Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

This important study is looking into a new treatment, VESLETEPLIRSEN (also known as SRP-5051), for Duchenne muscular dystrophy. Specifically, it's for male patients whose condition can be helped by a treatment method called 'exon 51-skipping'. The study has two main parts. The first part will help researchers figure out the safest and most effective dose of the medicine. The second part will then use this chosen dose to see how well the medicine works by measuring changes in a crucial muscle protein called dystrophin. Throughout the study, the research team will also be carefully monitoring any side effects to make sure the treatment is as safe as possible.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Sarepta Therapeutics Inc.

Enrolment target

Start

06 May 2024

Duchenne Muscular Dystrophy

What is this study about?

This study is looking into a new medication called VESLETEPLIRSEN for Duchenne muscular dystrophy (DMD). DMD is a serious genetic condition that causes muscles to weaken over time. This particular treatment is designed for a specific group of patients whose DMD is caused by a genetic problem that can be helped by a process called 'exon 51-skipping'.

The study has been planned in two main stages. The first stage, called Part A, will focus on finding the right amount (dose) of VESLETEPLIRSEN to give. The researchers will start with a small amount and gradually increase it, always keeping a close eye on how patients are reacting and if there are any side effects. Once they've found the best dose that is both safe and has a good chance of working, they will move to the second stage.

In the second stage, Part B, more patients will receive the chosen dose. Here, the main goal is to see how much the medicine can help produce a vital muscle protein called dystrophin. People with DMD don't make enough of this protein, which is essential for healthy muscle function. The study will also continue to monitor for any side effects throughout this longer period.

Key takeaways

Tests a new medicine (VESLETEPLIRSEN) for Duchenne muscular dystrophy.
Specifically for males over 18 with 'exon 51-amenable' DMD.
The study has two parts: finding the right dose and then seeing how well it works.
Aims to increase dystrophin, a vital muscle protein.
Requires regular clinic visits and health checks over potentially several years.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, you would need to be a male who is at least 18 years old. The study is specifically designed for men with Duchenne muscular dystrophy (DMD).

Additionally, your type of Duchenne muscular dystrophy must be suitable for a treatment approach known as 'exon 51-skipping'. This is a specific genetic feature that can be identified through special tests. If you meet these basic criteria, the study team would then carry out further checks to ensure the study is right for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you male?
Are you 18 years old or older?
Do you have Duchenne muscular dystrophy?
Has your doctor told you that your DMD type is suitable for 'exon 51-skipping' treatment?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would receive the study medication, VESLETEPLIRSEN, which is given into your bloodstream (intravenously). The study would involve regular visits to a clinic over a period that could last for quite some time – up to 75 weeks in the first part, and potentially much longer, up to 304 weeks (about 5.5 years), in the second part. During these visits, the medical team would collect blood and urine samples to check how the medicine is moving through your body and how much is there. They would also take muscle samples (biopsies) from time to time to see if the medicine is producing more dystrophin protein in your muscles. You'd also have regular health checks and tests to monitor your safety and overall well-being. The team would carefully explain the full schedule and what to expect at each visit before you make any decisions.

Potential risks and benefits

Participating in research always comes with potential benefits and risks. A possible benefit of joining this study is that you might be among the first to receive a new medicine that could potentially help with Duchenne muscular dystrophy. However, it's also possible that the medicine might not work for you, or it could cause side effects, some of which might be serious. The medical team will carefully explain all known and potential risks before you decide to join. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Netherlands
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Unverified
Spain
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Unverified
Belgium
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Unverified
Germany
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Unverified
Italy

Common questions

What is Duchenne muscular dystrophy (DMD)?

DMD is a genetic condition that causes muscles to become weak and damaged over time.

What does 'exon 51-skipping' mean?

It's a way to treat DMD for some patients by fixing a specific genetic mistake so the body can make more of an important muscle protein called dystrophin.

What is the main goal of this study?

The study aims to find the best dose of a new medicine (VESLETEPLIRSEN) and see if it helps create more dystrophin protein in muscles.

How long would I be in the study?

The study could last from over a year in the first stage to several years in the second stage, depending on which part you join.

Will I get the actual medicine or a dummy treatment?

This study focuses on giving the actual medicine at different doses. Please ask the study team for full details about how treatment is given.