At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Mexiletine granules for prolonged-release oral suspension (drug), Placebo (drug)
- How long the study runs
- Study runs about 28 months (dates as stated)
- About the drug or intervention
- Mexiletine granules for prolonged-release oral suspension — drug: Mexiletine PR · Placebo — drug: Matching Placebo
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 16 Years and over
- Who
- All
- Number of participants
- 176
- Started
- 2025-02-13
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for myotonic dystrophy
- • Phase3 - 176 participants
- • 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) Study website: https://myotonicdystrophystudy
Who can take part?
- • Ages 16 Years and over
- • Diagnosed with myotonic dystrophy
Where?
- • London - Saint George's University Hospitals NHS Foundation Trust
- • London - University College Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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) Study website: https://myotonicdystrophystudy.com/
More detail
This is a multicenter, randomized, double-blind, parallel-group, placebo-controlled study intended to evaluate the efficacy and the safety of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2). The study will consist of a 4- week screening period and a 26-week treatment phase with patient visits at screening, baseline, Weeks 1, 2, 14, and 26. Eligible patients will be randomized to mexiletine or placebo in a 1:1 ratio. Approximately 80 DM1 patients (40 active: 40 placebo) are planned to be enrolled. For the purpose of sample size re-estimation, an interim analysis will be conducted when a total of 40 patients in total complete/early terminate the study. In addition, 16 DM2 patients are planned to be enrolled (sub-group - 8 active: 8 placebo). Study website: https://myotonicdystrophystudy.com/
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 16 Years and over
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓DM1 or DM2 diagnosis confirmed genetically;
- ✓Ability to comprehend and willingness to sign an agreement to take part (ICF) or ICF of the parent(s)/legal guardian and...
- ✓Ability to understand the study requirements including intention to stay in the study until the end-of-study visit...
- ✓Male or non-pregnant female ≥16 years of age;
- ✓Body Mass Index (BMI) of 18.5 kg/m2 to 30 kg/m2, and weight ≥45 kg;
Who cannot take part
- ✗Are pregnant or lactating;
- ✗Severe kidney 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...
- ✗Severe arthritis or medical condition (other thanDM1/DM2) that would significantly impact ambulation;
See the full criteria
Where Is This Study? (2 UK sites)
Saint George's University Hospitals NHS Foundation Trust
London, United Kingdom
University College Hospital
London, United Kingdom
Rajinder Sidhu - Associate Director, Research Governance and Operations
uclh.jro-communications@nhs.net020 3447 9825How to Get in Touch
Nikki Adetoro
Sponsor contactCONTACT
