Dyne Therapeutics is discovering and advancing potentially life-transforming therapeutics for people living with genetically driven neuromuscular diseases.
Clinical trials, a type of research that studies the safety and efficacy of potential therapeutics in people, must be conducted before bringing new medicines to patients. The U.S. Food and Drug Administration (FDA) and other global healthcare regulatory agencies review preclinical data on investigational medicines before clinical trials 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:
DELIVERDELIVER is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The multiple ascending dose (MAD) portion of the study resulted in the selection of a registrational dose and regimen of 20 mg/kg every four weeks. A registrational expansion cohort to support potential regulatory submissions for expediated approvals, including accelerated approval in the U.S., is fully enrolled. The primary endpoint for this cohort is change from baseline in dystrophin protein levels measured by Western blot. For more information on the DELIVER trial, visit www.clinicaltrials.gov (NCT05524883) and euclinicaltrials.eu (EUCT2023-510351-31-00).
FAQ
ACHIEVEACHIEVE is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of DYNE-101 in patients with myotonic dystrophy type 1 (DM1). The multiple ascending dose (MAD) portion of the study resulted in the selection of a registrational dose and regimen of 6.8 mg/kg every eight weeks. A registrational expansion cohort was added to support potential regulatory submissions for expedited approvals, including accelerated approval in the U.S. The primary endpoint for this cohort is the change from baseline in the Composite Alternative Splicing Index (CASI). For more information on the ACHIEVE trial, visit www.clinicaltrials.gov (NCT05481879) and euclinicaltrials.eu (EUCT2023-510353-42-00).
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.
