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Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

Testing DYNE-101 for Myotonic Dystrophy Type 1 safety and effectiveness.

Recruiting
16 years and older
All
Phase 3

This study is checking how well a new medicine called zeleciment basivarsen (DYNE-101) works for people with myotonic dystrophy type 1 (DM1), a muscle and genetic condition. It will also look at how safe and easy it is to use. The study has three parts: a Screening period (up to 8 weeks), a Placebo-Controlled Period (48 weeks), and a Long-Term Extension Period (24 weeks). A group of experts will regularly review the safety of participants. To join, you need to be able to walk a short distance independently and have a specific genetic test result.

  • The study lasts about a year and a half, including all three periods.
  • Participants must not have had major surgeries recently, except for certain devices.
  • Some medications are not allowed before the study starts, like GLP-1 agonists.

Think about the study's length, any medication restrictions, and if you meet the health requirements before deciding to participate.

Study details
    Myotonic Dystrophy Type 1 (DM1)
    DM1
    Myotonic Dystrophy
    Steinert Disease
    Steinert

NCT07486934

Dyne Therapeutics

25 April 2026

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What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

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The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

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Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

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Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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