DYSTANCE 51: Phase 2/3 study of Suvodirsen (Formerly WVE-210201) for people with Duchenne amenable to Exon 51 Skipping

A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of Suvodirsen in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)

Questions and Answers

What stage is this research?

This is a Phase 2/3 clinical trial

What is the goal or purpose of this study?

The primary objective is to evaluate the efficacy and safety of the investigational drug suvodirsen compared to placebo in ambulatory boys with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

Who is funding this study?

This study is funded by Wave Life Sciences.

Who is eligible to participate in this study?

Patients and/or caregivers should discuss the eligibility requirements of the clinical trial with their doctor

Key eligibility criteria include:

  • Genetically-confirmed diagnosis of DMD that is amenable to an exon 51 skipping therapy
  • Male, aged 5 to 12 years old
  • Ambulatory, able to walk independently for at least 10 meters in 20 seconds or less

What do I have to do if I decide to participate in this study?

Patients and/or caregivers should discuss potential participation in the study with their doctor.

How long will this study last, and will I have access to the drug/treatment once the study has ended?

The duration of treatment during the placebo-controlled portion of the trial is 48 weeks

Patients may be eligible to enter into an open-label phase of the trial to receive ongoing treatment with suvodirsen after they complete the placebo-controlled portion of the phase 2/3 clinical trial.

Where does this study take place?

We currently expect that this study will be conducted in the U.S., Europe, Canada, Japan, and Australia

Will I get paid for participating in this study?

There is no compensation for participating in the trial, but reasonable expenses incurred due to the study requirements will be reimbursed.

Patients and/or caregivers should discuss specific questions regarding travel reimbursement in the clinical trial with the study investigator.

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