Frequently Asked Questions
1. Who is sponsoring the ACHIEVE trial?
This research trial is being sponsored by Dyne Therapeutics, a clinical stage company based in Waltham, Massachusetts.
2. What is the intent of the ACHIEVE trial?
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:
- Receive DYNE-101 or placebo by intravenous (IV) infusion every 4 or 8 weeks during the first 24-week placebo-controlled period,
- Receive DYNE-101 by IV infusion every 4 or 8 weeks throughout the OLE and LTE trial periods and
- Have his or her DM1 and overall health assessed regularly throughout the trial
3. Who is eligible to participate in the ACHIEVE trial?
A participant in the ACHIEVE trial must:
- Be between the ages of 18 and 49
- Have grip myotonia that creates a delayed hand opening time of 2 seconds or more
- Be able to safely perform a set of muscle function assessments without the use of assistive or mobility devices such as canes or walkers
- Not have advanced heart or breathing problems
- Avoid becoming pregnant or fathering a child during the trial
- Be able to read, understand and agree to the Informed Consent Form (ICF) and participate in the trial without assistance from others
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 on the ACHIEVE clinical trial page at clinicaltrials.gov and euclinicaltrials.eu.
4. Where is ACHIEVE being conducted?
Information on current ACHIEVE clinical trial sites and recruitment status is available on the ACHIEVE trial page at clinicaltrials.gov and euclinicaltrials.eu. As sites are added to the trial or recruitment status is updated, this information will be reflected on the ACHIEVE listing at clinicaltrials.gov and euclinicaltrials.eu.
5. How long is this clinical trial?
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.
6. Are there costs for participating?
Clinical trial participation, administration of the investigational therapy and all trial tests and procedures are provided at no cost to participants. Participants are reimbursed for approved trial-related travel expenses. Participant support services are available to participants who choose to have trial-related travel and accommodations arranged for them at no cost.
7. Has any data from the ACHIEVE study been shared?
Dyne shared the most recent data from the ACHIEVE study in May 2024. It is publicly available on our website and has been shared at multiple Patient Advocacy Organization events.
For more information:
Individuals interested in the ACHIEVE trial who would like to learn more can:
- Talk to their myotonic dystrophy treating physician about the trial
- Contact a doctor working on the ACHIEVE trial; for a list of participating physicians and sites, visit the ACHIEVE trial page at clinicaltrials.gov and euclinicaltrials.eu.
Questions for Dyne Therapeutics can be directed to clinicaltrials@dyne-tx.com.
Updated November 2024
