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Frequently Asked Questions

1. What is this trial studying?

The ACHIEVE study, sponsored by Dyne Therapeutics, is evaluating an investigational therapy called DYNE-101 for the first time in humans. The ACHIEVE study is designed to determine the safety, preferred dose levels and frequency, and how DYNE-101 works in study participants living with DM1.

2. How does this trial differ from other studies?

Dyne’s mission is to deliver life-transforming therapies for people living with serious muscle diseases. Dyne’s proprietary FORCE™ platform is designed to overcome the current limitations of muscle tissue delivery. The FORCE mechanism differs from other therapies focused on muscle delivery. The FORCE platform consists of three parts: an antigen-binding fragment (Fab) designed to enable targeted delivery of an antisense oligonucleotide (ASO) to muscle, a linker, and an ASO selected with the goal of targeting the genetic basis of disease.

In DM1, knockdown of DMPK is important to targeting the genetic mutation. Dyne designed an ASO with the goal of targeting toxic DMPK in the nucleus. The Dyne Fab is designed to bind to the transferrin receptor 1, or TfR1. TfR1 receptors are highly expressed across the surface of muscle cells, including skeletal, cardiac and smooth muscles. Dyne therefore designed the FORCE platform with the goal of harnessing receptors that already exist on the surface of muscle cells to deliver the potential therapeutic with this receptor-mediated mechanism.

3. What will I gain from participating in this trial?

The ACHIEVE study is part of Dyne’s efforts to develop therapies for people living with DM1. It will test DYNE-101 for the first time in humans. The ACHIEVE study is designed to determine the safety, preferred dose levels and frequency, and how DYNE-101 works in study participants living with DM1. Taking part in this study may or may not help to treat DM1. Your health could improve, stay the same, or get worse. However, the data collected during this study may help doctors learn more about the study drug and DM1 and this may help future affected individuals.

4. What does the trial consist of? Where does the trial take place?

The ACHIEVE study 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 and a 96-week long-term extension. 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. The number of participants may be expanded if the scientists and medical professionals involved believe it is appropriate.

Current trial sites are available in the ACHIEVE listing at clinicaltrials.gov.

5. What are the conditions for inclusion?

Current inclusion criteria are available in the ACHIEVE listing at clinicaltrials.gov.

The ACHIEVE study will enroll people with DM1 who:

  • Are between the ages of 18 and 49
  • Have grip myotonia that creates a delayed hand opening time of 2 seconds or more
  • Can safely perform a set of muscle function assessments without the use of assistive or mobility devices such as canes or walkers
  • Do not have advanced heart or breathing problems
  • Avoid becoming pregnant or fathering a child during the study
  • Can read, understand and agree to the Informed Consent Form (ICF) and participate in the study without assistance from others

There are other conditions that DM1 community members must meet to participate in the ACHIEVE study. Additional information on the ACHIEVE study is available in the ACHIEVE listing at clinicaltrials.gov.

6. Are the costs of participating in this trial covered?

Study treatment and all study tests and procedures are provided at no cost to study participants. Participants will be reimbursed for approved study-related travel expenses. Participant support services will be available to participants who choose to have study-related travel and accommodations arranged for them at no cost.

7. Can I quit during the trial if I want?

Participating in the ACHIEVE study is completely voluntary. Participants can leave at any time. Leaving the study will have no impact on care received outside the study.

8. I wish to participate in the trial. Who should I consult for advice?

People who are interested in the ACHIEVE study and want to know more can:

  • Talk to their myotonic dystrophy-treating physician or neurologist about this trial
  • Contact a doctor working on the ACHIEVE study

9. If I am approached to participate in the study, by whom and how will I be notified?

Potential participants will be contacted by members of the clinical research team at each site.

10. When are the first results scheduled?

Dyne anticipates sharing initial information on safety, tolerability and splicing data from the multiple ascending dose (MAD) portion of the ACHIEVE study in the second half of 2023. The ACHIEVE study is designed to run for up to three years.

For more information:

Individuals interested in the ACHIEVE study who would like to know more can:

  • Talk to their myotonic dystrophy treating physician about the study
  • Contact a doctor working on the ACHIEVE study; for a list of participating physicians and sites, visit the ACHIEVE study page at clinicaltrials.gov.

As of May 2023

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