Duchenne Muscular Dystrophy in Patients with Gene Mutations Amenable to Exon 44 Skipping Treated with Delpacibart Zotadirsen

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What is this study about?

This clinical trial is being done in Duchenne muscular dystrophy, a genetic disease that causes the muscles to become weak over time. The study is testing AOC 1044, also called delpacibart zotadirsen, given by vein, and comparing it with placebo. The purpose of the study is to see whether this treatment can help improve muscle function and to check its safety.

The study includes people with Duchenne muscular dystrophy whose gene changes may allow a treatment approach called exon 44 skipping, which means the medicine is designed to help the body work around a missing or faulty part of the gene. The study is planned in two parts. In the first part, treatment is assigned by chance and neither the study team nor the participant knows which treatment is given. Later, there is an open-label extension, which means the treatment is given openly after the blinded part ends. During the study, the medicine is given at regular visits and health checks are done over time.

1 study entry and treatment assignment

After you join the study, you are assigned to one of two treatment groups in a random way. This means the treatment is chosen by chance.

You receive either aoc 1044 or placebo. The placebo is sodium chloride 0.9% w/v, which is a salt water solution with no active study drug.

The study is double-blind, which means that you do not know which treatment you receive, and the study team does not know either during this part of the study.

2 treatment period

If you receive aoc 1044, it is given as an intravenous infusion. Intravenous means the medicine is given through a vein.

The dose of aoc 1044 is 5 mg/kg. This means 5 milligrams for each kilogram of body weight.

If you receive placebo, you receive sodium chloride 0.9% w/v by the same intravenous route.

The source information does not state how often the medicine is given or how long treatment lasts.

3 study assessments during the trial

During the study, your muscle function is checked over time. The main measure is the change in ttr velocity from the start of the study to week 54. TTR means the time needed to turn around, and velocity means speed.

Other checks may also be done during the study, including creatine kinase levels, 4 stair climb velocity, 10mWRT velocity, SV95C, NSAA, DMD-qol, patient/caregiver global impression of severity, patient/caregiver global impression of change, and qmt.

10mWRT means the 10-meter walk/run test, which measures how fast you can walk or run a short distance.

NSAA means the North Star Ambulatory Assessment, a set of movement checks used in duchenne muscular dystrophy.

DMD-qol means a questionnaire about quality of life in duchenne muscular dystrophy.

qmt means quantitative muscle testing, which measures muscle strength.

The study also checks for teaes, which means treatment-emergent adverse events. These are unwanted health effects that appear or get worse after treatment starts.

Your vital signs, laboratory tests, and ecg results are also monitored. ecg means electrocardiogram, a test that records the heart’s electrical activity.

4 open-label extension

After the blinded part of the study, there is an open-label extension.

Open-label means the treatment is known during this part of the study.

The source information does not give the specific treatment schedule for this extension.

5 study end

The study follows you through the planned trial period, with the main outcome measured up to week 54.

The overall study period is planned to run until the study end date listed in the source information.

Who Can Join the Study?

The participant and the participant’s legally authorized representative must have given written informed consent and/or assent, meaning they must have signed and dated the study permission forms.
The participant must be a male who can walk on his own (ambulatory), with a documented medical diagnosis of Duchenne muscular dystrophy or clear signs of the condition starting at age 6 years or younger.
The participant must have a genetic test that shows an out-of-frame dystrophin gene mutation that can be treated with exon 44 skipping. This means the gene change must be the type the study treatment is designed to target.
The participant must be 7 to 16 years old, including both 7 and 16, when consent is signed.
The participant must have been taking a stable dose of corticosteroids for at least 6 months before Day 1, and this dose must be expected to stay the same during the study. Corticosteroids are anti-inflammatory medicines often used to help slow muscle weakness.

Who Cannot Join the Study?

Previous treatment with a cell therapy or gene therapy product. Gene therapy means a treatment that changes or replaces genetic material, and cell therapy means a treatment using living cells.
Treatment with another oligonucleotide within 6 months before giving informed consent. An oligonucleotide is a short piece of genetic material used as a medicine. This does not include COVID-19 RNA vaccines.
If using growth hormone or testosterone, the treatment must have been stable for at least 1 month before informed consent; otherwise, the person cannot participate. A stable treatment means the dose has not been changed recently.
If using givinostat, the treatment must have been stable for at least 6 months before informed consent; otherwise, the person cannot participate. Givinostat is a medicine used for certain muscle conditions.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Katholieke Universiteit te Leuven	Leuven	Belgium
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany

Other Sites

Site Name	City	Country
Centre Hospitalier Regional De La Citadelle	Liege	Belgium
Ospedale San Raffaele S.r.l.	Milan	Italy
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Hopital Beaujon	Clichy	France
Centre Hospitalier Universitaire De Nantes	Nantes	France
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego	Warsaw	Poland
Ljsjt Ujfuqzkpoewa Meslrkn Cesleng (jjlun	Leiden	The Netherlands
Fajlxnxjq Ppkm Lv Ivvaepprdrufm Buxrwiwyg Dow Hfxcjawe Uygbbnkcnxdul Ly Pru	Madrid	Spain
Kvldbiiv dio Ubivoykyecph Msyccxkx Ayu	Munich	Germany
Uknbtyctywjstuyvrrygl Eomhz Anv	Essen	Germany
Cjpzgd Cazwain Ntqn	Milan	Italy
Fpajwcuqsj Sqyce Smxw	Sevilla	Spain
Ulgfgfhpyyckmk Cpqchgd Krbiyxovu	Gdansk	Poland

Trial status

Country	Status	Recruitment Start
Belgium	Not yet recruiting	26.05.2026
France	Not yet recruiting	26.05.2026
Germany	Not yet recruiting	26.05.2026
Italy	Not yet recruiting	26.05.2026
Poland	Not yet recruiting	26.05.2026
Spain	Not yet recruiting	26.05.2026
The Netherlands	Not yet recruiting	26.05.2026

Trial locations

AOC 1044 is the study medicine being tested in this trial. It is given through a vein as an infusion. The goal of the study is to see whether it can help improve muscle function in people with Duchenne muscular dystrophy caused by gene changes that may respond to exon 44 skipping. Researchers are also checking how safe it is and how well people tolerate it.

Investigated diseases:

Duchenne muscular dystrophy

Duchenne muscular dystrophy – Duchenne muscular dystrophy is an inherited disease that causes the muscles to become weaker over time. It usually begins in early childhood and mainly affects the muscles used for walking, climbing, and other movements. As the disease progresses, muscle tissue is gradually replaced by weaker tissue, and movement becomes increasingly difficult. The weakness often spreads to other muscle groups as the condition advances.

Trial ID:

2025-523087-20-00

Protocol code:

AOC 1044-CS3

Trial Phase:

Therapeutic confirmatory (Phase III)

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Duchenne Muscular Dystrophy in Patients with Gene Mutations Amenable to Exon 44 Skipping Treated with Delpacibart Zotadirsen

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?