AuthorisedPhase I and Phase II (Integrated)- First administration to humansInterventional

A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who are ≥18 to ≤65 Years

This research study, called ARO-DM1, is looking into a new medicine for people living with Type 1 Myotonic Dystrophy. We want to find out if this new medicine is safe for people to take and how well their bodies handle it. Researchers will also study how the medicine affects the disease. This first-stage study involves a small number of people and is focused on understanding side effects and proper dosing. Some participants will receive the new medicine, while others will receive a placebo – a harmless substance that looks like the medicine but has no active ingredients. Participants must be between 18 and 65 years old. This important step helps us learn if ARO-DM1 could be a helpful treatment in the future.

At a glance

Status

Authorised

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Sarepta Therapeutics Inc.

Enrolment target

Start

29 Jan 2026

Type 1 Myotonic Dystrophy

What is this study about?

This study is about a new medicine, ARO-DM1, for people who have Type 1 Myotonic Dystrophy. Myotonic Dystrophy Type 1 is a genetic condition that causes muscles to become weak and waste away over time. It can affect many parts of the body.

The main goal of this particular study is to check if ARO-DM1 is safe for people to take. Researchers will be carefully looking for any side effects and how strong these side effects might be. They also want to understand how the medicine moves through the body and how the body gets rid of it. This helps them work out the best dose and how often it should be given. We hope this research will bring us closer to new treatments for this condition.

This is an early-stage study, meaning it's one of the first times this medicine is being tested in humans. It's an important step for new medicines to go through these stages to make sure they are safe and potentially effective before they can be used more widely.

Key takeaways

This study is testing a new medicine (ARO-DM1) for Type 1 Myotonic Dystrophy.
The main goals are to check the medicine's safety and how the body handles it.
Participants will receive either the new medicine or a dummy medicine (placebo).
It's an early-stage study, focused on understanding side effects and dosing.
You must be an adult, aged 18 to 65, to be considered for participation.

Who may be eligible?

This study is designed for adults. To take part, you need to be aged 18 years or older. There is no upper age limit listed, which means people older than 65 might also be considered, but the current range specified is up to 65 years.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Are you 65 years old or younger?
Have you been diagnosed with Type 1 Myotonic Dystrophy?
Are you able to attend regular appointments and follow study instructions?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will likely have several visits to a clinic or hospital. These visits will involve various checks, such as blood tests, physical examinations, and possibly tests to see how your muscles are working. You will receive either the study medicine, ARO-DM1, or a placebo (a dummy medicine) as an injection. Neither you nor the study team will know which you are receiving. The study involves regular follow-up appointments to monitor your health, any side effects, and how your body is responding to the treatment. The total length of your participation, including the treatment period and follow-up, will be decided by the study doctors.

Potential risks and benefits

Taking part in a study like this might offer a chance to try a new treatment that isn't available yet, which could potentially help your condition. However, there are also risks, as the medicine is still being tested and could cause unexpected side effects. Some side effects might be uncomfortable or even serious. You will be closely monitored by doctors and nurses throughout the study. Remember, joining is always your choice, and you can withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Spain
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Unverified
Germany
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Unverified
Belgium
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Unverified
Italy

Common questions

What is Type 1 Myotonic Dystrophy?

It's a genetic condition that causes muscles to weaken and waste away over time, affecting movement and other body functions.

What is a 'placebo'?

A placebo is a substance that looks exactly like the study medicine but doesn't contain any active ingredients. It helps researchers compare the effects of the new medicine.

What does 'dose-escalating' mean?

It means that different participants will receive different doses of the medicine, starting with small amounts and gradually increasing, so researchers can find the safest and most effective dose.

Why is this an 'early-stage' study?

This is one of the first times the medicine is being tested in people. It's focused on checking safety and how the body handles the medicine before it moves to larger studies.

Will I know if I'm getting the real medicine or the placebo?

No, during the study, neither you nor the study team will know whether you are receiving the active medicine or the placebo. This is to ensure fair results.