ACHIEVE study

• Research type

  Research Study

• Full title

  A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1

• IRAS ID

  1005924

• Contact name

  Caitlin Abell

• Contact email

  cabell@dyne-tx.com

• Sponsor organisation

  Dyne Therapeutics, Inc

• Eudract number

  2022-000889-18

• Clinicaltrials.gov Identifier

  NCT05481879

• Research summary

  This is a randomised, double-blinded, placebo-controlled, multiple ascending dose study in adult patients with Myotonic dystrophy type 1 (DM1). DM1 is a rare, progressive, neuromuscular disease, characterized by many symptoms including prolonged muscle contractions (myotonia), and muscle weakness that gets worse over time and may lead to physical disabilities. DM1 is estimated to affect approximately 1 in 3,000 to 8,000 people worldwide.\n \nThe purpose of this study is to evaluate the safety, tolerability, pharmacodynamics, efficacy, and pharmacokinetics of the study drug (also called DYNE­101). All participants will undergo a Screening Period to assess eligibility, and a Placebo-Controlled Period. Participants will be randomised to DYNE­101 or Placebo. After the Placebo-Controlled Period, all participants will enter a Treatment Period and then Long Term Extension (LTE), where all participants will receive treatment with study drug. \n \nAll participants will be closely monitored during study drug administration. Study procedures include physical examinations, blood and urine samples, measures of heart and lung function, muscle biopsies, and motor function assessments. Up to 64 patients will participate in the study worldwide.

• REC name

  London - Brent Research Ethics Committee

• REC reference

  22/LO/0517

• Date of REC Opinion

  27 Sep 2022

• REC opinion

  Further Information Favourable Opinion