RecruitingPHASE1, PHASE2INTERVENTIONAL

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

This study is testing a new medicine called WVE-N531 for boys and young men with Duchenne muscular dystrophy. The main goals are to check if the medicine is safe, how well the body takes it in and uses it, and if it helps improve muscle function. It's for patients who have a specific genetic change that the medicine is designed to target. The study has different parts. Some parts have already been completed, and patients in those parts are continuing to receive the medicine. A new part of the study (Part C) is now open and looking for new participants. Taking part involves receiving the medicine, having some tests, and regular check-ups to see how you are doing.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Wave Life Sciences USA, Inc.

Enrolment target

Start

28 Sep 2021

Estimated completion

24 Apr 2027

Duchenne Muscular Dystrophy

What is this study about?

This study, called FORWARD-53, is testing a new medicine named WVE-N531 for Duchenne muscular dystrophy (DMD). DMD is a condition that causes muscles to get weaker over time. This medicine is designed to help patients who have a specific type of genetic change in their body that prevents them from making a vital protein called dystrophin. Dystrophin is like the glue that holds muscle cells together, and without it, muscles become damaged.

The main aim of this study is to find out if WVE-N531 is safe to use and how well people tolerate it. Researchers also want to understand how the medicine works in the body and if it has any positive effects on muscle strength and function. They will be looking at whether the medicine can help the body produce more dystrophin, which could eventually lead to stronger muscles.

This study is divided into different stages, called parts. Parts A and B have already been completed, and many of those patients are still receiving the medicine. Now, a new section called Part C is starting, and it's looking for new participants to join. This means that more boys and young men with DMD will have the opportunity to try this potential new treatment.

Key takeaways

This study is testing a new medicine (WVE-N531) for Duchenne muscular dystrophy.
It aims to check the medicine's safety, how it works, and if it improves muscle function.
It's for boys aged 4-18 with a specific genetic change in their DMD gene.
Participation involves IV medicine infusions, muscle biopsies, and regular health checks.
You can stop participating at any time.
Part C is currently enrolling new patients who are able to walk.

Who may be eligible?

This study is for boys and young men aged between 4 and 18 years old who have Duchenne muscular dystrophy. Importantly, your doctor will need to confirm that your specific genetic change related to DMD is the type that this medicine is designed to help. This means the medicine works by 'skipping' a particular part of your genetic code (called exon 53).

You would need to be able to have regular muscle biopsies, which are small samples taken from your muscle, usually in your arm. Your heart and lung health would also be checked to make sure they are stable and meet certain requirements. If you are already taking steroids for DMD, you need to have been on a steady dose for a significant period before joining.

For Part C, new patients are being enrolled. You wouldn't be able to join if you can't walk, as this part of the study is specifically for those who can walk. You also need to meet other health criteria to ensure your safety throughout the study.

Quick self-check

I am a boy or young man between 4 and 18 years old.
I have Duchenne muscular dystrophy with a genetic change that can be helped by 'exon 53 skipping'.
I am able to have small muscle samples (biopsies) taken.
My heart and lung health are stable, and my doctor agrees.
If I currently take steroids, my dose has been stable for at least 3 months.
For Part C, I am currently able to walk.

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

What does participation involve?

If you join the study, you will receive the study medicine, WVE-N531, through a drip into your vein (intravenous or IV). Initially, the medicine was given every two weeks, but now it's given once every four weeks.

Taking part involves regular visits to the clinic for check-ups and assessments. You will have muscle biopsies at the beginning of the study and again after about 24 weeks of treatment. These biopsies help the doctors see if the medicine is encouraging your body to make dystrophin. You'll also have tests to check your safety, such as blood tests, and assessments of your physical abilities, like how you move and function. Your heart and lung function will be monitored regularly.

The study medicine will be given for at least 24 weeks, and potentially longer, depending on the study part and your progress. There will be follow-up appointments for up to 10 months after your last dose to make sure you are still doing well.

Potential risks and benefits

Participating in research studies like this can have potential benefits. You might gain access to a new medicine that is not yet available to everyone, and it could potentially help improve your Duchenne muscular dystrophy. Your participation also helps doctors learn more about the condition and how to treat it, which can help others in the future. However, there are also potential risks. The study medicine might cause side effects, some of which could be serious. The muscle biopsies involve a minor surgical procedure and can cause pain or bruising. There's also the time commitment and inconvenience of regular hospital visits and tests. It's important to remember that you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

Arkansas Children's Hospital
Little Rock, United States· Recruiting
Rare Disease Research LLC
Atlanta, United States· Recruiting
Istiklal Hospital/ Clinical Research Unit
Amman, Jordan· Recruiting
The Specialty Hospital (TSH)/ Advanced Clinical Center
Amman, Jordan· Recruiting
Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust
Headington, United Kingdom· Recruiting

Common questions

What is Duchenne muscular dystrophy?

Duchenne muscular dystrophy is a genetic condition that causes muscles to become weak and damaged over time due to a lack of a protein called dystrophin.

What is WVE-N531?

WVE-N531 is the name of the new medicine being tested in this study. It's designed to help the body make dystrophin for specific genetic changes.

How often will I receive the medicine?

If you join Part C, you will receive the medicine once every four weeks through a drip into your vein.

What are muscle biopsies?

A muscle biopsy is when a small sample of muscle is carefully taken, usually from your arm, to be examined under a microscope. It helps doctors see if the medicine is working.

Can girls participate in this study?

No, this specific study is only open to boys and young men with Duchenne muscular dystrophy.

How to find out more

Clinical Operations

clinicaltrials@wavelifesci.com 855-215-4687 Official trial record

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