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Study on AOC 1020 for Adults with Facioscapulohumeral Muscular Dystrophy (FSHD)

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 Facioscapulohumeral Muscular Dystrophy (FSHD), a genetic disorder that causes progressive muscle weakness, particularly affecting the face, shoulders, and upper arms. The study is testing a new treatment called AOC 1020, which is a special type of medication known as a monoclonal antibody. This antibody is designed to target specific proteins in the body and is linked to a small piece of genetic material called siRNA, which aims to reduce the activity of a gene associated with FSHD. The treatment is given through an intravenous infusion, meaning it is administered directly into the bloodstream.

The purpose of the study is to evaluate the safety and tolerability of different doses of AOC 1020 in adults with FSHD. Participants will receive either the study medication or a placebo, which is a substance with no active ingredients, to compare the effects. The study will monitor participants for any side effects and measure how the body processes the medication. This includes checking the concentration of the medication in the blood and its presence in muscle tissue over time.

Participants in the study will undergo regular health assessments and tests to track their response to the treatment. The study aims to gather important information that could lead to new treatment options for people living with FSHD. The trial is expected to continue until 2025, providing valuable insights into the potential benefits and risks of AOC 1020 for managing this condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit

    Upon joining the study, you will attend an initial visit at the study site. During this visit, your eligibility will be confirmed based on specific criteria, such as a confirmed diagnosis of facioscapulohumeral muscular dystrophy (FSHD) through genetic testing, your ability to walk 10 meters, and the presence of muscle weakness in both upper and lower body.

    A healthcare professional will discuss the study details with you, and you will have the opportunity to ask questions. You will also undergo a physical examination and provide a medical history.

  2. Step 2

    Treatment administration

    You will receive the study medication, AOC 1020, or a placebo, which is a saline solution. The medication is administered intravenously, meaning it is given through a vein.

    The dosage and frequency of administration will be determined by the study protocol, and you will be informed of the schedule during your visits.

  3. Step 3

    Follow-up visits

    You will attend regular follow-up visits at the study site. These visits are designed to monitor your health and the effects of the treatment.

    During these visits, you may undergo various tests, such as blood tests and muscle biopsies, to assess the concentration of the medication in your body and its effects on your muscles.

  4. Step 4

    End of treatment evaluation

    At the end of the treatment period, you will have a final evaluation. This will include a comprehensive assessment of your health and any changes in your condition.

    The study team will discuss the results with you and provide information on any further steps or follow-up care that may be necessary.

Who can join the trial?

6 criteria

  • Must have a confirmed diagnosis of FSHD1 or FSHD2 through genetic testing. (FSHD stands for Facioscapulohumeral muscular dystrophy, a genetic muscle disorder.)
  • Must be able to walk 10 meters, either on their own or with the help of devices like a cane, walking stick, or braces.
  • Must have at least one muscle area that can be used for a biopsy. (A biopsy is a small sample of tissue taken for testing.)
  • Must have muscle weakness in both the upper and lower parts of the body, as determined by the study doctor.
  • Both males and females can participate.
  • Participants must be within the age range specified by the study.

Who cannot join the trial?

3 criteria

  • Patients who do not have Facioscapulohumeral muscular dystrophy (FSHD) cannot participate. This is a specific muscle disorder.
  • Patients who are not within the specified age range cannot participate. The age range is not specified here, but it is important to check if you fit the required age group.
  • Patients who are part of a vulnerable population may not be eligible. This means people who might need special protection or care.
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Investigated drugs

AOC 1020 is a medication being tested to see if it is safe and well-tolerated by people with a condition called Facioscapulohumeral Muscular Dystrophy (FSHD). This condition affects the muscles, causing them to weaken over time. AOC 1020 is given through an intravenous infusion, which means it is delivered directly into the bloodstream through a vein. The study aims to understand how the body processes this medication and how it affects the muscles. Researchers are also looking at whether AOC 1020 can help improve muscle function in people with FSHD. The goal is to find out if this medication can be a potential treatment for this muscle condition.

What is already known about the treatment

AOC 1020 – AOC 1020 is administered intravenously, meaning it is given directly into a vein through an injection. It is currently being studied in clinical trials to assess its safety and effectiveness for treating facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder. The main goal of this medication is to help manage symptoms of FSHD by targeting specific molecular pathways involved in the disease. AOC 1020 belongs to a class of drugs designed to interact with the body's cells at a molecular level, potentially altering the progression of the disease.

Investigated diseases

Facioscapulohumeral muscular dystrophy (FSHD) – Facioscapulohumeral muscular dystrophy is a genetic muscle disorder characterized by progressive muscle weakness and wasting. It primarily affects the muscles of the face, shoulders, and upper arms. The disease often begins with weakness in the facial muscles, leading to difficulties in closing the eyes or smiling. Over time, the shoulder blades may protrude due to weakness in the shoulder muscles, and the upper arms may also become weak. The progression of muscle weakness can vary significantly among individuals, with some experiencing mild symptoms and others having more severe muscle involvement. The condition is caused by genetic changes that affect the regulation of certain genes involved in muscle function.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2022-502096-32-00Protocol codeAOC 1020-CS1Estimated enrolment72 patientsSponsorAvidity Biosciences Inc.

sourced from the EU Clinical Trials Register and site verification

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

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