Clinical Research Alert: Phase 1/2 Study of DYNE-101 in Individuals with Myotonic Dystrophy Type 1

Researchers at Dyne Therapeutics, Inc. are seeking individuals with myotonic dystrophy type 1 (DM1) to participate in a phase 1/2 clinical trial (ACHIEVE) to evaluate the safety and efficacy of the investigational therapy DYNE-101 to treat DM1. DM1 is caused by a defect in the DMPK gene that leads to the production of abnormally long DMPK RNA transcripts, which interfere with protein synthesis and disrupt normal cellular functions. DYNE-101 is designed to reduce levels of the abnormal DMPK RNA in muscle cells. The current study will examine the ability of DYNE-101 treatment to produce improvements in muscle and central nervous system (CNS) function in people with DM1.

The study

This is a phase 1/2 randomized, placebo-controlled trial. This means that participants will be randomly assigned to receive the study drug, DYNE-101, or an inactive placebo control. The total duration of the study for participants will be up to four years. This will include a 60-day screening period and a 24-week placebo-controlled treatment period. Following this, all participants will be eligible to receive 24-weeks of DYNE-101 treatment and enroll in a 168-week extension period, in which they can continue to receive DYNE-101. During the study, participants will have regularly scheduled visits at their study site.

The drug will be administered by intravenous (in the vein) infusion. The effects will be evaluated using a number of tests and procedures including but not limited to: blood and urine collection, a heart function test (ECG), a physical exam conducted by the study doctor, muscle function and strength tests, including change from baseline in myotonia as measured by video hand opening time (vHOT), biopsies, and questionnaires to capture any changes in a participant’s DM1-related symptoms or activities.

Study criteria

To be eligible for the research study, individuals must meet the following inclusion criteria:

• Diagnosis of DM1 with trinucleotide repeat size >100
• Age of onset of DM1 muscle symptoms ≥12 years
• Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator
• Hand grip strength and ankle dorsiflexion strength
• Able to complete 10-MWRT and 5×STS at screening without the use of assistive devices

Individuals may not be eligible to participate if they are affected by another condition or receiving another treatment that might interfere with the ability to undergo safe testing.

Please visit this link for the full listing of inclusion and exclusion criteria.

Interested in participating?

Travel support is available for eligible participants.

To learn more about the study or inquire about participation, contact the closest participating hospital using Clinical Trials.gov,  EU Clinical Trials, or DYNE ACHIEVE FAQs.