Clinical Trials
Dyne Therapeutics is focused on advancing innovative, potentially life-transforming therapeutics for people living with genetically driven muscle diseases.
Before bringing new medicines to patients, clinical trials, a type of research that studies the safety and efficacy of potential therapeutics in people, must be conducted. The U.S. Food and Drug Administration (FDA) and other global healthcare regulatory agencies review data on investigational medicines before the clinical trial can start. Clinical trials would not be possible without the collaboration of the people living with the diseases we aim to treat, healthcare professionals and advocacy groups.
If you or someone you know is interested in participating in one of our clinical trials, please see our active studies below:
The DELIVER trial is a Phase 1/2 global clinical trial evaluating DYNE-251, 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, which is designed to be registrational, is enrolling ambulant and non-ambulant males with DMD who are ages 4 to 16 and have mutations amenable to exon 51 skipping therapy. The primary endpoints are to evaluate safety, tolerability and change from baseline in dystrophin levels as measured by Western blot. Secondary endpoints include measures of muscle function, exon skipping, and pharmacokinetics.
Visit the FAQs link below and the DELIVER trial page at www.clinicaltrials.gov (NCT05524883) and www.clinicaltrialsregister.eu/ctr-search/search (2021-005478-24) to learn more about the DELIVER clinical trial.
FAQ
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 and a 96-week long-term extension. The trial, which is designed to be registrational, 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.
Visit the FAQs link below and the ACHIEVE trial page at www.clinicaltrials.gov (NCT05481879) to learn more about the ACHIEVE trial.
FAQIf you have questions about Dyne Therapeutics’ clinical trials, please contact us at clinicaltrials@dyne-tx.com.
Patient Access
Expanded access (sometimes referred to as “managed access” or “early access”) programs (EAP) refer to the use of an investigational medicine when the primary purpose is to diagnose, monitor, or treat a patient’s disease rather than to obtain the kind of information about the drug that is generally derived from clinical trials.
- EAPs are appropriate when substantial scientific evidence has been established to support both the safety and the efficacy of an investigational medicine in patients.
- An investigational medicine addresses a specific disease and is not yet approved for commercial use or is only approved for testing in clinical trials for that disease by regulatory agencies, such as the FDA or EMA.
Currently, Dyne does not have investigational medicines available through an expanded access program. We will continue to assess this as our clinical programs progress.
Dyne believes that participating in clinical trials is the best way for patients to access investigational medicines. These carefully controlled, rigorous scientific studies are needed to obtain regulatory approval, which will be critical for enabling the broadest access to treatment for a specific condition.
- Dyne will strive to design trials that are inclusive of as many patients as possible, but we recognize that not every patient will be eligible for these trials.
- For those patients who have completed a Dyne clinical trial, it is our plan, when supported by the scientific data, to provide access to the investigational medicine until it is approved and available locally.
Dyne conducts regular review of its policies to ensure conformity with applicable laws and regulations, and we reserve the right to revise this policy at any time.
