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A study testing the safety and effects of ADS-019 in adults aged 18 to 65 years with type 1 myotonic dystrophy

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What is this trial about?

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

This study involves people with Type 1 Myotonic Dystrophy, a genetic condition that causes muscle weakness and stiffness, particularly difficulty relaxing muscles after use. The study will test a new medicine called ARO-DM1, which is given through an infusion into a vein. Some participants will receive a placebo instead of the actual medicine. The medicine is being developed to target the genetic cause of this condition.

The purpose of this study is to find out if ARO-DM1 is safe and well-tolerated in people with Type 1 Myotonic Dystrophy, and to understand how the body processes the medicine and how it affects the disease. The study will also look at whether the medicine can improve the condition in some participants. This is a dose-escalating study, which means that different groups of participants will receive different amounts of the medicine to find the best dose. The study will measure how often side effects occur and how severe they are, as well as how the medicine moves through the bloodstream over time.

Participants in this study will be adults between 18 and 65 years old who have a confirmed genetic diagnosis of the condition with a specific genetic pattern. They must have visible signs of muscle stiffness and be able to walk independently for at least 10 meters, though they may use braces. The study involves receiving either the study medicine or placebo through infusions, and participants will be monitored throughout the study period for safety and how well the treatment works.

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 treatment phase

    Upon joining the study, you will be assigned to receive either ARO-DM1 or placebo. The placebo is a solution containing sodium chloride 9 milligrams per milliliter, which is a salt water solution with no active medication.

    The treatment will be administered through intravenous infusion, which means the medication will be delivered directly into your vein through a needle inserted into your arm.

    The study follows a dose-escalating design, meaning different groups of participants may receive different doses of the medication to determine the safest and most effective amount.

  2. Step 2

    Treatment administration schedule

    You will receive either single doses or multiple doses of ARO-DM1 or placebo, depending on which group you are assigned to.

    The specific dosage, frequency, and duration of administration will be determined by the group you are assigned to in this dose-escalating study.

    If you are assigned to Cohort 5, you will receive multiple doses of the study medication.

  3. Step 3

    Safety and response monitoring

    Throughout the study, your safety will be monitored by tracking any adverse events, which are any unwanted or unexpected medical occurrences that happen during the trial.

    Blood samples will be collected at various times to measure the levels of ARO-DM1 in your bloodstream. This is called pharmacokinetics, which helps determine how your body processes the medication.

    The study will also assess pharmacodynamics, which examines how the medication affects your body and the disease.

  4. Step 4

    Efficacy assessments

    If you are assigned to Cohort 5, additional assessments will be conducted to evaluate how well the medication works in treating Type 1 Myotonic Dystrophy.

    These assessments will measure changes in your condition and symptoms over the course of the study.

  5. Step 5

    Study completion

    The study will continue until the end of study visit, when final safety assessments will be completed.

    After receiving your last dose of study medication or completing the final study visit, you must continue using highly effective contraception plus condoms for at least 90 days.

    You must not donate sperm or eggs during the study and for at least 90 days after your last dose or the end of the study, whichever occurs later.

    The estimated duration of the study extends until September 2026.

Who can join the trial?

6 criteria

  • You must be 18 to 65 years old at the time you agree to join the study.
  • You must have a genetically confirmed diagnosis of Type 1 Myotonic Dystrophy, which means a laboratory test has proven you have this condition by showing a specific genetic change with a CTG repeat length of 100 or more. CTG repeats are abnormal sections of genetic material that cause this disease.
  • You must have visible signs of Type 1 Myotonic Dystrophy that your doctor can observe, including myotonia, which is muscle stiffness that makes it hard to relax your muscles. This stiffness should be significant enough that it takes at least 2 seconds for you to open your hand after making a fist.
  • Your symptoms related to Type 1 Myotonic Dystrophy must have started after you were 12 years old.
  • You must be able to walk at least 10 meters on your own during the screening visit. You may use orthoses, which are supportive devices like leg braces, but you cannot use canes or walkers.
  • If you are able to have children, you must agree to use highly effective birth control methods along with a condom during the study and for at least 90 days after the study ends or after your last dose of the study medicine, whichever comes later. You must also agree not to donate sperm or eggs during the study and for at least 90 days after the study ends or after your last dose, whichever comes later.

Who cannot join the trial?

1 criterion

  • No exclusion criteria have been provided in the available study information
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Investigated drugs

ARO-DM1 is an investigational medication being studied for the treatment of Type 1 Myotonic Dystrophy. This medication is designed to work in the body by targeting specific genetic material involved in this condition. The study will test different amounts of this medication given either as a single dose or multiple doses to see how safe it is, how well the body processes it, and whether it has any beneficial effects on the disease.

What is already known about the treatment

ARO-DM1 – ARO-DM1 is an investigational medication currently being studied in early-phase clinical trials for the treatment of Type 1 Myotonic Dystrophy, a genetic disorder that causes progressive muscle weakness and wasting. This experimental drug is administered through injection and works at the molecular level by targeting and reducing the abnormal genetic material that causes the disease, using a technology that can silence specific disease-causing genes. The medication is being tested to determine its safety, how the body processes it, and whether it can effectively improve symptoms in patients with this condition.

Investigated diseases

Type 1 Myotonic Dystrophy – Type 1 Myotonic Dystrophy is a genetic disorder that affects multiple body systems, particularly the muscles. The condition causes progressive muscle weakness and wasting, especially in the lower legs, hands, neck, and face. People with this disease experience myotonia, which means their muscles have difficulty relaxing after contracting. The disorder also affects other organs including the heart, eyes, and endocrine system. Symptoms typically worsen gradually over time, leading to increased muscle weakness and stiffness. The severity and age of onset can vary significantly among affected individuals, ranging from birth to adulthood.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-513579-42-00Protocol codeARODM1-1001Estimated enrolment90 patientsSponsorSarepta Therapeutics Inc.

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