of childbearing potential must be using an acceptable form of birth control as determined by the investigator (e.g., oral contraception, implantable, injectable/transdermal hormonal contraception, intrauterine device (IUD), barrier methods), tubal ligation, have a vasectomized partner, or are practicing abstinence;
✓. No significant cardiac abnormalities as determined by a cardiologist's assessment of the electrocardiogram (ECG) and echocardiogram;
✓. Capable of swallowing capsules;
✓. Have sufficient finger flexor strength to grasp the handle of the dynamometer used to measure myotonia;

Exclusion criteria

✕. Are pregnant or lactating;
✕. 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;
✕. Severe renal impairment (glomerular filtration rate (GFR) \< 30 mL/min);
✕. 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 other medical condition (besides DM1/DM2) that would significantly impact ambulation;
✕. High incidence of falls or fall-associated fractures (\>5 falls during the past 12 months);
✕. 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;

What they're measuring

Assess the efficacy and safety of mexiletine for the symptomatic treatment of myotonia

Timeframe: 6 months

Trial details

NCT IDNCT04700046

SponsorLupin Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-07-01

View on ClinicalTrials.gov More Myotonic Dystrophy Type 1 and Type 2 trials