Efficacy, Safety, and Tolerability of DYNE-101 in Participants with Myotonic Dystrophy Type 1

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1

• IRAS ID

  1012625

• Contact name

  Fernanda Guipponi

• Contact email

  fernanda.guipponi@dyne-tx.com

• Sponsor organisation

  Dyne Therapeutics, Inc

• Research summary

  Myotonic dystrophy is a type of muscular dystrophy (a group of genetic disorders). Myotonic Dystrophy Type 1 (DM1) is characterised by muscle weakness and wasting, myotonia, cataracts and heart problems.

  The dystrophia myotonica protein kinase (DMPK) gene is important for making a protein needed by muscles, the heart, and other parts of the body. Normally, a gene creates ribonucleic acid (RNA), which then makes the protein our body needs. In DM1, the DMPK gene has too many repeats of a certain part, leading to abnormal DMPK RNA. This abnormal RNA causes the problems seen in DM1.

  Currently, there is no cure for DM1, but this study is investigating DYNE 101, an investigational drug designed to target and reduce abnormal DMPK RNA. The study will test DYNE-101 to determine its safety and to find out if it can improve muscle strength, function, and quality of life for people with DM1.

  The study will involve approximately 150 participants across multiple countries. It is a randomised, double blind study with a 48-week placebo controlled period and 24-week long term extension (LTE) period. This means participants will have a 50% chance of receiving DYNE-101 and neither the participant or the study team will know which treatment they are receiving.

  Participants will be in the study for approximately 18 months and will have multiple study visits where they will undergo different study procedures, such as physical examinations, blood tests, patient questionnaires and motor function assessments. The study is split into 4 periods as follows:
  • Screening Period where it is checked if the participant meets requirements to take part
  • Placebo-Controlled Period where participant receives doses of DYNE-101 or placebo every 8 weeks for the next 48 weeks
  • LTE Period where all participants receive DYNE-101 every 8 weeks for about 24 weeks
  • End of study visit: final study visit once the participant has completed all study visits

• REC name

  London - Surrey Borders Research Ethics Committee

• REC reference

  26/LO/0170

• Date of REC Opinion

  7 Apr 2026

• REC opinion

  Further Information Favourable Opinion