* MIND Study - MEX-DM-301 version 1.0

• Research type

  Research Study

• Full title

  A Randomized, Double-blind, Placebo-controlled, Multi-centre 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] (Phase 3)

• IRAS ID

  295675

• Contact name

  Werner Schneider

• Contact email

  Wernerschneider@hormosan.de

• Sponsor organisation

  Lupin Europe GmbH

• Eudract number

  2020-005430-15

• Clinicaltrials.gov Identifier

  NCT04700046

• Duration of Study in the UK

  2 years, 1 months, 15 days

• Research summary

  This study is a Randomized, Double-blind, Placebo-controlled, Multi-centre Study to Investigate the Efficacy and Safety of Mexiletine During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2. This study is for myotonic dystrophies, DM1 and DM2, as there is currently no approved treatment for the myotonia associated with DM in the UK or European Union (EU).
  Mexiletine is recommended as anti-myotonic choice in several EU guidelines and has been used off label in the myotonia indication for more than two decades. This treatment is given to alleviate muscle stiffness thereby decreasing the associated pain and fatigue giving increased movement and improving quality of life.
  This study is being conducted with a six-month treatment duration in patients with DM1 and DM2 to confirm the efficacy and safety of mexiletine.
  The study will include appropriate efficacy and safety endpoints including Quality of Life and functional outcome measures to demonstrate the positive benefit/risk profile of mexiletine in DM patients.
  Only adult patients (≥ 18-years-old) will be included in this study.
  Patients in the study will be randomised to receive either 167mg mexiletine once a day which can be titrated up at the Week 1 and Week 2 visits if well tolerated up to a maximum of 1 capsule three times a day or placebo.
  Approximately 158 DM1 patients (79 active:79 placebo) are planned to be enrolled in 10-15 sites across France, Germany, Italy, Spain, and the UK. Additionally, up to 16 DM2 patients are planned to be enrolled (sub-group: 8 active and 8 placebo)

• REC name

  London - Brent Research Ethics Committee

• REC reference

  21/FT/0030

• Date of REC Opinion

  15 Apr 2021

• REC opinion

  Further Information Favourable Opinion