Image

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

Join a study for Duchenne Muscular Dystrophy treatment research.

Recruiting
4-18 years
Male
Phase 1/2

This study is about trying a new medicine, **WVE-N531**, for boys with **Duchenne Muscular Dystrophy (DMD)**. DMD is a condition that makes muscles weak over time. The study checks if the medicine is safe and how it affects the body. The medicine is given through an **IV (intravenous)**, which means it goes directly into the blood through a vein.

**Key Points:**

  • **Participation Length:** The study has different parts, and Part C will last several months with muscle checks at the start and after 24 weeks.
  • **Visits and Tests:** Participants will have regular check-ups and undergo muscle biopsies, which involve taking a small piece of muscle for testing.
  • **Eligibility:** Participants must have a specific genetic change in their DMD gene and be on stable medication for at least 6 months.

New patients can join Part C, where they will be closely watched for safety and changes in muscle protein levels called **dystrophin**. Joining the study means regular hospital visits and tests, but it could help find new ways to treat DMD. Always talk to your doctor before deciding to join a study.

Study details
    Duchenne Muscular Dystrophy

NCT04906460

Wave Life Sciences Ltd.

7 March 2026

Step 1 Get in touch with the nearest study center
We have submitted the contact information you provided to the research team at {{SITE_NAME}}. A copy of the message has been sent to your email for your records.
Would you like to be notified about other trials? Sign up for Patient Notification Services.
Sign up

Send a message

Enter your contact details to connect with study team

Investigator Avatar

Primary Contact

  Other languages supported:

First name*
Last name*
Email*
Phone number*
Race
Ethnicity
Other language

FAQs

Learn more about clinical trials

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.

How long does a clinical trial take place?

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

Do I get compensated for taking part in clinical trials?

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

How safe are clinical trials?

Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.