AuthorisedTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (delpacibart zotadirsen) for the Treatment of DMD with Gene Mutations Amenable to Exon 44 Skipping

This research study is looking at a new medicine called AOC 1044, for Duchenne muscular dystrophy (DMD). It's for boys and men whose DMD is caused by a specific type of genetic change, where a 'jump' (exon 44 skipping) might help. The study will give some participants the new medicine and others a harmless substance (a placebo), but no one will know which they are getting until after the study finishes. This helps scientists fairly measure how well the medicine works and if it has any side effects. The main goal is to see if AOC 1044 can improve how muscles work over time, especially how fast people can walk, and to check if it's safe to use.

At a glance

Status

Authorised

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Avidity Biosciences Inc.

Enrolment target

Start

20 May 2026

Duchenne muscular dystrophy

What is this study about?

This study is specifically designed for boys and men living with Duchenne muscular dystrophy (DMD). DMD is a serious genetic condition that causes muscles to become weaker over time. This particular study is for people whose DMD is caused by a specific type of genetic change, which doctors call a 'gene mutation amenable to Exon 44 skipping'. This means their faulty gene might be helped by a new approach called 'exon skipping'. The new medicine being tested, AOC 1044, aims to 'skip' over the faulty part of the gene, which could allow the body to make a more functional protein and potentially slow down or improve the muscle weakening.

The study involves giving the medicine through a drip (intravenously). Some participants will receive the active medicine (AOC 1044), while others will receive a 'placebo', which looks exactly like the medicine but contains no active ingredients. Neither the participants nor their doctors will know who is receiving which, which is called a 'double-blind' study. This is a common and important way to make sure the results are fair and accurate, as it prevents anyone's expectations from influencing the outcomes.

The main aim over the 54 weeks (about a year) is to carefully watch if the new medicine improves a person's ability to walk or move, specifically looking at how quickly they can cover certain distances. Researchers will also be checking for any side effects and other changes in health. By doing this rigorous testing, scientists hope to find out if AOC 1044 is a safe and effective treatment that could help improve the lives of people with DMD.

Key takeaways

Tests new medicine (AOC 1044) for Duchenne muscular dystrophy (DMD).
Only for boys and men with specific 'Exon 44 skipping' genetic change.
Compares medicine to a harmless placebo; no one knows who gets what.
Aims to check if medicine improves muscle function and is safe.
Involves regular clinic visits for infusions and health checks over 54 weeks.

Who may be eligible?

This study is particularly looking for boys and men with Duchenne muscular dystrophy (DMD). To be considered, your DMD must be caused by a specific genetic change that doctors believe could be helped by a treatment called 'Exon 44 skipping'. If you're not sure about your specific genetic change, your doctor can help you understand if this study might be suitable for you.

There are no age limits mentioned for this study, meaning participants of all ages who meet the other criteria may be considered. However, the study is only open to male participants because DMD almost exclusively affects males.

Beyond these main points, doctors will also check other health information to make sure it's safe for you to take part. This might include reviewing your medical history, current health, and any other medicines you are taking. It's important that you or your caregiver discuss all your health details with the study team.

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?
Do you have Duchenne muscular dystrophy (DMD)?
Has your doctor confirmed your DMD is due to a genetic change suitable for 'Exon 44 skipping'?
Are you willing and able to attend regular clinic visits for infusions and tests?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would receive the study medicine (either AOC 1044 or the placebo) through a drip into your arm (intravenous infusion). This will happen regularly over 54 weeks, which is about a year. You will have regular visits to the study clinic for these infusions and for various tests.

During these visits, the study team will carry out several assessments. These will include checking your muscle function, such as how quickly you can climb stairs or walk a certain distance. They will also collect blood and urine samples, and do heart tests like ECGs, to monitor your health and look for any side effects. You might also be asked to fill out questionnaires about your quality of life. After the main 54-week study, there's a chance you could continue into an 'open-label extension', where everyone receives the active medicine.

The total duration of the main part of the study is 54 weeks, but the extension phase could last longer.

Potential risks and benefits

Taking part in any medical study has both potential benefits and risks. You might benefit from the new medicine if it proves to be effective in slowing down the progression of DMD or improving muscle function. However, there's also a chance you could receive the placebo and not get the active medicine during the main study period. Potential risks include side effects from the medicine, or discomfort from procedures like blood tests or infusions. The study team will closely monitor you for any changes in your health. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (7)

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

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Unverified
Italy
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Unverified
Germany
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Unverified
France
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Unverified
Spain
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Unverified
Belgium
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Unverified
Poland
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Unverified
Netherlands

Common questions

What is 'Exon 44 skipping'?

It's a way to try and 'skip over' a faulty section of a gene that causes DMD, so the body can make a more useful protein for the muscles.

What does 'double-blind' mean?

It means neither you nor your doctor will know if you're getting the new medicine or the placebo. This helps get accurate results.

How long will I be in the study?

The main part of the study lasts for 54 weeks (about a year), with the possibility of joining an extension phase afterwards.

Will I have to go to the clinic often?

Yes, you will need to go to the clinic regularly for infusions and health check-ups throughout the study.

What is a placebo?

A placebo looks just like the study medicine but doesn't contain any active ingredients. It's used to compare against the real medicine.