A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is an inherited condition that causes muscles to become weak and stiff, making it hard to relax your grip or move easily. This worldwide study is looking at an experimental medicine called AOC 1001 to find out if it is safe and effective. Participants will be placed into groups by chance. One group receives AOC 1001 through a drip into a vein (intravenously), while another receives a dummy treatment (a saline placebo) that contains no active medicine. Neither you nor your study doctor will know which one you are receiving. Over 54 weeks, doctors will test how quickly you can open your hands, measure your walking speed, test your grip strength, and ask about your quality of life to see if the drug helps manage symptoms.
At a glance
What is this study about?
Myotonic dystrophy type 1 (often shortened to DM1) is a genetic condition that affects the muscles and other parts of the body. One of the most common and frustrating symptoms is 'myotonia', which means muscles have trouble relaxing after use. For example, after shaking someone's hand or holding a door handle, a person's fingers may feel stuck in a tight grip. Over time, DM1 can also cause progressive muscle weakness, tiredness, and difficulties with everyday tasks.
Currently, there are few treatments that target the root cause of DM1. This study is testing an investigational drug called AOC 1001. It is designed to work inside muscle cells to address the underlying genetic message that causes the condition. Scientists hope that by targeting this process, the treatment will help muscles relax more easily, reduce weakness, and slow down the progression of the disease.
Because this is a Phase 3 trial, it is one of the final stages of research needed before health authorities can decide whether the medicine should be approved for general use. The study compares AOC 1001 against a harmless saltwater solution (a placebo). By comparing the two groups, researchers can accurately measure whether any improvements in muscle function and daily quality of life are truly caused by the new medicine.
Key takeaways
- The study tests a new drip treatment called AOC 1001 for myotonic dystrophy type 1.
- It aims to find out if the drug improves muscle stiffness, grip strength, and mobility.
- Participants are randomly assigned to receive either the active drug or a saltwater placebo.
- The trial lasts for around 54 weeks and involves regular clinic visits and muscle tests.
- Taking part is completely voluntary and you can leave at any point.
Who may be eligible?
This trial is open to adults aged 18 and older who have been diagnosed with myotonic dystrophy type 1. Men and women can both take part, provided they meet the health and safety requirements set by the research team.
To join, participants must be able to complete basic muscle tests, such as opening their hands and walking short distances. The study team will review your full medical history, current medications, and general health to confirm whether the trial is safe and appropriate for you.
People with certain other severe medical conditions, or those who are pregnant or breastfeeding, may not be able to participate. Your trial doctor will go through all the detailed criteria with you before any study procedures begin.
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.
- I am aged 18 or older.
- I have a confirmed diagnosis of myotonic dystrophy type 1 (DM1).
- I experience muscle stiffness or weakness, such as difficulty opening my hands after gripping.
- I am able to attend regular clinic visits over a 54-week period.
- I am willing to receive treatments through an intravenous (arm vein) drip.
What does participation involve?
Taking part in this trial lasts for at least 54 weeks (just over one year). You will need to make regular visits to a specialist hospital or research clinic. During these visits, the study medicine or a dummy saline solution will be given as a slow drip into a vein in your arm.
At your appointments, the medical team will carry out regular health checks. These include checking your blood pressure, taking blood and urine samples, and monitoring your heart. You will also take part in specific physical tests. One key test uses video to measure how quickly you can open your hand after making a tight fist. Other assessments include simple walking tests, grip strength measurements, and questionnaires about your energy levels, daily activities, and overall quality of life.
Travel expenses are often reimbursed, and the trial team will guide you through every step of your appointments and assessments.
Potential risks and benefits
Locations (7)
- —UnverifiedNetherlands
- —UnverifiedFrance
- —UnverifiedSpain
- —UnverifiedDenmark
- —UnverifiedItaly
- —UnverifiedIreland
- —UnverifiedGermany
Common questions
What is a placebo and why is it used?
A placebo is a harmless substance, in this case simple saltwater, that looks identical to the real medicine. It is used so researchers can compare results fairly and see if the study drug genuinely works better than no treatment at all.
Will I know if I am getting the real medicine?
No. This is a 'double-blind' trial, which means neither you nor the doctors running the tests will know which treatment you are receiving until the study is finished. This prevents bias.
How is the treatment given?
Both the study drug and the placebo are given as an infusion, which means liquid is slowly delivered through a small tube and needle into a vein in your arm.
What is the hand opening test?
The test measures how many seconds it takes for your fingers to fully open and relax after squeezing your hand tightly shut. It helps doctors see if muscle stiffness is improving.
Can I leave the study if I change my mind?
Yes. Participation is completely voluntary. You can withdraw at any point, and your usual healthcare and relationship with your doctors will not be affected.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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