RecruitingPHASE1, PHASE2INTERVENTIONAL

Study of ATX-01 in Participants With DM1

This research study is looking into a new medicine, ATX-01, for people who have Myotonic Dystrophy Type 1 (DM1). The main purpose is to find out if ATX-01 is safe to use and how well people tolerate it. Some participants will receive ATX-01, while others will get a placebo, which looks like the study medicine but has no active ingredients. This helps researchers understand the true effects of ATX-01. The study will involve giving either a single dose or several doses of the medicine. ATX-01 works by targeting tiny genetic instructions in the body called microRNAs to try and help with DM1 symptoms.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

ARTHEx Biotech S.L.

Enrolment target

Start

15 Oct 2024

Estimated completion

01 Jul 2027

Myotonic Dystrophy 1

What is this study about?

Myotonic Dystrophy Type 1 (DM1) is a genetic condition that causes muscles to become weak and waste away. It can also cause muscles to stay contracted for too long, a symptom called myotonia. This study is testing a new medicine called ATX-01, which is designed to help with DM1. Researchers want to know if ATX-01 is safe for people to take and how well their bodies cope with it. This kind of study is an early step in developing new treatments.

The study will involve two main parts. In one part, called 'single-ascending dose,' participants will receive just one dose of ATX-01 or a placebo (a dummy medicine). In the other part, called 'multiple-ascending dose,' participants will receive three doses of ATX-01 or the placebo. By comparing ATX-01 to a placebo, the researchers can better understand the medicine's effects.

ATX-01 works by affecting specific tiny instructions within our cells that are involved in the development of DM1. It’s hoped that by changing these instructions, the medicine could help improve the symptoms of DM1. This study is an important step in seeing if this new approach could be a useful treatment.

Key takeaways

This study is testing a new medicine called ATX-01 for Myotonic Dystrophy Type 1.
The main goal is to check if ATX-01 is safe and well-tolerated.
Participants will receive either ATX-01 or a placebo (dummy medicine).
It involves either a single dose or three doses of the study medicine.
You must be 18-64 years old and able to walk 10 metres to join.
You can leave the study at any time.

Who may be eligible?

To join this study, you must be between 18 and 64 years old. You need to have a confirmed diagnosis of Myotonic Dystrophy Type 1. Also, you should be able to walk 10 metres (about 33 feet) without needing walking aids like a cane or walker, though ankle-foot supports are allowed.

Researchers will also check for a specific type of muscle stiffness in your grip that lasts for more than 3 seconds.

You cannot join if you have congenital DM1 (meaning you had symptoms from birth). You also can't take part if you've recently used certain other medicines for myotonia, like mexiletine, within the last three weeks or so. There are also specific muscle strength requirements for your ankle muscles.

Quick self-check

Are you between 18 and 64 years old?
Do you have a confirmed diagnosis of Myotonic Dystrophy Type 1 (DM1)?
Can you walk 10 metres (about 33 feet) without needing walking aids (like a cane, but ankle-foot supports are okay)?
Have you not received a diagnosis of congenital DM1 (DM1 from birth)?
Have you avoided medicines like mexiletine for myotonia for at least the last three weeks?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

Taking part in this study means you will receive either the new medicine, ATX-01, or a placebo, which looks identical but contains no active ingredients. You won't know which one you're getting, and neither will the study team, to ensure the results are unbiased. The study involves giving either a single dose or three doses of the study medicine.

Throughout the study, you will have several visits to the clinic where doctors and nurses will check your health. This will include physical exams, blood tests, and other assessments to monitor how you're responding to the medicine and to check for any side effects. The total duration of your participation, including follow-up visits, will be explained in detail by the study team.

Potential risks and benefits

Participating in this study might offer a potential benefit of receiving an experimental new medicine that could help with Myotonic Dystrophy Type 1. However, as ATX-01 is new, there may be unknown side effects or the medicine might not be effective. Like any medicine, ATX-01 could cause side effects, which will be carefully monitored by the study team. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (12)

UCLA
Los Angeles, United States· Recruiting
University of Florida
Gainesville, United States· Recruiting
University of Iowa Health Care - Department of Neurology
Iowa City, United States· Recruiting
University of Kansas Medical Center, Department of Neurology
Fairway, United States· Recruiting
Virginia Commonwealth University
Richmond, United States· Recruiting
Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
Chicoutimi, Canada· Recruiting
Institute of Myology
Paris, France· Recruiting
The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
Milan, Italy· Recruiting
Fondazione Policlinico A. Gemelli- IRCCS
Rome, Italy· Recruiting
Radboudumc
Nijmegen, Netherlands· Recruiting
Hospital Universitario Donostia
Donostia / San Sebastian, Spain· Recruiting
St. George's University Hospital
London, United Kingdom· Recruiting

Common questions

What is Myotonic Dystrophy Type 1 (DM1)?

DM1 is a genetic condition that causes muscles to weaken, waste away, and stiffen. It can affect many parts of the body.

What is ATX-01?

ATX-01 is a new, experimental medicine being tested for DM1. It works by targeting specific genetic instructions in your body cells.

What is a placebo?

A placebo is a 'dummy' medicine that looks like the study medicine but has no active ingredients. It helps researchers compare the effects of the actual medicine.

Will I know if I'm getting ATX-01 or the placebo?

No, neither you nor the study team will know if you're receiving ATX-01 or the placebo. This is called 'blinding' and helps ensure reliable study results.

What does 'single-ascending dose' and 'multiple-ascending dose' mean?

In the single-dose part, you'll get one dose. In the multiple-dose part, you'll get three doses over time. This helps researchers study the medicine's effects with different amounts.

How to find out more

Project Manager

clinical@arthexbiotech.com +34676229821 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.