Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Double-blind, Placebo-Controlled, Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

This research study is about two potential new medicines, SRP-4045 and SRP-4053, for Duchenne muscular dystrophy (DMD). It's for boys with DMD whose condition could benefit from a specific type of treatment called 'exon skipping 45 or 53'. The main goal is to find out if these medicines can help improve how quickly boys can climb stairs. The study will also look at other things like how far they can walk in six minutes and how quickly they can get up from the floor. Participants will receive either one of the study medicines or a placebo (a dummy treatment, like salt water) initially. It's a 'Phase 3' study, which means it’s a big study to confirm if the medicines are effective and safe before they might be approved for wider use.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Sarepta Therapeutics Inc.

Enrolment target

Start

22 Jul 2024

Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping.

What is this study about?

This study is called a 'clinical trial' and it's looking into two new medicines, SRP-4045 and SRP-4053, for Duchenne muscular dystrophy (DMD). DMD is a condition that causes muscles to become weaker over time. These medicines are designed for a specific type of DMD where the genetic fault can be *skipped* over to help the body make a more complete dystrophin protein, which is vital for healthy muscles.

The main question the researchers want to answer is: Can either of these new medicines help boys with DMD climb stairs faster after about two years of treatment? They will measure this by seeing how quickly participants can go up four steps. They will also look at other important things, like how far participants can walk in six minutes, how quickly they can get up from the floor, and their overall movement and daily activity scores. They will also check if the medicines help the body make more dystrophin protein.

This is a 'Phase 3' study, which is a big and important step in testing new medicines. It means the medicines have already been tested in smaller groups and appear promising. In this phase, a larger group of people takes part to get a clearer idea of how well they work and if they are safe. Some participants will receive one of the study medicines, while others will get a placebo (a treatment that looks like the medicine but contains no active drug). This helps researchers fairly compare the effects of the real medicine.

Key takeaways

Tests two new medicines, SRP-4045 and SRP-4053, for Duchenne muscular dystrophy.
Specifically targets boys whose Duchenne is suitable for 'exon 45 or 53 skipping' therapies.
Aims to see if the medicines improve ability to climb stairs and other physical functions.
Involves regular clinic visits, assessments, and potentially muscle biopsies.
Initial part is 'double-blind' comparing medicines to a placebo, followed by an 'open-label' extension.

Who may be eligible?

This study is looking for boys who have Duchenne muscular dystrophy. To be considered, your specific type of Duchenne muscular dystrophy must be suitable for a treatment approach called 'exon 45 skipping' or 'exon 53 skipping'. This means that the particular genetic change in your DNA that causes Duchenne needs to be one that these medicines are designed to target.

There isn't a specific age limit mentioned, so boys of various ages might be able to participate as long as they meet the other requirements. Because Duchenne muscular dystrophy primarily affects boys, only male participants are being included in this study.

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 a male?
Do you have a diagnosis of Duchenne muscular dystrophy?
Has your doctor confirmed that your Duchenne is suitable for 'exon 45 skipping' or 'exon 53 skipping' treatment?
Are you able to participate in regular clinic visits and physical assessments?

Answer every question to see your result.

What does participation involve?

If you join this study, you would first go through some checks to make sure the study medicines are right for you. For part of the study (about 96 weeks or just under two years), you would either receive one of the new medicines (SRP-4045 or SRP-4053) or a placebo, which looks like the medicine but has no active drug. Neither you nor the study team would know which one you are receiving during this time – this is called 'double-blind'. After this period, all participants would have the option to receive the active study medicine in an 'open-label extension', meaning everyone would know they are getting the medicine.

Throughout the study, you would attend regular visits to the clinic. At these visits, doctors and nurses would perform various assessments to see how you are doing. This includes measuring how quickly you can climb four steps, how far you can walk in six minutes, and how fast you can get up from the floor. They would also take blood samples and potentially muscle biopsies (a small sample of muscle tissue) to check levels of dystrophin protein. These tests help the researchers understand if the medicines are working and if they are safe. The total duration of participation, including the initial blind period and potential open-label extension, could be quite long, possibly several years.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as potentially receiving a new medicine before it's widely available or getting close monitoring from medical experts. However, there are also potential risks involved, including possible side effects from the study medicines or the procedures, and the possibility that the medicine may not work for you. It's also possible to receive a placebo which has no active treatment. You would be fully informed of all known risks before deciding to join, and you always have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (9)

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

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Unverified
Belgium
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Unverified
Ireland
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Unverified
Poland
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Unverified
Bulgaria
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Unverified
Denmark
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Unverified
Italy
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Unverified
Spain
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Unverified
Czechia
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Unverified
Hungary

Common questions

What is 'exon 45 or 53 skipping'?

It's a way some medicines work to correct specific genetic mistakes in Duchenne muscular dystrophy, allowing the body to make a more complete muscle protein called dystrophin.

What does 'double-blind' mean?

It means that during the first part of the study, neither you nor your doctor will know if you are getting the active medicine or a dummy treatment (placebo). This helps make the study fair.

Will I have to take muscle biopsies?

Yes, the study description mentions taking small muscle samples (biopsies) to check for changes in the dystrophin protein, which is an important part of understanding if the medicine is working.

How long would I be in the study?

The first main part of the study lasts about 96 weeks (almost two years), but there's a chance to continue on the active medicine in an 'open-label' period after that, which could make your participation longer.

Can girls participate in this study?

No, this study is specifically for boys, as Duchenne muscular dystrophy primarily affects males.