This research trial is being sponsored by Dyne Therapeutics, a clinical stage company based in Waltham, Massachusetts.
The ACHIEVE trial is evaluating an investigational therapy called DYNE-101 for the first time in humans. The ACHIEVE trial is designed to determine the safety, preferred dose levels and frequency of administration of DYNE-101, and how DYNE-101 works in trial participants living with DM1. “Investigational” means DYNE-101 is currently being tested to understand whether it can help people with DM1. It has not yet been approved for use in treating DM1 by any governmental regulatory agency.
The ACHIEVE trial is a Phase 1/2 global clinical trial evaluating DYNE-101 consisting of a 24-week multiple ascending dose-randomized placebo-controlled period, a 24-week open-label extension (OLE) period and a 96-week long-term extension (LTE) period. The trial is enrolling adult patients with DM1 who are 18 to 49 years of age. The primary endpoints are safety and tolerability; secondary endpoints include pharmacokinetics and pharmacodynamics, including change from baseline in splicing, as well as measures of muscle strength and function.
A DM1 community member who meets all criteria for participation and is accepted into the trial will:
A participant in the ACHIEVE trial must:
There are other conditions that DM1 community members must meet to participate in the ACHIEVE trial. Additional information on the ACHIEVE clinical trial is available at clinicaltrials.gov.
Information on current ACHIEVE clinical trial sites and recruitment status is available on the ACHIEVE trial page at clinicaltrials.gov. As sites are added to the trial or recruitment status is updated, this information will be reflected on the ACHIEVE listing at clinicaltrials.gov.
Participation in the trial could last approximately 3 years. However, being in this trial is completely voluntary. Participants can leave at any time. Leaving this trial will have no impact on care received outside the trial.
Clinical trial treatment and all trial tests and procedures are provided at no cost to participants. Participants are reimbursed for approved trial-related travel expenses. Patient support services are available to participants who choose to have trial-related travel arranged for them at no cost.
Dyne shared initial data from the ACHIEVE study in early January 2024. We anticipate reporting additional data in the second half of 2024.
For more information:
Individuals interested in the ACHIEVE trial who would like to learn more can:
Questions for Dyne Therapeutics can be directed to clinicaltrials@dyne-tx.com.
Updated March 2024