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Enrolling by invitationPHASE2INTERVENTIONAL

Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy

This is an important ongoing study for boys with Duchenne muscular dystrophy. It's called an 'open-label extension' study, which means everyone involved knows they are receiving the study medicine, WVE-N531. The main goal is to understand how safe WVE-N531 is over a longer period and how well boys tolerate it. Researchers will also look at how effective the medicine is and how it acts in the body. Only boys who have already participated in a previous study of WVE-N531 can join this current study. This allows doctors to continue monitoring the treatment's effects and ensure the safety of those involved, providing crucial information for future development of this potential medicine.

At a glance

Status
Enrolling by invitation
Phase
PHASE2
Sponsor
Wave Life Sciences USA, Inc.
Enrolment target
175
Start
28 Dec 2025
Estimated completion
01 Mar 2029
Duchenne Muscular Dystrophy

What is this study about?

This study is for boys who have Duchenne muscular dystrophy and have already been involved in an earlier study of a medicine called WVE-N531. Think of it as a continuation, allowing doctors to keep a close eye on the boys who are taking this medicine over a longer period. The main aim is to understand the long-term safety, meaning how well boys tolerate the medicine and if there are any side effects when taken for an extended time.

Doctors will also be assessing if the medicine is still effective and how it acts in the boys' bodies. This information is gathered by carrying out regular checks and tests. This type of study, called an "open-label extension," is common in medical research. It allows patients who have done well on a study medicine to continue receiving it while more data is collected. This helps researchers build a full picture of the medicine's effects over time.

All the information collected from this study, combined with previous studies, is very important. It helps researchers decide if WVE-N531 is safe and helpful enough to be considered for wider use in the future. Their findings will contribute to the understanding of Duchenne muscular dystrophy and potential new treatments.

Key takeaways

  • This study is an extension for boys already taking WVE-N531 for Duchenne muscular dystrophy.
  • The main goals are to check long-term safety and how well the body handles the medicine.
  • Participants will receive WVE-N531 through an IV every four weeks for about two years.
  • There will be regular check-ups and monitoring throughout the study and for nearly a year after treatment.
  • This research helps gather important information for possible future treatments for Duchenne.

Who may be eligible?

You might be able to join this study if you are a boy with Duchenne muscular dystrophy and have already taken part in a previous study involving the medicine WVE-N531. It's important that you haven't had any serious problems from the medicine in the past, and your general health hasn't significantly worsened since your last dose or visit in the prior study.

However, you would likely not be able to join if your doctor believes that taking WVE-N531 now could put your health at risk, or if you have developed other significant health issues not related to Duchenne muscular dystrophy since your last study participation.

Also, if you've used any recreational substances (excluding alcohol and nicotine) within two months before your first dose in this study, or are unable to stop using them for the study's duration, you would not be eligible. If there's been a long break (more than 31 days) between your last dose in the previous study and the start of this one, the study doctors would need to discuss your situation specially.

Quick self-check
  • Am I a boy with Duchenne muscular dystrophy?
  • Have I already taken part in a study for WVE-N531?
  • Have I been generally healthy without major new problems since my last WVE-N531 dose?
  • Have I avoided recreational substances (other than alcohol/nicotine) for at least two months?
  • Am I willing to avoid recreational substances for the entire study?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you will continue to receive the study medicine, WVE-N531. It will be given directly into your veins (through an IV) every four weeks. Each dose will be 10 mg for every kilogram you weigh. This treatment will continue for about 96 weeks, which is roughly two years.

Throughout this time, you will have regular hospital visits for assessments. These will involve doctors checking your health, looking for any side effects, and doing tests to see how the medicine is working and how your body is handling it. The study will also keep monitoring your safety for about 11 months after you have received your final dose of the medicine.

Potential risks and benefits

Participating in this study means you could continue to receive a medicine that might help manage your Duchenne muscular dystrophy, and you'll be closely monitored by medical professionals. This also helps other boys with Duchenne by providing important information about the long-term effects of WVE-N531. However, like all medicines, WVE-N531 may have side effects, and some may not be known yet. There's no guarantee the medicine will benefit you, and some tests or procedures during the study might cause minor discomfort. You are always free to leave the study at any time, for any reason, without it affecting your medical care.

Locations (3)

  • Istiklal Hospital/ Clinical Research Unit
    Amman, Jordan
  • The Specialty Hospital (TSH)/ Advanced Clinical Center
    Amman, Jordan
  • Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust
    Headington, United Kingdom

Common questions

What is Duchenne muscular dystrophy?

Duchenne muscular dystrophy is a serious genetic condition that causes muscles to weaken and waste away over time.

What does 'Open-Label' mean?

Open-label means that both you (and your parents) and your doctors will know that you are receiving the study medicine, WVE-N531. It's not a 'blinded' study where some people get a placebo.

Will I get WVE-N531 or a dummy medicine (placebo)?

In this specific study, everyone who participates will receive the active study medicine, WVE-N531. There is no dummy medicine (placebo).

Why is this study only for boys who were in a previous study?

This is an 'extension' study, meaning it's designed to follow up on people who have already started the treatment to understand its long-term effects and safety. It's not a study for new patients.

How long will I be in the study?

You will receive the medicine for about two years, and then your health will be monitored for approximately another 11 months after your last dose.

How to find out more

Official trial record

Always speak to your GP or specialist before deciding to take part in a study.

Interested in taking part?

Register your interest

Share your details and the research team for "Open-Label Extension Study of WVE-N531 in Patients With Duch…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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