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

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 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. Step 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. Step 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. Step 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 trial?

5 criteria

  • 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 trial?

4 criteria

  • 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.
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Investigated drugs

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.

What is already known about the treatment

  • Sodium Chloride 0.9% w/v

    This is a sterile salt solution given as an infusion into a vein, and it is commonly used in medicine as a placebo or as a basic fluid for hydration and dilution of other medicines. It is a well-established product in medical practice and the medical literature, with a long history of safe use when given correctly. It does not treat Duchenne muscular dystrophy directly and has no specific disease-targeted action; its main role in trials is to act as a comparison treatment. Pharmacologically, it is an isotonic electrolyte solution that helps maintain fluid and salt balance in the body.

  • Delpacibart zotadirsen

    This medicine is given by intravenous infusion, meaning it is slowly delivered through a vein, usually in a hospital or clinic setting. It is an investigational treatment, so its full place in medicine is still being studied, but it has been developed for Duchenne muscular dystrophy in people whose gene changes may allow exon 44 skipping. Its main goal is to help the body make a more useful form of dystrophin, a protein needed for muscle strength, by binding to the messenger RNA and changing how the gene message is read. Pharmacologically, it is an antisense oligonucleotide, a targeted genetic medicine designed to modify RNA rather than act like a traditional drug.

Investigated diseases

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 detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2025-523087-20-00Protocol codeAOC 1044-CS3Estimated enrolment70 patientsSponsorAvidity Biosciences Inc.

sourced from the EU Clinical Trials Register and site verification

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