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Study on AOC 1001 for Treating Myotonic Dystrophy Type 1 in Patients

Fast replyInvestigationalNo placebo
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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 focused on studying a condition called Myotonic Dystrophy Type 1, which is a genetic disorder that affects muscle function. The study is testing a new treatment called AOC 1001, which is given as a solution through an intravenous infusion. This means the treatment is delivered directly into the bloodstream through a vein. AOC 1001 is a special type of medication that combines a humanized antibody with a small piece of genetic material designed to target and reduce the effects of the disease.

The purpose of the study is to evaluate how effective AOC 1001 is in improving hand function in people with Myotonic Dystrophy Type 1. Participants in the study will receive either the AOC 1001 treatment or a placebo, which is a substance with no active medication. The study will last for a period of 54 weeks, during which participants will have regular visits to monitor their progress and assess any changes in their condition.

Throughout the study, researchers will focus on measuring changes in hand function, as well as other aspects of muscle strength and daily activities. The goal is to determine if AOC 1001 can help improve the quality of life for those living with Myotonic Dystrophy Type 1. Participants will be closely monitored to ensure their safety and to gather important information about the treatment's effects.

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

    Joining the study

    Upon joining the study, eligibility is confirmed based on criteria such as age (16 to 65 years, or 18 to 65 years in Denmark and Germany), a clinical and genetic diagnosis of myotonic dystrophy type 1, and the ability to walk independently for at least 10 meters.

  2. Step 2

    Treatment administration

    The treatment involves the administration of AOC 1001, a solution for infusion, given intravenously. This medication is a humanized antibody linked to a specific genetic material designed to target myotonic dystrophy type 1.

    The infusion is administered under controlled conditions to ensure safety and effectiveness.

  3. Step 3

    Monitoring and assessment

    Throughout the study, regular assessments are conducted to monitor changes in hand function, specifically the time taken to open the hand, known as video Hand Opening Time (vHOT).

    Additional assessments include hand grip strength, muscle strength scores, and overall health and quality of life evaluations.

  4. Step 4

    Evaluation of results

    Primary evaluation occurs at Week 30, focusing on changes in hand function from the start of the study.

    Secondary evaluations include various muscle strength and health assessments at Week 30 and Week 54, providing a comprehensive view of the treatment's impact.

  5. Step 5

    Completion of the study

    The study is expected to conclude by October 2026, with all data collected and analyzed to determine the efficacy and safety of AOC 1001 for treating myotonic dystrophy type 1.

Who can join the trial?

3 criteria

  • Age between 16 and 65 years old. In Denmark and Germany, the age range is 18 to 65 years old.
  • Have a clinical and genetic diagnosis of **Myotonic Dystrophy Type 1**. This means having a specific genetic change called a **CTG repeat length** of 100 or more.
  • Be able to walk on your own for at least 10 meters during the screening. You can use supports like orthoses or ankle braces if needed.

Who cannot join the trial?

5 criteria

  • Patients who have a different condition than Myotonic Dystrophy Type 1 cannot participate. This is a specific genetic disorder that affects muscles and other systems in the body.
  • Individuals who are not within the specified age range for the study are excluded. The age range is not specified here, but it means that only people of certain ages can join.
  • People who are not part of the specified clinical trial groups are excluded. This means that only certain groups of people, based on specific criteria, can participate.
  • Both females and males can participate, but if someone does not fit the gender criteria, they cannot join.
  • Individuals who are considered part of a vulnerable population are excluded. This term refers to groups of people who might be at a higher risk of harm or exploitation, such as children, pregnant women, or those with certain disabilities.
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Investigated drugs

AOC 1001 is an investigational medication being studied for its potential to improve hand function in patients with Myotonic Dystrophy Type 1. It is administered intravenously, meaning it is given directly into a vein. The study aims to determine how effective and safe this medication is for treating this specific condition.

What is already known about the treatment

AOC 1001 – AOC 1001 is administered intravenously and is currently being studied in a Phase 3 clinical trial for its effectiveness and safety in treating Myotonic Dystrophy Type 1. This medication is not yet widely available in medical practice, as it is still under investigation. The main therapeutic indication for AOC 1001 is to improve hand function in individuals with Myotonic Dystrophy Type 1. At the molecular level, AOC 1001 works by targeting specific genetic components to potentially correct or mitigate the effects of the disease. It is classified pharmacologically as a therapeutic agent under investigation for neuromuscular disorders.

Investigated diseases

Myotonic Dystrophy Type 1 – This is a genetic disorder characterized by progressive muscle wasting and weakness. It often begins in adulthood and affects the muscles of the face, neck, and limbs. Individuals with this condition may experience prolonged muscle contractions and have difficulty relaxing certain muscles after use. Over time, the disease can lead to muscle stiffness and weakness, impacting daily activities such as hand function. Other symptoms may include cataracts, heart conduction problems, and endocrine changes. The severity and progression of symptoms can vary widely among affected individuals.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-511378-60-00Protocol codeAOC 1001-CS3Estimated enrolment150 patientsSponsorAvidity Biosciences Inc.

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