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Study of ADS-010 for Adults with Facioscapulohumeral Muscular Dystrophy Type 1

Fast replyRegistered drugNo 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 Type 1 (FSHD1), a genetic disorder that causes muscle weakness, particularly in the face, shoulders, and upper arms. The study will evaluate a new treatment called ARO-DUX4, which is a solution for injection developed by Arrowhead Pharmaceuticals Inc. The treatment involves the use of a substance known as ADS-010, which is administered through an intravenous infusion. The study will also use Sodium Chloride as a comparison treatment, which is a common chemical used in medical settings.

The purpose of the study is to assess the safety and tolerability of ARO-DUX4 in adult patients with FSHD1. Participants will receive either single or multiple doses of the treatment, and the study will monitor how the body processes the drug, known as pharmacokinetics, and how the drug affects the body, known as pharmacodynamics. The study will take place over a period of time, with participants being observed for any side effects or changes in their condition.

Throughout the study, participants will undergo various assessments, including blood tests and possibly imaging tests like MRI to evaluate muscle condition. The study aims to gather information on the incidence, frequency, and severity of any treatment-related side effects. This information will help determine the potential benefits and risks of ARO-DUX4 for individuals with FSHD1. The study is expected to continue until 2026, with recruitment starting in 2024.

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

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a genetic confirmation of facioscapulohumeral muscular dystrophy Type 1 (FSHD1) and a clinical severity score between 3 and 8.

    An MRI is performed to identify an eligible lower extremity muscle for biopsy. The muscle must have a specific fat fraction between 10% and 40%.

  2. Step 2

    Consent and baseline procedures

    Written informed consent is obtained before any study-specific procedures.

    Baseline procedures include a 12-lead ECG to ensure no abnormalities that could compromise safety, and a body mass index (BMI) assessment to confirm it is between 18.0 and 30.0 kg/m².

  3. Step 3

    Treatment administration

    The treatment involves the administration of ARO-DUX4 via intravenous infusion. The dosage is adjusted in a dose-escalating manner to evaluate safety and tolerability.

    Sodium chloride is also administered as part of the solution for injection.

  4. Step 4

    Monitoring and follow-up

    Regular monitoring is conducted to assess the incidence, frequency, and severity of any treatment-emergent adverse events.

    Plasma and urinary excretion of ARO-DUX4 are measured to understand how the body processes the medication.

  5. Step 5

    End of study evaluation

    The study concludes with an evaluation of the primary and secondary endpoints. This includes a final assessment of adverse events and pharmacokinetic data.

    The study duration varies, with Part 1 ending at Day 90 and Part 2 at Day 360.

Who can join the trial?

9 criteria

  • Must have a **genetically confirmed** diagnosis of **FSHD1**. This means a test has shown changes in your genes that confirm you have this condition.
  • Have a **clinical severity score** between 3 and 8. This score measures how severe your condition is, on a scale from 0 to 10.
  • Must have a suitable lower leg muscle for a **biopsy**. A biopsy is a small sample of muscle tissue that will be checked. This is determined by an **MRI** scan, which is a type of imaging test.
  • Be a male or a nonpregnant, nonlactating female aged 18 to 70 years. This means you should not be pregnant or breastfeeding and fall within this age range.
  • Must be able and willing to sign a **written informed consent**. This means you agree to participate after understanding the study details.
  • Have a **body mass index (BMI)** between 18.0 and 30.0. BMI is a measure of body fat based on height and weight.
  • Have a normal **12-lead ECG** at screening. An ECG is a test that checks your heart's electrical activity.
  • If you can have children, you and your partner must agree to use effective birth control during the study and for at least 12 weeks after it ends. Men must not donate sperm during this time.
  • Must be willing and able to follow all study assessments and stick to the schedule.

Who cannot join the trial?

4 criteria

  • Individuals who do not have facioscapulohumeral muscular dystrophy (FSHD) cannot participate. FSHD is a genetic muscle disorder that affects the muscles of the face, shoulder blades, and upper arms.
  • Participants must be within a specific age range, typically adults, to be eligible.
  • Both males and females can participate, but certain health conditions or factors may exclude some individuals.
  • People who are considered part of a vulnerable population, such as those with certain health conditions or disabilities, may not be eligible.
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Investigated drugs

ARO-DUX4 is an investigational medication being studied for its potential to treat facioscapulohumeral muscular dystrophy Type 1 (FSHD1). This study aims to understand how safe and tolerable the medication is for patients, as well as how the body processes and responds to it. The trial involves giving patients increasing amounts of the medication to see how it affects their condition.

What is already known about the treatment

ARO-DUX4 – ARO-DUX4 is administered through injection and is currently being studied in clinical trials for its safety and effectiveness. It is primarily being investigated for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder. The medication works by targeting and reducing the expression of the DUX4 gene, which is believed to play a key role in the development of FSHD. ARO-DUX4 is classified as a gene-silencing therapy, aiming to modify the underlying genetic cause of the disease.

Investigated diseases

Facioscapulohumeral muscular dystrophy (FSHD) – This is a genetic muscle disorder characterized by progressive muscle weakness and wasting. It primarily affects the muscles of the face, shoulders, and upper arms. Over time, individuals may experience difficulty with facial expressions, lifting objects, and raising their arms. The progression of muscle weakness can vary significantly among individuals, with some experiencing mild symptoms and others facing more severe challenges. The condition is caused by genetic changes that affect the DUX4 gene, leading to muscle cell damage. Symptoms often begin in adolescence or early adulthood, but the rate of progression can differ widely.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2023-509748-89-00Protocol codeARODUX4-1001Estimated enrolment70 patientsSponsorSarepta Therapeutics Inc.

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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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