WVE-210201 – Exon 51 Skipping

A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne (WVE-DMDX51-001)

Questions and Answers

What stage is this research?

This is a Phase 1 clinical trial that is actively recruiting.

What is the goal or purpose of this study?

The primary objective is to evaluate the safety and tolerability of a single dose of the investigational drug WVE-210201 compared to placebo. WVE-210201 is for patients with Duchenne who are 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 Duchenne that is amenable to an exon 51 skipping therapy
  • Male, aged 5 to 18 years old
  • Ambulatory or non-ambulatory

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?

Patients will receive a single dose of the study drug (WVE-210201 or placebo).

Wave intends to initiate an open-label extension (OLE) study, and patients who complete participation in the Phase 1 clinical trial may be eligible for participation in the OLE.

Where does this study take place?

This study will be conducted at multiple sites in the US, Europe, and Canada. Since this is the first clinical study of WVE-210201, it is important that patients live within close proximity to one of the sites for participation.

Will I get paid for participating in this study?

There is no compensation for participating in the trial, but reasonable travel 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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